Tag Archives: OSDD

Making ‘National Policy For Rare Diseases’ More Meaningful With ‘Orphan Drugs Act’

Posted on by
Reply

In November 2016, while hearing a related case, the Delhi High Court reportedly directed the Union Government to finalize and implement a policy on rare diseases, with a provision for free treatment with the expensive medications, as will be required by patients.

Earlier in 2014, while passing the judgement in response to a petition filed by a seven-year-old son of a rickshaw puller seeking affordable treatment for the rare disease that he is suffering from, the Delhi High Court concluded that, “every person has a fundamental right to quality health care that is affordable, accessible and compassionate.”

Currently, the treatment for rare diseases costs the patients an arm and a leg, ranging between Rs. 40 lakhs (US$ 62,000 approx.) to Rs 1.70 crores (US$ 267,000 approx.), which is way beyond the reach of most Indians.

Subsequently, on May 26, 2017, the Ministry of Health and Family Welfare filed an affidavit before the honorable Court, submitting a copy of the National Policy for Rare Diseases 2017, stating that it will aim to facilitate effective diagnosis and affordable treatment. This development is indeed good news, especially considering around 6 to 8 percent of the world population suffer from ‘rare diseases’, and India is no exception.

The key highlights of the new policy:

As per available information, following are the 10 major highlights of the National Policy for Rare Diseases 2017:

  • The Union Government to create a corpus with an initial funding of Rs 100 crores (US$ 16 million). The State Governments would also provide for a similar fund with a 60 percent contribution from the Centre. This corpus is primarily for the treatment of genetic disorders, excluding rare blood diseases, such as, thalassemia and sickle-cell anemia.
  • For the sustainability of the corpus, Public Sector Units and Corporates will be encouraged for the contribution in these earmarked funds, as part of their corporate social responsibility.
  • Appropriate institutions will be accredited by the government for diagnosis and treatment of rare diseases.
  • To ensure adequate availability and reasonable affordability of the drugs for rare diseases, the Drug Controller General of India (DCGI) will consider amending the Drug and Cosmetics Act with requisite provisions to make clinical trials and import of ERTs possible.
  • The Department of Financial Services to ensure coverage of rare diseases under insurance schemes.
  • Employees State Insurance Corporation (ESIC) will explore whether the ceiling limit of funding the treatments for rare diseases can be increased through suitable amendments.
  • The policy recognizes that rare diseases are, in most cases, serious, chronic, debilitating and life-threatening, often requiring long – specialized treatments, and may also lead to some form of handicap, at times extremely severe in nature.
  • About 50 percent of new cases of rare diseases are in children and responsible for 35 percent of deaths before the age of one, 10 percent between the ages of one, and five years and 12 percent between five and 15 years.
  • As a preventive measure, the policy may consider the feasibility of providing pre-conception and ante-natal genetic counselling and screening programs for diagnosing genetic disorders, which would provide a choice to parents about giving birth to children with genetic disorders, especially for families that have a diagnosed genetic disorder, or a high risk profile for it.
  • The policy gives Indian Council for Medical Research (ICMR) the responsibility of creating a patient registry, as India has no epidemiological data on rare diseases.

Rare diseases – definition:

There is no universal definition of rare diseases. For example, while the US defines a rare disease as one that affects less than 200,000 people nationwide, in China, this number changes to 1 in 500,000 people (or neonatal morbidity of less than 1 in 10,000). India doesn’t yet have a clear definition for the same – not even in its new policy for rare diseases.

However, according to Rare Diseases India (RDI) – a foundation for research on rare diseases and disorders, any disease having fewer than 100 patients per 100,000 population fall into this category. Whereas, those ones affecting 2 patients per 100,000 population are described as ultra-rare diseases.

Rare Diseases in India:

The Organization for Rare Diseases in India (ORD), states that 1 in 20 Indians is affected by such diseases. About 6000 to 8000 rare diseases, mostly genetic in nature, have been identified in India. It was initially estimated that over 31 million Indians are suffering from such disorders in the country, many of which still do not have any cure. Moreover, epidemiological data for most of these ailments is hardly available.

To increase awareness for rare diseases, Rare Diseases Day was observed for the first time in India (New Delhi) on February 28, 2010.

Orphan diseases and orphan drugs:

According to RDI, rare diseases are often referred to as ‘orphan’ diseases. Consequently, the drugs that are specifically developed to treat ‘orphan’ or ‘rare disease conditions’ are called ‘orphan drugs’. The reason being, pharma companies do not generally take such drugs through further stages of development for market launch, or in other words, these are orphaned for economic considerations, though are important to save many precious lives.

Need to encourage orphan drug development in India:

According to SanOrphan SA, Geneva, Switzerland, around 65 percent of rare diseases is serious and disabling. Interestingly, about 250 new rare diseases are discovered each year, corresponding to five new rare diseases per week. As the scenario is no different in India, it prompts the need to encourage development of effective and affordable orphan drugs in the country.

However, without appropriate ecosystem being in place, developing an orphan drug in India, specifically to treat a very small number of such patient populations, through a cost intensive R&D initiative with a low potential of return on investments, is indeed a challenging proposition for many pharma players. Although, in the western world, this trend has started changing now, driven by various other commercial reasons.

Why should ‘Orphan Drugs Act’ follow the National Policy on rare diseases?

National Policy for Rare Diseases is undoubtedly a good beginning, though was brought under the directive of Delhi High Court. Nevertheless, to encourage ‘Orphan Drugs’ development within the country, a robust ‘Orphan Drugs Act’ should now logically follow.

One may well ask, why is this Act is so necessary in India? This is because, the new ‘National Policy for Rare Diseases’ charts just the pathway of a course of action that the Government is planning to take in this area. Policies, as we know, though, are a set of well-articulated intents, do not guarantee that these will be successfully followed to achieve the pre-set long-term goals. Whereas, all legislative Acts or duly enacted laws, are legally enforceable. It is worth noting, while the national policies can be formulated by the government, an Act must be passed by the lawmakers in the Parliament.

Consequently, it is now a well-accepted fact that ‘Orphan Drugs Act’ encourages development of drugs for rare diseases. In an article titled, “What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis”, published by the National Center for Biotechnology Information (NCBI), U.S, National Library of Medicine, the authors highlighted that in the U.S. 1138 orphan drugs were designated and 148 received marketing approval, of which 38 (26 percent) were for pediatric diseases, from 2000 to 2009. The percentage of approvals for pediatric products increased from 17.5 (10 of 57) in the first half of the decade, as compared to 30.8 (28 of 91) in the second half. Based on these data, the paper concluded that the incentives provided in the ‘Orphan Drugs Act (ODA)’ of the United States of America, have led to increased availability of specific drugs for the treatment of ‘Rare Diseases’ in the country.

Other countries did – why not India?

1983 signaled the importance of ‘Orphan Drugs’ with the ‘Orphan Drugs Act (ODA) in the U.S.A. A decade after, in 1993, Japan took similar initiative followed by Australia in 1999. Currently, Singapore, South Korea, Canada and New Zealand are also having their country specific ODAs.

Following similar footsteps, India should also encourage its domestic pharmaceutical industry to get engaged in research to discover drugs for rare diseases by putting an ‘Orphan Drugs Act’ in place, extending financial support, tax exemptions and regulatory concessions like smaller and shorter clinical trials, among several other areas, without delay.

Opportunities galore:

The above constraints in the development of orphan drugs have now been turned into an opportunity galore by the global pharma industry, where the domestic players should not lag much behind. Orphan drugs, backed by adequate financial incentives provided by laws in different countries, are now seen as a research and development priority to significantly boost the top and the bottom-line of pharma business.

As IgeaHub has highlighted, orphan drugs, though, cater to a small patient pool, the remunerative price of these drugs offsets the commercial challenges, as mentioned earlier. For example, in 2010, Soliris, which treats paroxysmal nocturnal hemoglobinuria (PNH) that affects 1 out of 500,000, was considered as the industry’s most expensive drug amounting to US$ 409,000 per year of treatment, which generated a total of US$ 541 million revenue for Alexion Pharmaceuticals in that year. In 2012, Soliris recorded a sales turnover of US$ 1.13 billion, which is expected to cross the mark of US$ 3.40 billion in 2018. Further, in 2012, the top selling orphan drug in the USA – Rituxan of Roche – used for the treatment of chronic lymphocytic leukemia, generated US$ 7.15 billion in total sales. Post patent expiry, in 2018, the same drug is expected to yield a revenue of US$ 6.99 billion.

