Rare diseases, often referred to as orphan diseases, affect a small percentage of the population, yet their impact on individuals and families is profound. As of October 2021, the ICMR’s rare diseases registry lists a total of 4,001 rare diseases in India.
Recognising the unique challenges faced by patients with rare diseases, the government introduced the National Policy for Rare Disease, 2021 (NPRD’21) with focusing heavily on the prevention. It has failed to provide timely treatment by facilitating access to necessary medicines.
The NRDP’21 classifies rare diseases into three categories: disorders amenable to one-time curative treatment; diseases requiring long term/lifelong treatment having relatively lower cost of treatment and diseases for which definitive treatment is available at a high cost and lifelong therapy.
While the Centre is implicitly taking the responsibility of providing treatment for the group 1 and 3 there is no public provisioning of treatment to all rare diseases falling in these three groups. NRDP’21 offers ₹50 lakh as a one-time payment and also established a crowd funding platform for further assistance.
Financial support
While a sum of ₹50 lakh offers substantial support for patients battling Group 1 diseases, it falls significantly short when addressing the complex and costly treatments required for Group 3 diseases.
The crowdfunding platform initiative established under NRDP’21 has raised a mere ₹3,08,775 to date, despite over 1,897 patients registered for funding.
Many rare diseases often encompass severe and chronic conditions that demand advanced medical interventions, expensive imported drugs and prolonged treatment durations, all of which escalate the financial burden on patients and their families.
In such cases, the financial support provided by the government becomes inadequate.
The Centre and Ministry of Health launched India-made drugs for 14 rare diseases, significantly reducing their cost. However, these drugs address only four conditions: Tyrosinemia Type 1, Gaucher’s Disease, Wilson’s Disease, and seizures related to Dravet/Lennox Gastaut Syndrome. Reports indicate a few are awaiting approval. However, these list covers only off patented medicines.
A significant challenge lies in the government’s struggle to address the monopolistic control pharmaceutical companies hold over patents for rare disease medications.
In numerous cases, these companies have prioritised patenting and monopolising their drugs over making them accessible to the Indian population.
Positive step
The government’s recent announcement of global procurement for 120 patented medicines, including nearly 17 for rare diseases, marks a positive stride towards addressing public health needs.
However, this initiative is insufficient to resolve the access issues faced by patients with rare diseases. The high cost of patented medications, coupled with limited availability continues to hinder equitable access.
To address the problem of high prices of patented drugs, Para XI of the implementation of strategy of NRDP’21, states that: “Department of Pharmaceuticals, Department for Promotion of Industry and Internal Trade (DPIIT) will be requested to promote local development and manufacture of drugs for rare diseases by public and private sector pharmaceutical companies at affordable prices and take legal/legislative measures for creating conducive environment for indigenous manufacturing of drugs for rare diseases at affordable prices..”
Terms “legal/legislative measures” clearly conveys the use of public health safeguards and the legal safeguard available to the government. One such safeguard under the Patents Act is the government use licenses under Section 100. The immediate enactment of Para XI, coupled with public provisioning of medicines and treatments, is essential to ensure justice for rare disease patients and their families.
Rao is a Legal and Policy Advisor with Third World Network; Tekwani is a member of Cystic Fibrosis Trust India