By: Shaily Baag
In July 2017, the Central Government of India created the National Policy for Treatment of Rare Diseases (hereinafter ‘NPTRD’) on the orders of the Delhi High Court. The Court had given this direction after reviewing several writ petitions appealing for the state-sponsored treatment of rare diseases as the treatment costs of such diseases were exorbitantly high. The Court had directed the Ministry of Health and Family Welfare (hereinafter ‘MoHFW’) to come up with a national policy for the treatment of rare diseases.
The Court felt that the need for a national policy is based on at least four reasons. Firstly, Articles 21, 38 and 47 mention that the health care services provided by the state should be open, dependable and affordable. Secondly, there is no motivation in producing these low-volume medicines. Thirdly, even if these medicines are provided, they are highly-priced. Therefore, regulations are required to control prices. Fourthly, a policy will mean that all the people having rare diseases are to be treated equally by the state.
The policy, therefore, should come up with an approach that should have multiple distinct aspects, elements and sectors such that the country would have enough capacity to treat such diseases, develop the ability to gather and store epidemiological data, define the term ‘rare disease’ and estimate the cost of treatment of such rare diseases.
However, the MoHFW soon found out that efficient implementation was a challenge and that there were new evidence and updates accessible which could be used for further enhancement and a more comprehensive recommendation of the policy. The biggest challenge was to bring in the states as a part of the procedure. The question was how much a state should support the cost of such diseases which requires care at the third level – the tertiary level. There was no clarity on this issue.
The problem was that public health is a state subject and that the policy formulation in 2017 had taken place without any consultation of the states as critical stakeholders. The issues such as sharing of cost between the Centre and the States, priority of timely intervention in a cost-effective way by the state with limited resources versus other health priorities, and flexibility in adapting the policy, including bringing in changes per demanding situation, were not covered.
The MoHFW decided to review the policy, have a broad consultation and reframe the policy with the suggested amendments and required changes. Accordingly, it kept the policy in a state of temporary disuse and formed an expert committee in November 2018 to draft a new national policy for rare diseases, define ‘rare disease’, and suggest the vision and strategy for implementation.
Finally, on 13 January 2020, the review committee’s reframed draft policy was placed before the public for their views and comments as well as those of other stakeholders till 15 February 2020.
SALIENT FEATURES OF THE NEWLY DRAFTED POLICY
The draft policy cites recording 450 rare diseases. In the process, it firstly defined ‘rare disease’ by comparing it with the definitions of the WHO, the US, the EU, Japan, South Korea, Canada, Australia and Taiwan, and found that it correlated to the occurrence threshold instead of severity, inheritability or alternative treatment availability. Whereas some studies suggested this to be connected to rarity, location and research ability. The policy concluded that it is not possible to define ‘rare disease’ in India at this moment because of lack of data and advised to arrive at a definition at the earliest by starting a registry of the Indian Council of Medical Research.
The policy further includes genetic diseases, degenerative diseases, transmissible tropical diseases and rare cancers as rare diseases, and discusses the problems of diagnosing them as well as the lack of proper training and tools, R&D facilities, and medical and clinical trial history. The policy also discusses the challenges of treating these rare diseases such as the availability of an approved procedure to treat these diseases, high cost of treatment and pricing of drugs.
The policy mentions that, in India, there is no data on rare diseases mortality, morbidity and the expected economic burden which makes it difficult to form a plan. It compared and reviewed policies and schemes of countries like the US, the UK, Singapore, Australia and Malaysia, and the EU, which have such policies, funds and subsidy schemes. The policy cites a lack of economic resources and the burden to address the issue of the cost of treatment of rare diseases versus the benefit of society as a whole.
As no definition is available at the moment, the policy divided the rare diseases into three groups based on clinical data. The first group requires one-time remedial treatment and includes immunodeficiency disorders and osteopetrosis. The second one includes diseases the treatment of which requires low cost but is long and the benefits of which are well-recorded. The third group includes diseases the treatment of which requires high cost and is lengthy with less availability of records. Various categories and sub-categories are mentioned in each group.
The policy, as a strategy, directs integrated preventive measures to develop awareness to prevent such rare diseases from birth. The center will support the states in attempts at preventing such rare diseases as well as with the assistance of Rs. 15 lakhs for one-time curative treatments under the Rashtriya Arogya Scheme to the beneficiaries of the Pradhan Mantri Jan Arogya Yojana (hereinafter ‘PMJAY’).
The policy suggests the states to take care of the second group. The policy is silent on the third group but indicates that the Centre will come up with alternative fund mechanisms like voluntary crowdfunding through a digital platform to treat rare diseases at identified tertiary centers of excellence. The policy finally discusses a strategy to prevent and control rare diseases at antenatal and neonatal stages through screening and diagnosis by education and awareness at different levels such as primary, secondary and tertiary levels.
ARGUMENTS FOR AND AGAINST THE NEW POLICY
There may be many arguments for the newly drafted policy. Firstly, given that very few nations have a fully free scheme, it is a start for a developing country to provide Rs. 15 lakhs to 40% of the population for the one-time treatment of rare diseases. Secondly, hospitals have been identified for the tertiary treatment with the cost being borne through voluntary digital schemes. Thirdly, a strategy has been initiated for implementation for the prevention of rare diseases. Fourthly, the states have been told to bear the cost of treatment of the diseases in the second group. Fifthly, initiatives have been taken to define ‘rare disease’ and collect data for a future framework.
On the other hand, the policy cited lack of economic resources and the burden to provide healthcare to the poor. This may be true for the states as healthcare is a state subject but is not valid for the Centre. Additionally, fixing Rs. 15 lakh as the limit for the poor may not help all patients. It covers only 40% of the people who are under the PMJAY which is discriminatory. Finally, citing a lack of availability of data to define ‘rare disease’ and being silent about the third group of rare diseases renders the policy incomplete.
While the policy provides some relief to a part of the population which falls under the first group of rare diseases, it leaves the responsibility of treating the second group of rare diseases to the states. The policy is also silent on the treatment of the third group of rare diseases. However, the purpose for which the Delhi High Court directed the MoHFW to frame a policy is still not satisfied since many more issues such as the definition of ‘rare disease’, filling the implementation gap, given that the states have not been consulted yet again, and devising a definitive method for treating the third group of rare diseases are yet to be resolved.
(Shaily is currently a law undergraduate at National Law University, Cuttack. She may be contacted at https://www.linkedin.com/in/shaily-baag-462989181https://www.linkedin.com/in/shaily-baag-462989181.)
Cite as: Shaily Baag, ‘National Policy for Treatment of Rare Diseases: A Critical Analysis of Recent Developments’ (The RMLNLU Law Review Blog, 02 April 2020) <https://rmlnlulawreview.com/2020/04/02/national-policy-for-treatment-of-rare-diseases-a-critical-analysis-of-recent-developments> date of access.