The market:

Evaluate Pharma’s Orphan Drug Report 2017 estimates the worldwide Orphan Drug Sales of total US$ 209 billion, with CAGR of +11 percent for 2017 to 2022 period, which is double of the overall prescription Market Growth. Excluding generics orphan drugs are set to contribute 21.4 percent of Worldwide Prescription Sales by 2022.

Big pharma dominates this segment. Seven of the top 10 companies’ orphan drug sales are from global industry players, who have won approval for their biggest products in various niche indications.

Other commercial benefits:

Thomson Reuters reported additional commercial opportunities with an appropriate ODA, which in the United States are as follows:

  • 15 percent of the ‘Orphan Drugs’ analyzed by them had subsequent launches for other rare illnesses.
  • 6 out of the top 10 ‘Orphan Drugs’ had more than one rare disease indication, with an average peak sales of US$ 34.3 billion in overall sales potential, against around US$ 8.1 billion of the same for drugs with single indication.
  • Time taken for Clinical Trials (CT) focused on orphan drugs is significantly shorter with much quicker review time than trials involving non-orphan drugs.

Conclusion:

Some of the ‘orphan diseases’ are now being diagnosed also in India, and with precision. As the nation takes rapid strides in the medical science, more of such rare diseases are likely to be diagnosed in our country. The global pharma industry has already started taking rapid strides in this area, supported by ODA in various countries. Similar opportunities, both for the patients, as well as, for the industry, need to be made available in India too.

One of the ways to encourage the orphan drug development in India is to follow the model of the Council of Scientific and Industrial Research (CSIR) for ‘Open Source Drug Discovery’ (OSDD)’ with both global and local partnerships and collaboration.

However, speedy enactment of an appropriate ODA for the country, providing adequate financial incentives to the pharma players, for developing and marketing such drugs, both in the local and global markets, at a reasonably affordable price, would go a long way, and be a win-win situation for all.

Alongside, leveraging the knowledge of OSDD acquired by the CSIR, and framing a robust win-win Public Private Partnership (PPP) model to discover and commercialize the orphan drugs, India could well demonstrate the zeal of the country to move beyond the National Policy for Rare Diseases. In that process, it would be able to offer more meaningful and sustainable benefits, both to the domestic pharma industry and the patients, alike, for a long time to come.

By: Tapan J. Ray

Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.

Awaiting The Two To Tango: Pharma Innovation And Public Health Interest

Posted on by
Reply

“The rewards for the breakthrough drug discovery must be substantial, but if prices are the only mechanism through which returns on research flow, affordability will be compromised,” articulated an article titled, ‘Pharmaceutical Policy Reform – Balancing Affordability with Incentives for Innovation’, published in The New England Journal of Medicine (NEJM) on February 25, 2016.

The article arrived at this conclusion, on the backdrop of the high prices of prescription drugs becoming an issue of paramount concern, not just in the United States, but across the world. This concern is so acute that it found its way into policy proposals from both the prime candidates, in the American Presidential election held on November 8, 2016.

Through last several decades, healthcare sector in general and particularly the pharmaceutical industry, witnessed many innovations that cure and effectively manage ailments to improve the general quality of life. It enormously impacted the lives of many in the developed countries, and a few others which offer high quality Universal Health Care in a comprehensive format, for all.

A trickle-down impact:

Nevertheless, even no more than its just a trickle-down impact, helped increase overall life expectancy of the population in many developing and poor countries, mostly driven by the expanding number of cheaper generic drugs, fueled by more treatment and disease management options.

The paper titled, ‘World Population Prospects – The 2015 Revision’ of the Department of Economic and Social Affairs, Population Division of the United Nations’ reported that the life expectancy at birth rose by 3 years between 2000-2005 and 2010-2015, that is from 67 to 70 years. All major areas shared in the life expectancy gains over this period, but the greatest increases were in Africa, where life expectancy rose by 6 years in the 2000s, after rising by only 2 years in the previous decade.

Similarly, the global life expectancy at birth is projected to rise from 70 years in 2010-2015 to 77 years in 2045- 2050 and to 83 years in 2095-2100. Africa is projected to gain about 19 years of life expectancy by the end of the century, reaching 70 years in 2045-2050 and 78 years in 2095-2100. Such increases are contingent on further reductions in the spread of HIV, and combating successfully other infectious as well as non-communicable diseases.

The availability of cheaper generics gave some respite:

Out of a total population of 7.3 billion, as the above report says, the World Bank estimated that in 2013, 767 million people still lived on less than US$ 1.90 a day. Unfortunately, despite the greater availability of a large variety of cheaper generic drugs, the basic health care remains elusive to hundreds of millions of people in the world.

What causes more concern is the fact that 6 percent of people in low and middle-income countries are tipped into or pushed further into extreme poverty because of health spending, as the June 12, 2015 report of the World Health Organization (W.H.O) and the World Bank highlights. W.H.O has estimated that over a billion population of the world still suffer from neglected tropical diseases.

How many people benefitted from pricey patented drugs?

Nevertheless, despite so much innovation in the pharma industry, access to these new drugs remains elusive to a large section of even some the most developed nations, such as the United States, as they can’t afford these high-priced drugs. The overall situation, in this regard, is going from bad to worse. For example, the March 16, 2015 study published in the Mayo Clinic Proceedings reveals that the average annual cost of cancer drugs increased from roughly US$ 10,000 prior to 2000 to an astounding over US$  100,000 by 2012.

Further, an August 31, 2015 article published in the ‘Health Affairs’ also gave examples of Biogen Idec’s multiple sclerosis drug, Tecfidera, which costs US$ 54,900 per patient per year; hepatitis C cures from Gilead Sciences, with a sticker price of $84,000 per patient; and Orkambi, a cystic fibrosis drug from Vertex Pharmaceuticals approved this month, priced at a whopping US$ 259,000 per year. A Kaiser Health Tracking Poll last July 2015 found that 73 percent of Americans find the cost of drugs to be unreasonable, and most blamed drug manufacturers for setting prices too high, the article stated.

The health care scenario in India is no better:

A study conducted by the ‘National Sample Survey Organization (NSSO)’ from January to June 2014, which was the 71st round of the ‘National Sample Survey’, and published in the ‘Health in India’ report, narrates a very gloomy picture for India, especially for a clear majority of those who incur ‘out of pocket’ expenses on medicines. The report states, out of all health expenditure, 72 percent in rural and 68 percent in urban areas was for buying medicines for non-hospitalized treatment.

Thus, many patients cannot afford health services, even when these are needed the most. As many as 68 percent of patients in urban India and 57 percent in rural areas attributed “financial constraints” as the main reason to take treatment without any medical advice, the report adds.

In this situation, the challenges that the Governments and the civil society are facing in many developing, and to some extent even in some developed countries, though for different reasons, are multi-factorial. It has been well established that the humongous global health care challenges are mostly of economic origin.

Pharma innovation benefitted the developed countries more:

A study  titled, ‘Pharmaceutical innovation and the burden of disease in developing and developed countries’ of Columbia University and National Bureau of Economic Research, to ascertain the relationship across diseases between pharmaceutical innovation and the burden of disease both in the developed and developing countries, reported that pharmaceutical innovation is positively related to the burden of disease in the developed countries but not so in the developing countries.

