National Policy on Treatment of Rare Diseases: Issues and Policy Dilemmas

Rare Disease (Representation Only)

A rare disease is a health condition of a particularly low prevalence that affects a small number of people compared with other prevalent diseases in the general population. Rare diseases include genetic diseases, infectious tropic diseases and degenerative diseases. There is no universally accepted definition of rare diseases.

World Health Organisation defines rare disease as often debilitating lifelong disease or disorder condition with a prevalence of 1 or less, per 1000 population. Three elements to the definition as used in various countries are as follows:
1. The total number of people having the disease
2. Its prevalence
3. Non-availability of treatment for the disorder

The most common rare diseases include Haemophilia, Thalassemia, Sickle-cell Anaemia etc

Issues with Rare disease

1. Challenges in research and development: Rare diseases are difficult to research upon

2. Varying definitions: The use of inconsistent definitions and diverse terminology can result in confusion

3. Lack of access to correct diagnosis: Doctors are typically unaware about them and therefore do not diagnose them. Delay in diagnosis increases the suffering of the patients.

4. Lack of quality Information: The lack of quality data on incidence and prevalence of rare diseases impedes understanding.

5. High cost of treatment: Given that the market is small, the field of rare diseases has not been considered profitable for drug developers and manufacturers globally.

6. Lack of scientific knowledge: The field of rare diseases is complex and suffers from a deficit of medical and scientific knowledge.

7. Unavailability of treatment: About 95% rare diseases have no approved treatment and less than 1 in 10 patients receives disease specific treatment.

National policy for rare disease:

Delhi High court had directed the Ministry of Health & Family Welfare to frame a “national policy on treatment of rare diseases. Delhi High Court has categorically stated that low disease incidence cannot be the state’s basis for denying someone the right to life enshrined under Article 21 of the Constitution.

Key features:
1. It recommends that families below the poverty line are given free and supportive services in private or government hospitals.
2. Creating a patient registry
3. It provides corpus fund of 100 Crores for part funding treatment of rare diseases.
4. Developing materials for generating awareness in the general public
5. Constituting a Rare Diseases Cell within MoHFW, ICMR and DoP in the Ministry of Chemicals and Fertilizers to be the nodal for the activities related to rare diseases.

Policy Dilemma with rare disease

1. There is no clarification as to whether the financial help for treatment would only be provided for BPL patients or would also include APL patients.

2. Definition of rare diseases: The new policy does not define this either, leaving it unresolved.

3. No Corpus fund: Though the corpus fund of 100 Crores was provided in the affidavit, no such corpus was created under National Health Mission.

Way Ahead

The government should create a new and inclusive rare disease policy as soon as possible. New policy should allocate substantial resources to research for the development of new platform. There is an immediate need to create awareness.

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