In November 2016, while hearing a related case, the Delhi High Court reportedlydirected 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......
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