Making the two to tango:

These facts prompt the need to make the pharma innovation and public health interest to tango. Several suggestions have been made and initiatives taken in this direction. Some of which are as follows:

  • Responding to this need, in 2006 W.H.O created the ‘Intergovernmental Working Group on Public Health, Innovation and Intellectual Property (IGWG)’. The primary focus of IGWG is on promoting sustainable, needs-driven pharmaceutical R&D for the diseases that disproportionately affect developing countries. One positive effect of this global debate is that some global pharmaceutical companies have initiated their R&D activities for neglected tropical diseases, such as, Malaria and Tuberculosis. Many charitable organizations like, Bill & Melinda Gates Foundation and Clinton Foundation, are allocating significant funds for this purpose.
  • A paper  titled, “Optional reward for new drugs for developing countries” published by the Department of Economics, University of Calgary, Institute of Health Economics, proposed an optional reward fund for pharmaceutical innovation aimed at the developing world to the pharmaceutical companies, which would develop new drugs while ensuring their adequate access to the poor. The paper suggests that innovations with very high market value will use the existing patent system, as usual. However, the medicines with high therapeutic value but low market potential would be encouraged to opt for the optional reward system. It was proposed that the optional reward fund should be created by the governments of the developed countries and charitable institutions to ensure a novel way for access to innovative medicines by the poor.
  • ‘Open Innovation’ or the ‘Open Source Drug Discovery (OSDD)’ is another model of discovering a New Chemical Entity (NCE) or a New Molecular Entity (NME). Imbibing ‘Open Innovation’ for commercial results in pharmaceuticals, just has what has happened to android smartphones, would encourage drug discovery initiatives, especially for the dreaded disease like cancer, to make these drugs affordable for a very large section of people across the globe. In this model, all data generated related to the discovery research will be available in the open for collaborative inputs. In ‘Open Innovation’, the key component is the supportive pathway of its information network, which is driven by three key parameters of open development, open access and open source. This concept was successfully used in the ‘Human Genome Project’ where many scientists, and microbiologists participated from across the world to sequence and understand the human genes. Currently, pharmaceutical R&D is a well-protected in-house initiative of innovator global companies to maximize commercial benefits. For this reason, only a limited number of scientists working for the respective innovator companies will have access to these projects. In India, the Council of Scientific and Industrial Research (CSIR) is the champion of the OSDD movement, locally. CSIR believes that for a developing country like India, OSDD will help the common man to meet his or her unmet medical needs in the areas of mainly neglected tropical diseases.

Conclusion:

Thus, the ongoing heated debate on Innovation, Intellectual Property Rights (IPR) and Public Health Interest is gathering steam all over the globe.

Argumentative Indians are also participating in this raging debate. I reckon rightly so, as India is not only the largest democracy of the world contributing 16.7 percent of the global population, it is also afflicted with 21 percent of the global burden of disease. Considering this, the reason for similar heated debate in our country is indeed no-brainer to anyone.

Many would possibly not disagree, both encouraging innovation and safeguarding the public health interest are equally important to any society, be it in the developed nations or developing countries. Nevertheless, some constituents of ‘Big Pharma’ and their trade association still highlight that ensuring access to high price innovative drugs is the responsibility of the respective Governments. Any other regulatory mechanism to bring down such prices will be construed as a barrier to encouraging, protecting and rewarding innovation.

Be that as it may, most other stakeholders, across the world, especially the patients, are awaiting these two goals to tango. From that point, I reckon, giving a quick shape to commercially well-tested initiatives, such as, ‘Open Innovation’ model could well be an important step to ensure access to innovative new medicines for a larger number of patients of the world, meeting their unmet medical needs with greater care.

By: Tapan J Ray

Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.

A Potential Game Changer For Pharma R&D

Posted on by
Reply

The ghost of ‘Patent Cliff’ has been haunting the ‘Big Pharma’ since quite some time. This situation has been further aggravated by cost containment pressures of various Governments both in the developed and the emerging markets together with contentious issues on Intellectual Property Rights (IPR).

The ‘dream run’ that the innovator companies enjoyed in launching patented products so frequently and making many those blockbuster drugs of billions of dollars, is no longer a reality.

According to the findings of ‘Pharmaceutical R&D returns performance’ by Deloitte and Thomson Reuters of December 2012, the R&D Internal Rate of Return (IRR) of leading pharmaceutical companies had fallen to 7.2 percent in 2012 from 7.7 percent in 2011.

Many would, therefore, tend to believe that the paradigm is changing significantly. The new paradigm in the brand new millennium throws some obnoxious challenges, including some related to IPR, triggering a process of churning in the global pharma industry. Some astute CEOs of ‘Big Pharma’, having a deep introspection, are bracing for restructuring, not just in the business processes, but also in the process of organizational behavior, mindset, ethics and values. Unfortunately, there are many who seem to believe that this giant wheel of change can be put on the reverse gear again with might.

A new PPP initiative in pharma research:

This trying situation calls for collaborative initiatives to achieve both knowledge and cost synergies for a quantum leap in harnessing R&D output.

One such big laudable initiative has come to the fore recently in this arena. Having experienced something like the ‘law of diminishing return’ in pursuit of high resource intensive R&D projects aimed at critical disease areas such as Alzheimer’s, 10 big global pharma majors reportedly decided in February 2014 to team up with the National Institutes of Health (NIH) of the United States in a ‘game changing’ initiative to identify disease-related molecules and biological processes that could lead to future medicines.

This Public Private Partnership (PPP) for a five-year period has been named as “Accelerating Medicines Partnership (AMP)”. According to the report, this US federal government-backed initiative would hasten the discovery of new drugs in cost effective manner focusing first on Alzheimer’s disease, Type 2 diabetes, and two autoimmune disorders: rheumatoid arthritis and lupus. The group considered these four disease areas among the largest public-health threats, although the span of the project would gradually expand to other diseases depending on the initial outcome of this project.

Not the first of its kind:

AMP is not the first PPP initiative of its kind. The Biomarkers Consortium was also another initiative, not quite the same though, of a major public-private biomedical research partnership managed by the Foundation for the NIH with broad participation from a variety of stakeholders, including government, industry, academia, patient advocacy groups and other not-for-profit private sector organizations.

Open innovation strategy of GlaxoSmithKline (GSK) to discover innovative drugs for malaria is yet another example, where GSK collaborated with the European Bioinformatics Institute and U.S. National Library of Medicine to make details of the molecule available to the researchers free of cost with an initial investment of US$ 8 million to set up the research facility in Spain, involving around 60 scientists from across the world to work in this facility. 

Nearer home, ‘Open Source Drug Discovery (OSDD)’ project of the Council of Scientific and Industrial Research (CSIR) is a now a global platform to address the neglected tropical diseases like, tuberculosis, malaria, leishmaniasis by the best research brains of the world working together for a common cause.

Challenges in going solo:

In this context, it is worth mentioning that the CEO of Sanofi, Chris Viehbacher reportedly said in an interview on April 15, 2013 that his company “Won’t push hard to find an Alzheimer’s treatment because the science isn’t advanced enough to justify the costs to develop a drug. Therefore, Sanofi definitely won’t commit major resources seeking to discover an Alzheimer’s therapy.” He further stated, “I think we have to do a lot more basic science work to understand what’s going on. We really, at best, partially understand the cause of the disease. It’s hard to come up with meaningful targets.”

The above report also mentioned that the first Alzheimer’s drugs, should they prove successful, would lead to a market worth US$ 20 billion as estimated in 2012.

Long desired OSDD model:

The new AMP R&D model in the United States seems to have derived its impetus from the “open-source” wave that has swept the software industry. Keeping that spirit unchanged, in this particular ‘open source’ model too, the participants would share all scientific findings with the public and anyone would be able to use these results freely for their own research initiatives.

The collaborators of this PPP project are expected to gain a better understanding of how each disease type works, and thereafter could make use of that collaborative knowledge to discover appropriate new molecules for the target disease areas.

AMP is also expected to arrive at methods to measure a disease progression and its response to treatment much more precisely. This will enable the pharma participants getting more targets right and early, thereby reducing the high cost of failures. Just to cite an example, there have been reportedly 101 failures since 1998 in late-stage clinical trials by Pfizer, J&J and Elan Corp.

Commendable initiative in the uncharted frontier:

The ‘open source’ AMP initiative of ‘Big Pharma’ in the uncharted frontier is indeed very unusual, as the innovative drug companies are believed to be not just quite secretive about the science that they are engaged in, but also near obsessive in pursuing and clinging-on to the Intellectual Property Rights (IPR) through patents for each innovative steps related to potential new drugs.

It is worth noting that like any OSDD model, this PPP agreement also denies the participating players from using any discovery for their own drug research up until the project makes all data public on that discovery.

However, as soon as the project results will be made public, fierce competition is expected all around to develop money-spinning winning drugs.

Participating companies:

Ten pharma companies participating in AMP are reportedly, AbbVie, Biogen Idec, Bristol-Myers Squibb, GlaxoSmithKline, Johnson & Johnson, Eli Lilly, Merck & Co., Pfizer, Sanofi and Takeda. It is good to find within the participants some staunch business rivals. According to a report, a number of foundations, including the American Diabetes Association and the Alzheimer’s Association have also agreed to get involved in the project.

