‘Keep nose to the grindstone while lifting eyes to the hills.’ Quite a while ago, all-time global management guru – Peter Drucker used this essential acrobatic feat as an example, for the business strategists. This illustration signifies the criticality of harmonizing decisions affecting both the short and the long-term goals of an organization, for a sustainable business excellence.
In most recent times, the pharma major AstraZeneca that has virtually become one of the household names, for developing a Covid-19 vaccine candidate with the University of Oxford, has performed the above acrobatic feat – with precision. During the prevailing unprecedented health crisis, the Company has unequivocally proven that it remains on course – in achieving its dual objectives, as Drucker had prescribed in his management classic – ‘The Practice of Management.’
It happened in tandem – without getting overwhelmed by the disruptive forced of Covid pandemic, unlike most others. Immediately, the Company focused on an urgent objective of saving the humanity – by developing, manufacturing and delivering a Covid vaccine to the world, in a record time. This was possibly a relatively short-term goal. And closely followed the other – a critical strategic decision for the organization’s long-term sustainable business excellence.
I have discussed before, the Company’s first initiative – developing a Covid vaccine candidate with the University of Oxford. Hence, in this article, I shall focus on two other related areas:
- Deadly impact of rare diseases in some Covid infected young patients.
- Why not some large Indian companies also explore similar strategies as demonstrated by AstraZeneca – and the reasons behind the same?
Before going into those areas up front, let me start with a brief description of AstraZeneca’s intent to expand its footprint in the of area of rare diseases, besides immunology area to help treat rare types of cancer.
On December 12, 2020 AstraZeneca announced that to accelerate its strategic and financial development, the company will acquire Alexion valuing $39 billion. Subject to all statutory approvals, the deal is expected to close in the third quarter of 2021. Interestingly, Alexion’s top brand – Soliris, is the world’s one of the most expensive drugs in the world. It is prescribed to treat a rare – life-threatening blood disease paroxysmal nocturnal hemoglobinuria (PNH). Incidentally, rare diseases have also some significant relevance for Covid infected patients. Let me now recapitulate, some key aspects of rare diseases.
Some key aspects of rare diseases:
Rare Diseases (RD) – also referred to as Orphan Disease (OD), are diseases affecting a small percentage of the population, and include genetic diseases, rare cancers, infectious tropical diseases and degenerative diseases. There is no universally accepted definition of a rare disease, yet. Different countries define these differently. However, the common considerations in the definitions are, primarily, disease prevalence and to a varying extent – severity and existence of alternative therapeutic options.
Impact of some rare diseases in Covid infected patients:
Since the beginning of the Covid pandemic, people with underlying diseases, such as, hypertension, diabetes, cardiovascular and kidney disorders, are considered to fall in the high-risk group. They are more likely to have severe disease and complications and need to be extra cautious of the infection. Importantly, it has been recently reported that some rare diseases also increase risk of dying during Covid-19 pandemic at a younger age.
For example, as reported on December 07, 2020, recent studies indicate, rare autoimmune rheumatic diseases increase risk of dying during Covid-19 pandemic for younger patients. The researchers also found that women with rare autoimmune rheumatological diseases (RAIRD) had a greater increase in all-cause mortality rates during the pandemic when compared to men with RAIRD. However, there seems to be an India specific issue also in this situation, as well.
India specific issue for Covid infected patients with some rare diseases:
Some India specific issues on RD, could have a significant adverse impact on Covid infected patients in the country. One such critical issues is the ‘Baseline Knowledge of Rare Diseases in India.’ This fact was well captured in an important survey that was published with the same name, as an original article, in the ‘International Journal of Rare Diseases & Disorders,’ on November 06, 2019.
The study noted, among others:
- Although, rare diseases have recently received worldwide attention, the developing countries are seriously behind in regard to awareness, drug development, diagnosis, and social services, in this area. India, which has one-third of the world’s rare disease population, has no accurate assessment of the problem.
- The drugs for ‘Rare Diseases (RD), also called Orphan Drugs (OD)’, often cost exorbitant with difficulties in diagnosis and treatments.
- Indian policymakers want to find out the number of RD and the extent of the population suffering from them and help provide treatment for them, which is a challenging task with 1.45% GDP health care budget for 1.3 billion people.
- The health care professionals appear to have some awareness as compared with non-healthcare professionals, but even among health care professionals, only one third had a rudimentary understanding of RD and OD, whereas three-fourths have virtually no knowledge of RD.
- Forty-three percent of health professionals had not seen rare disease patients, and a large percent of practicing physicians had not seen even one rare disease patient in their entire professional practice.
