A rudderless approach
People believe that certain diseases are fatal due to their nature, but sometimes more so due to the treatment cost. Rare diseases are among them. For 95 per cent of the 7,000 to 8,000 rare diseases, the approved treatment is unavailable and for the remaining, the treatment costs are exorbitant.
The economic status of the family and treatment options become two important factors in case of a rare disease. Hence, the National Policy for Rare Diseases, 2021 announced on March 30, 2021, comes at the right time.
The first policy was formulated in 2017 but was kept in abeyance due to limiting factors like ‘bringing states on board’. This was necessary as public health is primarily a state subject. A new policy was framed after an expert committee reviewed the earlier policy to remove gaps and the new draft was published in January 2020 to invite views and suggestions. It was announced after the Delhi High Court ordered to announce the policy before March 31, 2021.
Having such a policy is a good beginning considering that there are an estimated 7 crore rare diseases patients in India. However, experts in the field have found some lacunae in the policy. The first and foremost, pointed out by the Organisation for Rare Diseases India (ORDI) is that the policy lacks a definition of rare diseases.
Though rare diseases are defined on three parameters – the number of people affected by the disease, prevalence and, fewer treatment options – the main criteria for definition is the small number of people affected by the disease. ‘How small a number’ is the question. The US has defined it as a disease affecting less than 2,00,000 people. The World Health Organisation (WHO) defines it as a disease having a frequency of less than 6.5-10 per 10,000 people. We should have defined it in the context of our population. India maintains that it lacks epidemiological data on the prevalence here and hence has only classified certain diseases as ‘rare.’
The policy aims to lower the treatment cost and increase focus on indigenous research and local production of drugs, creating a national hospital-based registry of rare diseases for ensuring adequate data. It also provides for early detection, suggests a crowdfunding mechanism, and classifies rare diseases into three categories. The categories are disorders amenable to onetime treatment, those requiring long-term or lifelong treatment, and diseases for which definitive treatment is available but the challenge is to make an optimal patient selection for benefit.
The policy announces financial support up to Rs 20 lakh for one-time treatment to all those who are eligible as per the 23 norms of Pradhan Mantri Jan Arogya Yojana (PMJAY). This support is for the diseases classified in the first category. For those in the second category, the policy suggests that the state governments should consider giving the aid. But the caregivers, advocacy groups, and ORDI are not satisfied with the amount pointing out that it does not reflect the actual cost.
The annual cost for a child’s treatment for a rare disease is estimated to be between Rs 10 lakh and Rs 1 crore per year and it increases with the age and weight of the child. Experts estimate that the total cost of treating the patients will be Rs 80 to 100 crore per year. So the Union Government must partner with states for sharing the cost. That will put a burden of only Rs 40 to 50 crore on the Union Government. The drugs for rare diseases have to be imported as there are no domestic pharma producers for these drugs. In this context, the Delhi High Court’s directive to set up a National Consortium for Research, Development, and Therapeutics (NCRDT) is very important for domestic drug development.
When a policy is formulated for the first time, it is, no doubt, a good beginning. But if the main stakeholders like ORDI find any gaps then the government should consider their say as final in the interest of the patients.