Some key non-participants:

For various different reasons some key pharma majors, such as, Amgen, Roche and AstraZeneca have decided not to participate in AMP.

AMP project and cost:

AMP reportedly has reportedly articulated its intent to: “Map molecular paths that each disease follows and to identify key points that could be targets for treatment. In Type 2 diabetes, for instance, researchers hope to catalog the genetic changes that raise or lower a person’s risk for developing the disease. It also will seek novel methods to measure each disease’s course while assessing if a potential drug is working. Being able to measure a disease’s progress in that way, could speed drug development by raising a company’s confidence that an experimental drug is working, or let it more quickly end a project if a drug isn’t working.”

The participating companies and the NIH have jointly agreed that the AMP would put together a research system on cost sharing basis by pooling the brightest minds who are experts on each disease, along with the best drug discovery laboratories, relevant data and samples from clinical trials to decipher the diseases in ways, which none of these pharma players has been able to achieve just yet on its own.

To achieve all these, the total cost has been estimated at roughly just US$ 230 million, as compared to US$135 billion that the global drug industry claims to spend in a year on R&D.

This should also be seen in context of a study of December 2012 carried out by the Office of Health Economics (OHE), UK with a grant from AstraZeneca, which estimated that the cost of developing new medicine has risen by ten times from US$100 million in the 1970s to as high as US$ 1.9 billion in 2011.

As a head honcho of a global pharma biggie had put it earlier, a large part of these R&D expenses are the costs of failure, as stated above.

Criticism:

As usual, criticism followed even for this path-breaking project. Critics have already started questioning the rationale of the choice of the above four disease areas, with an exception perhaps for Alzheimer’s and wondered whether the participating players are making use of the federal fund to push hard the envelope of their respective commercial intents.

Another new collaborative approach: 

In another recently announced collaborative initiative, though not of the same kind, where Merck & Co has reportedly entered three separate collaboration agreements to evaluate an immunotherapy cancer treatment that is part of a promising new class of experimental drugs that unleash the body’s immune system to target cancer cells.

Conclusion:

There could still be some hiccups in the process of effective implementation of the AMP project. Hope, all these, if any, will be amicably sorted out by the participants of stature for the benefits of all.

Be that as it may, ‘open source’ model of drug discovery, as believed by many, would be most appropriate in the current scenario to improve not only profit, but also to promote more innovative approaches in the drug discovery process.

On May 12, 2011, in an International Seminar held in New Delhi, the former President of India Dr. A.P.J. Abdul Kalam highlighted the need for the scientists, researchers and academics to get effectively engaged in ‘open source’ philosophy by pooling talent, patents, knowledge and resources for specific R&D initiatives from across the world for newer and innovative drugs.

According to available reports, one of the key advantages of the ‘open source’ model would be substantial reduction in the high cost of failures of R&D projects, which coupled with significant saving in time would immensely reduce ‘mind-to-market’ span of innovative drugs in various disease areas, making these medicines affordable to many more patients.

Thus, PPP initiatives in pharmaceutical R&D, such as AMP, are expected to have immense potential to create a win-win situation for all stakeholders, harvesting substantial benefits both for the pharmaceutical innovators and the patients, across the world.

By: Tapan J. Ray

Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.

 

 

 

 

R&D: Is Indian Pharma Moving Up the Value Chain?

Posted on by
Reply

It almost went unnoticed by many, when in the post product patent regime, Ranbaxy launched its first homegrown ‘New Drug’ of India, Synriam, on April 25, 2012, coinciding with the ‘World Malaria Day’. The drug is used in the treatment of plasmodium falciparum malaria affecting adult patients.  However, the company has also announced its plans to extend the benefits of Synriam to children in the malaria endemic zones of Asia and Africa.

The new drug is highly efficacious with a cure rate of over 95 percent offering advantages of “compliance and convenience” too. The full course of treatment is one tablet a day for three days costing less than US$ 2.0 to a patient.

Synriam was developed by Ranbaxy in collaboration with the Department of Science  and Technology of the Government of India. The project received support from the Indian Council of Medical Research (ICMR) and conforms to the recommendations of the World Health Organization (WHO). The R&D cost for this drug was reported to be around US$ 30 million. After its regulatory approval in India, Synriam is now being registered in many other countries of the world.

Close on the heels of the above launch, in June 2013 another pharmaceutical major of India, Zydus Cadilla announced that the company is ready for launch in India its first New Chemical Entity (NCE) for the treatment of diabetic dyslipidemia. The NCE called Lipaglyn has been discovered and developed in India and is getting ready for launch in the global markets too.

The key highlights of Lipaglyn are reportedly as follows:

  • The first Glitazar to be approved in the world.
  • The Drug Controller General of India (DCGI) has already approved the drug for launch in India.
  • Over 80% of all diabetic patients are estimated to be suffering from diabetic dyslipidemia. There are more than 350 million diabetics globally – so the people suffering from diabetic dyslipidemia could be around 300 million.

With 20 discovery research programs under various stages of clinical development, Zydus Cadilla reportedly invests over 7 percent of its turnover in R&D.  At the company’s state-of-the-art research facility, the Zydus Research Centre, over 400 research scientists are currently engaged in NCE research alone.

Prior to this in May 14, 2013, the Government of India’s Department of Biotechnology (DBT) and Indian vaccine company Bharat Biotech jointly announced positive results, having excellent safety and efficacy profile in Phase III clinical trials, of an indigenously developed rotavirus vaccine.

The vaccine name Rotavac is considered to be an important scientific breakthrough against rotavirus infections, the most severe and lethal cause of childhood diarrhea, responsible for approximately 100,000 deaths of small children in India each year.

Bharat Biotech has announced a price of US$ 1.00/dose for Rotavac. When approved by the Drug Controller General of India, Rotavac will be a more affordable alternative to the rotavirus vaccines currently available in the Indian market. 

It is indeed interesting to note, a number of local Indian companies have started investing in pharmaceutical R&D to move up the industry value chain and are making rapid strides in this direction.

Indian Pharma poised to move-up the value-chain:

Over the past decade or so, India has acquired capabilities and honed skills in several important areas of pharma R&D, like for example:

  • Cost effective process development
  • Custom synthesis
  • Physical and chemical characterization of molecules
  • Genomics
  • Bio-pharmaceutics
  • Toxicology studies
  • Execution of phase 2 and phase 3 studies

According to a paper titled, “The R&D Scenario in Indian Pharmaceutical Industry” published by Research and Information System for Developing Countries, over 50 NCEs/NMEs of the Indian Companies are currently at different stages of development, as follows:

Company Compounds Therapy Areas Status
Biocon 7 Oncology, Inflammation, Diabetes Pre-clinical, phase II, III
Wockhardt 2 Anti-infective Phase I, II
Piramal Healthcare 21 Oncology, Inflammation, Diabetes Lead selection, Pre-clinical, Phase I, II
Lupin 6 Migraine, TB, Psoriasis, Diabetes, Rheumatoid Arthritis Pre-clinical, Phase I, II, III
Torrent 1 Diabetic heart failure Phase I
Dr. Reddy’s Lab 6 Metabolic/Cardiovascular disorders, Psoriasis, migraine On going, Phase I, II
Glenmark 8 Metabolic/Cardiovascular /Respiratory/Inflammatory /Skin disorders, Anti-platelet, Adjunct to PCI/Acute Coronary Syndrome, Anti-diarrheal, Neuropathic Pain, Skin Disorders, Multiple Sclerosis, Ongoing, Pre-clinical, Phase I, II, III

R&D collaboration and partnership:

Some of these domestic companies are also entering into licensing agreements with the global players in the R&D space. Some examples are reportedly as follows:

  • Glenmark has inked licensing deals with Sanofi of France and Forest Laboratories of the United States to develop three of its own patented molecules.
  • Domestic drug major Biocon has signed an agreement with Bristol Myers Squibb (BMS) for new drug candidates.
  • Piramal Life Sciences too entered into two risk-reward sharing deals in 2007 with Merck and Eli Lilly, to enrich its research pipeline of drugs.
  • Jubilant Group partnered with Janssen Pharma of Belgium and AstraZeneca of the United Kingdom for pharma R&D in India, last year.

All these are just indicative collaborative R&D initiatives in the Indian pharmaceutical industry towards harnessing immense growth potential of this area for a win-win business outcome.