Thus, it is clear from this survey that the most important issues are awareness and diagnosis, as many rare diseases are not diagnosed or possibly misdiagnosed. Besides, the survey also observed, since 1983, many global companies started developing orphan drugs after the Orphan Drug Act implementation. There is none at this time in India, although in 2017, the Drugs & Cosmetic Act. 1945 has been amended to include “rare diseases and orphan drugs”.
The National Policy on Rare Diseases flagged some of these facts:
The ‘National Policy on Rare Diseases 2020,’ for India, released by the Union Ministry of Health on February 07, 2020, acknowledged many of these important facts. It also said, ‘Considering the limited data available on rare diseases, and in the light of competing health priorities, the focus of the draft policy is on prevention of rare diseases as a priority as identified by experts.’
Interestingly, the first of such policy was prepared by India in 2017 and a committee was appointed to review it in 2028. However, recently published the National Policy on Rare diseases, has also noted one more important point. It noted: ‘Paradoxically, though rare diseases are of low prevalence and individually rare, collectively they affect a considerable proportion of the population in any country, which according to generally accepted international research is – between 6% and 8%.’ Currently, India, reportedly, doesn’t have any registry of rare disease, which has now been entrusted to the Indian Council of Medical Research (ICMR) in the National policy.
Common symptoms can hide underlying rare diseases, leading to misdiagnosis:
The above policy has also noted, rare diseases are characterized by a wide diversity of signs and symptoms that not only, reportedly, vary from disease to disease, but also from patient-to-patient suffering from the same disease. Importantly, relatively common symptoms can hide underlying rare diseases, leading to misdiagnosis.
During Covid treatment, similar circumstances could lead to a serious life-threatening situation. The 2020 RD Policy also reiterates: “Early diagnosis of rare diseases is a challenge owing to multiple factors that include lack of awareness among primary care physicians, lack of adequate screening and diagnostic facilities etc.” That said, yet another key question arises – will developing and marketing such drugs be profitable for the pharma industry?
Will developing such drugs be profitable for the pharma industry?
It is worth noting that the National Policy on Rare Diseases 2020, aims more at lowering the incidence and prevalence of rare diseases based on an integrated and comprehensive prevention strategy, rather than ensuring patient access to affordable treatments. Nonetheless, it also says, within the constraints on resources and competing health care priorities, India will try to enable access to affordable health care to patients of rare diseases which are amenable to one-time treatment. In general, the policy suggests, ‘voluntary crowdfunding for treatment’ of rare diseases.
With this being the prevailing situation in India, even during Covid pandemic, an interesting article – ‘How Orphan Drugs Became a Highly Profitable Industry,’ published in The Scientist, noted some important facts in this area. It highlighted: ‘Government incentives, advances in technology, and an army of patient advocates have spun a successful market—but abuses of the system and exorbitant prices could cause a backlash.’
It also articulated, despite higher costs and less-certain returns, investments in drug development on the rare diseases side appear to ‘be bucking the trend.’ The result of the global focus on RD nowadays is: ‘Firms with marketing authorization for orphan products, are now more profitable than those without.’
This also partly explains the financial rationale behind AstraZeneca’s recent acquisition of Alexion Pharmaceuticals, valuing $39 billion.
As of December 20, 2020 morning, India recorded a staggering figure of 10,031,659 of new Coronavirus cases with 145,513 deaths. The country has already crossed 10 million in Covid cases as the vaccine approval remains pending. The threat of subsequent waves for further spread of Covid infection continues to loom large in many states. Meanwhile, many studies indicate that comorbidity should now include rare diseases, as well, especially to prevent deaths in younger patients. From this perspective effective diagnosis and treatment of RD are also coming under spotlights. Curiously, the National Policy on Rare Diseases 2020 focuses more on awareness and prevention of RD rather than access to affordable treatment, particularly in Covid infected patients to save precious younger lives. As I wrote previously and still believe, the ‘National Policy on Rare Diseases’ becomes more meaningful with ‘Orphan Drugs Act.’
Vaccines to prevent Covid infections are also expected to get emergency approval in India, shortly. At lease, some of these being available at affordable prices, including AstraZeneca-Oxford vaccine, according to reports. As recent reports indicate the same company, is also entering into RD therapy areas, through a key acquisition, yet another hope looms large. A hope for availability of relevant RD drugs at an affordable price for Covid infected patients, despite other apprehension, as I wrote before.
That apart, purely from the business management perspective, as well, this rare strategic acrobatic feat of AstraZeneca - ‘Keep nose to the grindstone while lifting eyes to the hills,’ during the Covid crisis, I reckon, is exemplary for the practicing managers.
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.