A recent article published in India’s Pharma Literati cites The World Health Organization as not having endorsed a definition for rare diseases. The European Union defines ‘rare’ as less than fifty people per one hundred thousand. The United States considers ‘rare’ as less than two hundred thousand affected individuals.
In fact, most movement disorders may be considered rare.
Currently, over seven thousand disorders have been identified of which eighty percent are genetic. Rare diseases represent six to eight percent of the total population or about three hundred million people.
Non genetic rare diseases may result from autoimmune diseases, environmental factors, degenerative diseases and infections.
It is noteworthy that the drug market for rare diseases may reach $242 billion by the year 2024. The compound annual growth rate is 12.3 percent.
India’s Ministry of Health
The Government of India cites its recording of four hundred fifty rare diseases. Often called ‘orphan disease’, some of the more prevalent diseases are sickle-cell anemia, hemophilia, thalassemia and primary immunodeficiency.
Dr. Sudheendra Rao, Scientific Advisor, points out that many rare diseases are diagnosed at various stages in life and may be fatal. Others are chronic and can drastically lower quality of life. There are many subtypes and genotypes (genetic makeup of an organism).
Almost four hundred fifty rare diseases have been identified and recorded in India to date. India’s total represents about one fifth of the number of rare diseases worldwide.
This number may be much higher due to cases that are misdiagnosed. A spokesman for the Organization for Rare Diseases India cites not having an appropriate national rare disease registry for the lack of adequate data.
Rare Disease Status in the United States
Since 1983, the U.S. FDA has granted an orphan drug designation to 5,198 drugs. Specifically, during the last ten years, the number of orphan drug designations has increased six-fold.
New drugs developed in the U.S. were intended to treat 363 health conditions with many applying to rare genetic disorders. About eighty-one percent, or 4,208 filings for orphan drugs, originated in the United States.
About Research in India
By contrast, between 2003 and 2019, India submitted ten drugs for orphan drug designation applicable to thirteen health conditions. During this period, India was in twenty-first place by country for drug development.
However, turning the numbers around, Dr. Rao reminds fellow scientists that India has not always had the advantage of a powerful economy. But during the past sixty years the country has managed to capture thirty percent of the United State’s generic market.
Dr. L. Prashanth at Vikram Hospital emphasizes that the numbers depicting India’s drug development together with the size of its population are clear evidence that India should address the need for more research.
But Why the Edge?
There are many reasons why the United States has an edge over drug developers in other countries. One of the main reasons may be attributed to an insurance industry that readily approves and pays for the treatments.
Despite common perception, the U.S. regulatory process is well designed. Research and development centers are well funded. These factors are definite motivators for investors as it has become clear that the rare disease market can be extremely lucrative. Countries in Asia and Europe are aware of its future potential and are focusing on the increasing need.
Dr. Rao points to purchasing power and inadequate health insurance as two factors impeding India’s research. In addressing these issues, research institutes and pharma companies are stepping up to overcome India’s rare disease burden.
NOVEL AGENTS
India has approximately seventy million cases of rare diseases indicating a serious shortage of research and drug development in this area.
However, researchers are becoming aware that there is an upside to developing therapies for rare diseases. Most of the rare diseases have easily identifiable characteristics (e.g. genetic mutations) as opposed to the more common diseases.
Most of the drugs developed for rare diseases will be first on the market. This gives the drugs significant advantage over emerging competition.
That opens the door for developers to create formulations and novel targets leading to intellectual property opportunities. Researchers have recently relied on the newer, advanced treatments. Among these therapies are gene and cell therapy, gene editing and RNA therapy.
And one other consideration is anticipated return on investment (ROI). The ROI for phase III filed Orphan Drugs is 1.89 times greater than it is for non-orphan drugs.
Yet effective research in Orphan Drugs has still not taken hold in India with only a handful of companies researching Orphan Drugs.
One reason for the lack of enthusiasm by pharma companies is that unlike the U.S. FDA, the government of India does not issue incentives.
Dr. Rao suggests tax credits and incentives such as:
- Tax credits for research and development costs
- Access to national research
- Facilities to aid industrial research and development
- Grants for phase I and II clinical trials
- Market exclusivity
- Subsidies on market value of orphan drugs
These incentives and subsidies will substantially increase orphan drug development.
There is currently a growing focus on the biosimilar market, with an estimated value of $240 billion and a number of patents approaching their expiration dates.
Patient Assistance
The availability of drugs presents an immense problem in India. The healthcare system is primarily self-funded. When drugs are available the need for financial assistance is self-evident. Help might come from the pharma companies’ charitable programs, non-government organizations, or patient foundations.
In 2019 the Modi government announced its intention to provide a one-time payment to assist rare disease patients living below the poverty level. The assistance, for specified procedures, would be open to approximately seventy million people living in India.
Dr. Rao commented that this type of financial assistance is especially beneficial for surgeries.
Current and Future Challenges
Doctors agree that the cost of treatment for rare diseases and the facilities available for treatment create an atmosphere that is prohibitively expensive. Therefore, the government and pharma companies need to step up their efforts in this regard.
In the meantime, it is important for people living in India, if possible, to practice healthy lifestyles and self-examination.
And lastly, the insurance company must cover a greater number of rare diseases and ease the financial burden on patients.