The critical mass:

An international study estimated that out of 10,000 molecules synthesized, only 20 reach the preclinical stage, 10 the clinical trials stage and ultimately only one gets regulatory approval for marketing. If one takes this estimate into consideration, the research pipeline of the Indian companies would require to have at least 20 molecules at the pre-clinical stage to be able to launch one innovative product in the market.

Though pharmaceutical R&D investments in India are increasing, still these are not good enough. The Annual Report for 2011-12 of the Department of Pharmaceuticals indicates that investments made by the domestic pharmaceutical companies in R&D registered an increase from 1.34 per cent of sales in 1995 to 4.5 percent in 2010. Similarly, the R&D expenditure for the MNCs in India has increased from 0.77 percent of their net sales in 1995 to 4.01 percent in 2010.

Thus, it is quite clear, both the domestic companies and the MNCs are not spending enough on R&D in India. As a result, at the individual company level, India is yet to garner the critical mass in this important area.

No major R&D investments in India by large MNCs:

According to a report, major foreign players with noteworthy commercial operations in India have spent either nothing or very small amount towards pharmaceutical R&D in the country. The report also mentions that Swiss multinational Novartis, which spent $ 9 billion on R&D in 2012 globally, does not do any R&D in India.

Analogue R&D strategy could throw greater challenges:

For adopting the analogue research strategy, by and large, the Indian pharma players appear to run the additional challenge of proving enhanced clinical efficacy over the known substance to pass the acid test of the Section 3(d) of the Patents Act of India.

Public sector R&D:

In addition to the private sector, research laboratories in the public sector under the Council for Scientific and Industrial Research (CSIR) like, Central Drug Research Institute (CDRI), Indian Institute of Chemical Technology (IICT) and National Chemical Laboratory (NCL) have also started contributing to the growth of the Indian pharmaceutical industry.

As McKinsey & company estimated, given adequate thrust, the R&D costs in India could be much lower, only 40 to 60 per cent of the costs incurred in the US. However, in reality R&D investments of the largest global pharma R&D spenders in India are still insignificant, although they have been expressing keenness for Foreign Direct Investments (FDI) mostly in the brownfield pharma sector.

Cost-arbitrage:

Based on available information, global pharma R&D spending is estimated to be over US$ 60 billion. Taking the cost arbitrage of India into account, the global R&D spend at Indian prices comes to around US$ 24 billion. To achieve even 5 percent of this total expenditure, India should have invested by now around US$ 1.2 billion on the pharmaceutical R&D alone. Unfortunately that has not been achieved just yet, as discussed above.

Areas of cost-arbitrage:

A survey done by the Boston Consulting Group (BCG) in 2011 with the senior executives from the American and European pharmaceutical companies, highlights the following areas of perceived R&D cost arbitrage in India:

Areas % Respondents
Low overall cost 73
Access to patient pool 70
Data management/Informatics 55
Infrastructure set up 52
Talent 48
Capabilities in new TA 15

That said, India should realize that the current cost arbitrage of the country is not sustainable on a longer-term basis. Thus, to ‘make hay while the sun shines’ and harness its competitive edge in this part of the world, the country should take proactive steps to attract both domestic as well as Foreign Direct Investments (FDI) in R&D with appropriate policy measures and fiscal incentives.

Simultaneously, aggressive capacity building initiatives in the R&D space, regulatory reforms based on the longer term need of the country and intensive scientific education and training would play critical role to establish India as an attractive global hub in this part of the world to discover and develop newer medicines for all.

Funding:

Accessing the world markets is the greatest opportunity in the entire process of globalization and the funds available abroad could play an important role to boost R&D in India. Inadequacy of funds in the Indian pharmaceutical R&D space is now one of the greatest concerns for the country.

The various ways of funding R&D could be considered as follows:

  • Self-financing Research: This is based on:
  1. “CSIR Model”: Recover research costs through commercialization/ collaboration with industries to fund research projects.
  2. “Dr Reddy’s Lab / Glenmark Model”: Recover research costs by selling lead compounds without taking through to development.
  • Overseas Funding:  By way of joint R&D ventures with overseas collaborators, seeking grants from overseas health foundations, earnings from contract research as also from clinical development and transfer of aborted leads and collaborative projects on ‘Orphan Drugs’.
  • Venture Capital & Equity Market:  This could be both via ‘Private Venture Capital Funds’ and ‘Special Government Institutions’.  If regulations permit, foreign venture funds may also wish to participate in such initiatives. Venture Capital and Equity Financing could emerge as important sources of finance once track record is demonstrated and ‘early wins’ are recorded.
  • Fiscal & Non-Fiscal Support: Should also be valuable in early stages of R&D, for which a variety of schemes are possible as follows:
  1. Customs Duty Concessions: For Imports of specialized equipment, e.g. high throughput screening equipment, equipment for combinatorial chemistry, special analytical tools, specialized pilot plants, etc.
  2. Income tax concessions (weighted tax deductibility): For both in-house and sponsored research programs.
  3. Soft loans: For financing approved R&D projects from the Government financial institutions / banks.
  4. Tax holidays: Deferrals, loans on earnings from R&D.
  5. Government funding: Government grants though available, tend to be small and typically targeted to government institutions or research bodies. There is very little government support for private sector R&D as on date.

All these schemes need to be simple and hassle free and the eligibility criteria must be stringent to prevent any possible misuse.

Patent infrastructure:

Overall Indian patent infrastructure needs to be strengthened, among others, in the following areas:

  • Enhancement of patent literacy both in legal and scientific communities, who must be taught how to read, write and file a probe.
  • Making available appropriate ‘Search Engines’ to Indian scientists to facilitate worldwide patent searches.
  • Creating world class Indian Patent Offices (IPOs) where the examination skills and resources will need considerable enhancement.
  • ‘Advisory Services’ on patents to Indian scientists to help filing patents in other countries could play an important role.

Creating R&D ecosystem:

  • Knowledge and learning need to be upgraded through the universities and specialist centers of learning within India.
  • Science and Technological achievements should be recognized and rewarded through financial grants and future funding should be linked to scientific achievements.
  • Indian scientists working abroad are now inclined to return to India or network with laboratories in India. This trend should be effectively leveraged.

Universities to play a critical role:

Most of Indian raw scientific talents go abroad to pursue higher studies.  International Schools of Science like Stanford or Rutgers should be encouraged to set up schools in India, just like Kellogg’s and Wharton who have set up Business Schools. It has, however, been reported that the Government of India is actively looking into this matter.

‘Open Innovation’ Model:

As the name suggest, ‘Open Innovation’ or the ‘Open Source Drug Discovery (OSDD)’ is an open source code model of discovering a New Chemical Entity (NCE) or a New Molecular Entity (NME). In this model all data generated related to the discovery research will be available in the open for collaborative inputs. In ‘Open Innovation’, the key component is the supportive pathway of its information network, which is driven by three key parameters of open development, open access and open source.

Council of Scientific and Industrial Research (CSIR) of India has adopted OSDD to discover more effective anti-tubercular medicines.

Insignificant R&D investment in Asia-Pacific Region:

Available data indicate that 85 percent of the medicines produced by the global pharmaceutical industry originate from North America, Europe, Japan and some from Latin America and the developed nations hold 97 percent of the total pharmaceutical patents worldwide.

MedTRACK reveals that just 15 percent of all new drug development is taking place in Asia-Pacific region, including China, despite the largest global growth potential of the region.

This situation is not expected to change significantly in the near future for obvious reasons. The head start that the western world and Japan enjoy in this space of the global pharmaceutical industry would continue to benefit those countries for some more time.

Some points to ponder:

  • It is essential to have balanced laws and policies, offering equitable advantage for innovation to all stakeholders, including patients.
  • Trade policy is another important ingredient, any imbalance of which can either reinforce or retard R&D efforts.
  • Empirical evidence across the globe has demonstrated that a well-balanced patent regime would encourage the inflow of technology, stimulate R&D, benefit both the national and the global pharmaceutical sectors and most importantly improve the healthcare system, in the long run.
  • The Government, academia, scientific fraternity and the pharmaceutical Industry need to get engaged in various relevant Public Private Partnership (PPP) arrangements for R&D to ensure wider access to newer and better medicines in the country, providing much needed stimulus to the public health interest of the nation.

