Rare Diseases Crisis in India: The Urgent Need for Locally Developed Drugs

Rare diseases, or what is popularly known as orphan diseases usually affect a small portion of the total population, but it has a significant impact on those who are affected and their families.

As per the Indian Council of Medical Research’s (ICMR) rare diseases registry, there are a total of 4,001 rare diseases which were identified in India as of October 2021. The government has also launched the National Policy for Rare Disease, 2021 (NRPD’21) to understand the difficulties faced by people with rare diseases. The policy focuses on prevention; however, the success rates have not been great as the policy was unable to provide timely treatment or access to the necessary medicines.

What Are Rare Diseases?

NRPD’21 has categorised rare diseases into three groups:

Disorders that can be cured with a one-time treatment

Diseases that require long-term or lifelong treatment but at a relatively lower cost

Diseases that need high-cost, lifelong therapy

While the government is to take responsibility for treating diseases under groups 1 and 3, the policy does not cover all the rare diseases within the categories. NPRD’21 only provides a one-time payment of Rs. 50 lakh and has set up a crowdfunding platform for additional support.

Financial Assistance

Though the initial amount of Rs. 50 lakh offered to the families and affected persons under Group 1 is helpful, but this is far from the actual need and does not help the treatments needed for the Group 3 diseases. Under the crowdfunding initiative, only Rs. 3,08,775 have been raised so far, for the 1,897 patients who were registered for the funding.

The Centre and Ministry of Health have launched India-made drugs for at least 14 rare diseases. This has significantly reduced the cost of the medicines; however, they only treat four conditions. These are Tyrosinemia Type 1, Gaucher’s Disease, Wilson’s Disease, and seizures linked to Dravet/Lennox Gastaut Syndrome. As per the reports, some of the registered patients are still awaiting approval for the medicines.

A major challenge is the government’s difficulty in addressing the control that pharmaceutical companies have over patients for rare disease medications. These companies often focus on patenting and monopolising their drugs rather than making them accessible to people.

Call For Local Medicines

The government has also recently announced a plan to globally procure 120 patented medicines for 17 rare diseases. While this is a positive step for addressing public health needs, it is not enough to solve the inaccessibility that the patients face. The high cost and the limited availability of these patent drugs are the reasons why the treatment is not fair for everyone.

NPRD’21 has suggested promoting local development and manufacturing of drugs for rare diseases by public and private pharmaceutical companies at affordable prices. This has also led to a call for locally produced medicine to cut down the cost of importing.

This will solve the issue of high-priced patent drugs. The NRPD policy also calls for legal measures that can create a conducive environment for producing drugs in India.

For instance, under Section 100 of the Patents Act, government use licenses are promoted to safeguard public health. The section states: Government, or any person authorized by it, is empowered to use the patented invention 'for purposes of Government.

With these measures, public health can be made accessible, and fair treatment can be ensured for the patients and their families.

Indian Organisation for Rare Diseases (IORD) since its inception in 2005 has been working towards bridging the gap between treatment and its accessibility for the patients and their families. IORD also highlights the gap that exists due to a lack of trained professionals and diagnostic facilities. With the government's initiatives and IORD's efforts to remove the social stigma around rare diseases, patients and families affected by rare diseases can avail the required treatment.