Conclusion:

R&D initiatives, though very important for most of the industries, are the lifeblood for the pharmaceutical sector, across the globe, to meet the unmet needs of the patients. Thus, quite rightly, the pharmaceutical Industry is considered to be the ‘lifeline’ for any nation in the battle against diseases of all types.

While the common man expects newer and better medicines at affordable prices, the pharmaceutical industry has to battle with burgeoning R&D costs, high risks and increasingly long period of time to take a drug from the ‘mind to market’, mainly due to stringent regulatory requirements. There is an urgent need to strike a right balance between the two.

In this context, it is indeed a proud moment for India, when with the launch of its home grown new products, Synriam of Ranbaxy and Lipaglyn of Zydus Cadilla or Rotavac Vaccine of Bharat Biotech translate a common man’s dream of affordable new medicines into reality and set examples for others to emulate.

Thus, just within seven years from the beginning of the new product patent regime in India, stories like Synriam, Lipaglyn, Rotavac or the R&D pipeline of over 50 NCEs/NMEs prompt resurfacing the key unavoidable query yet again:

Has Indian pharma started catching-up with the process of new drug discovery, after decades of hibernation, to move up the industry ‘Value Chain’?

By: Tapan J. Ray

Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.

Open Innovation: Quo Vadis, Pharmaceutical R&D?

Posted on by
Reply

Is the Pharmaceutical R&D moving from the traditional models to much less uncharted frontiers?

Perhaps towards this direction, in November, 2010 in a report titled, “Open Source Innovation Increasingly Being Used to Promote Innovation in the Drug Discovery Process and Boost Bottomline”, Frost & Sullivan underscored the urgent need of the global pharmaceutical companies to respond to the challenges of high cost and low productivity in their respective Research and Development initiatives, in general.

‘Open Innovation’ model, they proposed, will be most appropriate in the current scenario to improve not only profit, but also to promote more innovative approaches in the drug discovery process.  Currently, on an average it takes about 8 to 10 years to bring an NCE/NME to market with a cost of around U.S$ 1.7 billion.

The concept of ‘Open Innovation’ is being quite successfully used by the Information Technology (IT) industry since nearly three decades all over the world, including in India.  Web Technology, the Linux Operating System (OS) and even the modern day ‘Android’ – the open source mobile OS, are excellent examples of ‘Open source innovation’ in IT.

In the sphere of Biotechnology Human Genome Sequencing is another remarkable outcome in this area.

On May 12, 2011, in an International Seminar held in New Delhi, the former President of India Dr. A.P.J. Abdul Kalam commented, “Open Source Drug Discovery (OSDD) explores new models of drug discovery”. He highlighted the need for the scientists, researchers and academics to get effectively engaged in ‘open source philosophy’ by pooling talent, patents, knowledge and resources for specific R&D initiatives from across the world. In today’s world ‘Open Innovation’ in the pharmaceutical R&D has a global relevance, especially, for the developing world of ‘have-nots’.

The ‘Open Innovation’ model: 

As the name suggest, ‘Open Innovation’ or the ‘Open Source Drug Discovery (OSDD)’ is an open source code model of discovering a New Chemical Entity (NCE) or a New Molecular Entity (NME). In this model all data generated related to the discovery research will be available in the open for collaborative inputs. The licensing arrangement of OSDD where both invention and copyrights will be involved, are quite different from any ‘Open Source’ license for a software development.

In ‘Open Innovation’, the key component is the supportive pathway of its information network, which is driven by three key parameters of open development, open access and open source.

As stated earlier, ‘Open Innovation’ concept was successfully used in the ‘Human Genome Project’ where a large number of scientists, and microbiologists participated from across the world to sequence and understand the human genes. However, this innovation process was first used to understand the mechanics of proteins by the experts of the Biotech and pharmaceutical industries.

The Objectives of ‘Open Innovation’: 

The key objective of ‘Open Innovation’ in pharmaceuticals is to encourage drug discovery initiatives, especially for the dreaded disease like cancer and also the neglected diseases of the developing countries to make these drugs affordable to the marginalized people of the world.

Key benefits of ‘Open Innovation’:

According to the above report of Frost & Sullivan on the subject, the key benefits of ‘Open Innovation’ in pharmaceuticals will include:

• Bringing together the best available minds to tackle “extremely challenging”   diseases

• Speed of innovation

• Risk-sharing

• Affordability

Some issues:

Many experts feel that the key issues for ‘Open Innovation’ model are as follows:

  • Who will fund the project and how much?
  • Who will lead the project?
  • Who will coordinate the project and find talents?
  • Who will take it through clinical development and regulatory approval process?

However, all these do not seem to be an insurmountable problem at all, as the  saying goes, ‘where there is a will, there is a way’.

Current Global initiatives for ‘Open Innovation’:

  1. In June 2008, GlaxoSmithKline announced in Philadelphia that it was donating an important slice of its research on cancer cells to the cancer research community to boost the collaborative battle against this disease. With this announcement, genomic profiling data for over 300 sets of cancer cell lines was released by GSK to the National Cancer Institute’s bioinformatics grid. It has been reported that over 900 researchers actively contribute to this grid from across the industry, research institutes, academia and NGOs. Many believe that this initiative will further gain momentum to encourage many more academic institutions, researchers and even smaller companies to add speed to the drug discovery pathways and at the same time make the NCEs/NMEs coming through such process much less expensive and affordable to a large section of the society, across the globe.
  2. The Alzheimer’s  Disease Neuroimaging Initiative (ADNI) is another example of a Private Public Partnership (PPP) project with an objective ‘to define the rate of progress of mild cognitive impairment and Alzheimer’s disease, develop improved methods for clinical trials in this area and provide a large database which will improve design of treatment trials’. 
  3. Recently announced ‘Open invitation’ strategy of GlaxoSmithKline (GSK) to discover innovative drugs for malaria is yet another example where GSK has collaborated with European Bioinformatics Institute and U.S. National Library of Medicine to make the details of the molecule available to the researchers free of cost with an initial investment of US $ 8 million to set up the research facility in Spain involving around 60 scientists from across the world to work in this facility.

‘Open Innovation’ in India: 

In India, Dr. Samir Brahmachari, the Director General of the Council of Scientific and Industrial Research (CSIR) is the champion of the OSDD movement. CSIR believes that for a developing country like India OSDD will help the common man to meet his unmet medical needs in the areas of neglected tropical diseases.

‘Open Innovation’ project of CSIR is a now a global platform to address the neglected tropical diseases like, tuberculosis, malaria, leishmaniasis by the best research brains of the world working together for a common cause.

To fund this initiative of the CSIR the Government of India has allocated around U.S $40 million and an equivalent amount of funding would be raised from international agencies and philanthropists.

Success of ‘Open Innovation’ initiative of CSIR: 

Sometime in late November 2009, I received a communication from the CSIR informing that their OSDD project, since its launch in September 2009, has crossed 2000 registered users in a very short span of time. The pace of increasing number of registered users indeed reflects the confidence that this initiative has garnered among the interested researchers across the world.

CSIR has indicated that the next big leap planned by them in the area of ‘Open Innovation’ is to completely re-annotate the MTb genome for which they have already launched a project titled ‘Connect to Decode’ 2010.

Conclusion: 

Currently pharmaceutical R&D is an in-house initiative of innovator global companies. Mainly for commercial security reasons only limited number of scientists working for the respective innovator companies will have access to these projects.

‘Open Innovation’ on the other hand, has the potential to create a win-win situation, bringing in substantial benefits to both the pharmaceutical innovators and the patients.

The key advantage of the ‘Open Innovation’ model will be substantial reduction in the costs and time of R&D projects, which could be achieved through voluntary participation of a large number of researchers/Scientists/Institutions in key R&D initiatives. This in turn will significantly reduce the ‘mind-to-market’ time of more affordable New Chemical/Molecular Entities in various disease areas.

Thus, to answer to ‘Quo Vadis, Pharmaceutical R&D’, I reckon, ‘Open Innovation’ model  could well be an important direction for tomorrow’s global R&D initiatives to improve access to innovative affordable Medicines to a larger number of ailing patients of the world, meeting their unmet medical needs, more effectively and with greater care.

Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.

From Cross-Licensing to ‘Patent Pools’ and… India: Will there be a ground swell?

Posted on by
Reply

Since many years, the global pharmaceutical industry has been making effective commercial use of cross-licensing, however, by and large, the industry still does not seem to be quite in favor of  ‘Patent Pools’ for various reasons.

The ‘Patent Pool’, as I understand is defined as, “an agreement between different owners, including companies, governments and academic bodies to make available patent rights on non-exclusive basis to manufacturers and distributor of drugs against payment of royalties.”

Thus one of the often repeated key benefits of the ‘Patent Pools’, as considered by its proponents, is that the system enables the use of innovation against payment of royalties, without the risk of patent infringement. Many believe that the concept of ‘Patent Pool’ can play an immensely useful role for productive use of Intellectual Property (IP) in the global pharmaceutical industry.

The difference between cross-licensing and ‘Patent Pools’:

The basic purposes of both Cross-Licensing and patent pools may appear to be similar, however the key difference is that in ‘Patent Pool’ system the patent owners usually agree to license to third parties who may not even contribute any patents to the pool. Moreover, ‘Patent Pools’ involve a large number of parties with its scope being narrow and well standardized.

“Patent Pools”- still a contentious issue:

The concept of ‘Patent Pools’ has become a contentious issue within the global pharmaceutical industry. Some opinion leaders vehemently argue that creation of a ‘patent pool’ will bring down the cost of any innovation significantly and save huge time, ensuring speedier and improved access to such medicines to a vast majority of ailing population across the world. This section of the experts also feels, “in the case of blocking patents as a commercial strategy, it would only be a reasonable method for making the innovation publicly available.”
In the midst of this high decibel debate, on February 13, 2009, ‘The Guardian’ reported the following comment of Andrew Witty, CEO of GlaxoSmithKline (GSK) on the same issue:
“GSK will put any chemicals or processes over which it has intellectual property rights that are relevant to finding drugs for neglected diseases into a patent pool, so they can be explored by other researchers”.
Andrew Witty in that interview also commented, “I think it’s the first time anybody’s really come out and said we’re prepared to start talking to people about pooling our patents to try to facilitate innovation in areas where, so far, there hasn’t been much progress… I think the shareholders understand this and it’s my job to make sure I can explain it. I think we can. I think it’s absolutely the kind of thing large global companies need to be demonstrating, that they’ve got a more balanced view of the world than short-term returns.”
Quoting Andrew Witty, ‘The Guardian’ reported, “his stance may not win him friends in other drug companies, but he is inviting them to join him in an attempt to make a significant difference to the health of people in poor countries”.
Yet another ‘out of box’ comment:
As if to prove ‘The Guardian’ right on their above comment, during his visit to India on March 2010, though in a slightly different context, Witty made the following comments, while answering a question of “The Economic Times”:
“I am relatively relaxed with the Indian regulatory environment. The government has made it clear about the direction to have an Intellectual Property (IP) mechanism and to be TRIPS compliant. Some people are unrealistic and want everything to change overnight. But we should be absolutely realistic about pricing to keep it affordable for India. If someone has the IP right, it does not mean that it should make it inaccessible for lower income people. Over the next 10-15 years India will become increasingly IP defined market.”
The rationale for ‘Patent Pools’ system:
Many experts in this area feel that the conventional patent system does not really work for the diseases of the poor, all over the world. Though the concept of ‘Patent Pools’ is quite new in the global pharmaceutical industry, this system is being very successfully and widely practiced within the Information Technology (IT) industry. ‘Patent Pool’ system, if effectively used, as stated earlier, can also help the global pharmaceutical companies to improve access of such medicines to many more developing countries of the world.

Key requirements for the ‘Patent Pools’:
Careful identification of various patents, which will be essential for the pool, will be one of the key requirements to initiate a ‘Patent Pool’ system. It makes the need to obtain individual patents, required in the process of a drug discovery, less important.

National Institute of Health (NIH), USA initiated the process:
On September 30, 2010, NIH became the first patent-holder to share its intellectual property with the Medicines Patent Pool, supported by UNITAID, by licensing a patent for ‘Darunavir’ to increase access of HIV and AIDS medicines to the suffering patients in the developing countries of the world.

UNITAID, an innovative global health financing mechanism is funded by a levy on airline tickets. This initiative was co-founded by the U.K, France, Norway, Brazil and Chile at the United Nations General Assembly in 2006 and buys drugs against HIV/AIDS, malaria and tuberculosis.
The above move of NIH towards the noble cause was appreciated by many all over the world, urging the global pharmaceutical industry, in general, to take a leaf out of it.

India was kept out of UNITAID “Patent Pool”:

In 2009-10, UNITAID reportedly had opposed the move to include countries like, India, China and Brazil from the proposed patent pool for AIDS drugs. At least seven civil society groups from India like, the Centre for Trade and Development, the National Working Group on Patent Laws, the All India Peoples Science Network openly stated that UNITAID does not intend to share the patent pool implementation plan with these civil society groups of India. They also alleged that this development in UNITAID will have a significant impact on the ability of Indian Pharmaceutical industry to manufacture low-cost versions of patented HIV/AIDS medicines for the developing countries of the world.

At that time, it was also reported that large global pharmaceutical players had indicated to UNITAID that they could contribute to the ‘patent pool’ on a selective basis, however, over 100 middle income countries such as India, Brazil and China should not have rights to manufacture generic versions of these HIV/AIDS medicines. They felt that ‘patent pool’ will be meaningless if poor countries, who do not have the capability to manufacture these medicines, are included in the process.

However, according to UNITAID, “the patent pool in no way a means to replace or override other provisions contained in the Trade Related Aspects of Intellectual Property Rights (TRIPS) Agreement or the Doha Declaration on TRIPS and Public Health. The patent pool represents an additional tool to increase access to HIV treatment, and an opportunity for patent holders to voluntarily contribute to the attainment of crucial health-related goals endorsed by the international community.”

GSK kick-started the process:

Andrew Witty of GSK is undoubtedly the first CEO of a global pharmaceutical company to announce a ‘Patent Pool’ system for research on 16 neglected tropical diseases like, tuberculosis, malaria, filariasis, leprosy and leishmaniasis. GSK has, in a real sense, kick started the process by putting more than 500 granted pharmaceuticals patents and over 300 pending applications in the ‘Patent Pool’.

J&J followed suit:

Johnson and Johnson (J&J) in January 2011 expressed its willingness to assist ‘Medicines Patent Pool Foundation (MPPF)’ to implement ‘Medicines Patent Pool (MPP)’, which aims to improve access to affordable and appropriate HIV medicines in developing countries. MPPF works through voluntary licensing of patents for public health interest, at the same time extending compensation to the innovator pharmaceutical companies.

‘Medicines Patent Pools’:

On April 7, 2011. ‘Intellectual Property Watch’ reported that the ‘Medicines Patent Pools’, an initiative to improve access to HIV drugs through voluntary licenses of patented drugs, have launched a new database of patent information on HIV medicines in developing countries. The database has been developed with the support of the World Intellectual Property Organization (WIPO) and Regional Patent Offices across the world. Intellectual Property Watch

Key issues with the ‘Patent Pools’ concept:
The report from a WHO conference held in April, 2006 ‘Innovation Strategy Today’ indicates that the start-up cost of a ‘Patent Pools’ for vaccines will be economically viable only if more than 25 participants holding relevant patents join the initiative.
Moreover, various types of litigation related to patents, which are being currently witnessed within the global pharmaceutical industry, could also be an impediment in getting more patents in the pool.

Recommended ‘General Principles’ for “Patent Pools”:
International Federation of Pharmaceutical Manufacturers and Associations (IFPMA), Switzerland, suggested the following guidelines for the ‘Patent Pool’ initiatives:
1. Patent pools should be voluntary associations of entities formed without coercion 2. Objectives of any patent pool should be clearly defined 3. Patent pools should complement rather than replace elements of existing intellectual property regimes 4. Rights and obligations of contributors and licensees of contributed rights should be clear 5. Patent pools should reduce transaction costs, and not increase administrative costs, relative to other options such as direct licensing
Conclusion:
There is certainly an urgent need to communicate more on how innovation and IPR could help rather than hinder public health. At the same time all stakeholders of the pharmaceutical industry need to come out with a robust solution to ever increasing problem of improving access to innovative medicines to the ailing population of the world, in the best possible way.
However, these are still very early days, before such a disrupting idea get widely accepted by the global innovators and implemented religiously not just for the ‘public health interest’, across the world, but also to create a sustainable business model to harvest ‘Fortune at the Bottom of the Pyramid’.

Only future will tell us whether or not the ‘Patent Pools’ initiatives become the footprints on the sands of time as the global pharmaceutical industry keeps  navigating through the challenges of change.

By: Tapan J Ray

Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.

Open Source Drug Discovery (OSDD) initiative for the tropical diseases by CSIR and cancer by GlaxoSmithKline deserves a big applaud and support from all concerned.

Posted on by
Reply

As the name suggest the ‘Open Source Drug Discovery (OSDD)’ is an open source code model of discovering a New Chemical Entity (NCE) or a New Molecular Entity (NME). In this model all data generated related to the discovery research will be available in the open for collaborative research inputs. The licensing arrangement of OSDD where both invention and copyrights will be involved, are quite different from any ‘Open Source’ license for a software development.

In OSDD, the key component is the supportive pathway of its information network, which is driven by three key parameters of open development, open access and open source.

The Objectives of OSDD:

The key objective of OSDD is to encourage drug discovery initiatives, especially for the neglected diseases of the world to make these drugs affordable to the marginalized people, especially of the developing world.

International initiative:

In June 2008, GlaxoSmithKline (GSK) announced in Philadelphia, “It was donating an important slice of its research on cancer cells to the cancer research community to boost the collaborative battle against this disease.”

With this announcement genomic profiling data for over 300 sets of cancer cell lines was released by GSK to the National Cancer Institute’s bioinformatics grid. It has been reported that around 1000 researchers actively contribute to this grid from across the industry, research institutes, academia and NGOs.

Many believe that the OSDD initiative will gain momentum to encourage many more academic institutions, researchers and even smaller companies to add speed to the drug discovery process and at the same time make the NCEs/NMEs coming through such process much less expensive and affordable to a large section of the society.

On an average it takes about 8 to 10 years to bring an NCE/NME to market with a cost of around U.S$ 1.7 billion.

OSDD in India:

In India, Dr. Samir Brahmachari, the Director General of the Council of Scientific and Industrial Research (CSIR) is the champion of the OSDD movement. CSIR believes that for a developing country like India, OSDD will help the common man to meet his unmet medical needs in the areas of neglected tropical diseases.

OSDD in India is a global platform to address the neglected tropical diseases like, tuberculosis, malaria, leishmaniasis by the best research brains of the world, together.

To fund the OSDD initiative of the CSIR the Government of India has allocated around U.S $40 million and an equivalent amount of funding would be raised from international agencies and philanthropists.

It has been reported that current priority of CSIR in its OSDD program is the tuberculosis disease area.

Why tuberculosis?

The published reports indicate, in every 1.5 minutes one person in India dies of tuberculosis and about 33 percent of the global population is infected primarily with Mycobacterium tuberculosis. The world is still quite far from having an effective vaccine or drug, which can offer long term protection against this dreaded disease.

Partnerships of Industry with belief in Open Source systems and models with CSIR in its OSDD project for tuberculosis, could help finding out a suitable answer to this long standing problem, sooner than later.

Success of OSDD initiative of CSIR:

Late November 2009, I received a communication from the CSIR informing that their OSDD project, since its launch in September 2009, has crossed 2000 registered users. The pace of increase in the number of registered users indeed reflects the confidence this initiative has generated among the interested researchers, the world over.

OSDD community of CSIR has several credits to be proud of including open peer review, open funding review, large number of real time data on open lab notebook.

CSIR has also indicated that the next big leap planned by them is to completely re-annotate the MTb genome for which OSDD has launched ‘Connect to Decode’ 2010 (http://crdd.osdd.net). They initially expected about 150 participants to join, but within a week, they got about 450 participants. That is really the strength of collaboration on OSDD!

Congratulations CSIR and its leader Dr. Samir Brahmachari.

By Tapan Ray

Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.

‘Patent Pool’ – is GSK setting a new trend for the global pharmaceutical industry?

Posted on by
Reply

On February 13, 2009, The Guardian reported that Andrew Witty, CEO of GlaxoSmithKline (GSK) announced some significant changes to the way his company will operate in the developing countries of the world.

GSK, as Witty said, will:

• “Cut its prices for all drugs in the 50 least developed countries to no more than 25% of the levels in the UK and US – and less if possible – and make drugs more affordable in middle- income countries such as Brazil and India.

• Put any chemicals or processes over which it has intellectual property rights that are relevant to finding drugs for neglected diseases into a “patent pool”, so they can be explored by other researchers.

• Reinvest 20% of any profits it makes in the least developed countries in hospitals, clinics and staff.

• Invite scientists from other companies, NGOs or governments to join the hunt for tropical disease treatments at its dedicated institute at Tres Cantos, Spain.”

Quoting Andrew Witty, The Guardian reported, “his stance may not win him friends in other drug companies, but he is inviting them to join him in an attempt to make a significant difference to the health of people in poor countries”.

“We work like crazy to come up with the next great medicine, knowing that it’s likely to get used an awful lot in developed countries, but we could do something for developing countries. Are we working as hard on that? I want to be able to say yes we are, and that’s what this is all about – trying to make sure we are even-handed in terms of our efforts to find solutions not just for developed but for developing countries,” Witty envisioned.

“I think the shareholders understand this and it’s my job to make sure I can explain it. I think we can. I think it’s absolutely the kind of thing large global companies need to be demonstrating, that they’ve got a more balanced view of the world than short-term returns,” he expressed Knowing full well that his comments will be considered as quite radical within the global pharmaceutical Industry.

The unorthodox young CEO of GSK continued, “I think it’s the first time anybody’s really come out and said we’re prepared to start talking to people about pooling our patents to try to facilitate innovation in areas where, so far, there hasn’t been much progress.”

Definition of ‘Patent Pool’:

The ‘Patent Pool’ is defined as, “an agreement between different owners, including companies, governments and academic bodies to make available patent rights on non-exclusive basis to manufacturers and distributor of drugs against payment of royalties”

Thus one of the often repeated key benefits of the ‘Patent Pool’, as considered by its proponent, is that the system enables the use of innovation against payment of royalties, without the risk of patent infringement.

The rationale for ‘Patent Pool’ system:

Many experts in this area feel that the conventional patent system does not really work for the diseases of the poor, all over the world. Though the concept of ‘Patent Pool’ is quite new in the global pharmaceutical industry, this system is being very successfully and widely practised within the Information Technology (IT) industry. ‘Patent Pool’ system, if effectively used, can also help the global pharmaceutical companies to improve their access to many more developing countries of the world.

GSK appears to have kick started the process:

Andrew witty of GSK is undoubtedly the first CEO of a global pharmaceutical company to announce a ‘Patent Pool’ system for research on 16 neglected tropical diseases like, tuberculosis, malaria, filariasis leprosy and leishmaniasis. GSK has, in a real sense, kick started the process by putting more than 500 granted pharmaceuticals patents and over 300 pending applications in the ‘Patent Pool’.

Key requirements for the ‘Patent Pool’:

Careful identification of various patents, which will be essential for the pool, will be one of the key requirements to initiate a ‘Patent Pool’ system. It makes the need to obtain individual patents, required in the process of a drug discovery, less important.

Key issues with the ‘Patent Pool’ concept:

It has been reported, from a WHO conference held in April, 2006 ‘Innovation Strategy Today’ worked out that the start-up costs of a ‘Patent Pool’ for vaccines will be economically viable only if more than 25 participants holding relevant patents join the initiative.

Moreover, various types of litigations, related to patents, which we are currently witnessing within the global pharmaceutical industry, could also be impediment in getting more patents in the pool.

Conclusion:

The initiative to create a ‘Patent Pool’ system in the global pharmaceutical industry, especially for the diseases of the poor, as enunciated by the CEO of GSK, is indeed a path breaking one. Such initiatives are likely to have very positive contribution in solving the problem of access to affordable medicines, especially in the developing world.

In fact, the Council of Science and Industrial Research of the Government of India, lead by its Director General, Dr. Samir Brahmachari has already undertaken similar initiatives in the country where global experts including academia are actively participating.

Though ‘Patent Pool’ is still an untested model in the global pharmaceutical industry, the recent announcement of GSK towards this direction does appear to offer a realistic and practical approach to address the critical global issue of improving ‘access to affordable innovative modern medicines’ to a vast majority of population in the developing countries of the world.

By Tapan Ray

Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.