Urgent Measures Required for Affordability of Rare Disease Medications

In recent developments, patient advocacy groups and organizations focused on rare diseases have urged the national authorities to exercise their rights to make expensive patented medicines for rare conditions more accessible and affordable within the Indian market. These groups emphasize the necessity for swift governmental, judicial, and legislative actions to combat patent monopoly abuses in the realm of rare diseases. They further appeal to the government to stimulate the generic production of patented rare disease drugs, allowing India to solidify its role as the developing world’s pharmaceutical provider, aiding both Indian patients and those in other economically challenged nations.

This call is accentuated by the legal case involving Swiss pharmaceutical giant Roche, seeking a perpetual injunction against India’s Natco Pharma from producing generic versions of the spinal muscular atrophy drug, Risdiplam, due to patent infringement claims. Risdiplam’s patent is valid until May 2035, and its staggering price of Rs. 6 lakh per bottle results in an annual expense of roughly Rs. 70 lakh for affected patients. Advocates argue that allowing generic production could lower the drug’s cost to approximately Rs. 3,000 per bottle. Referencing expert cost evaluations, they note the production cost is Rs. 2,418 per bottle, with a proposed generic price around Rs. 3,024. Similar challenges are faced by patients with other rare conditions, such as cystic fibrosis, due to patent restrictions.

The issue of accessible, reasonably priced medications for rare diseases is pressing and persistent in the country. With over 7,000 rare conditions documented worldwide, around 450 exist within India. Global rare disease patients amount to 300 million, with 70 million in India. Most endure limited or nonexistent treatment options, relying heavily on government financial aid due to the exorbitant costs of rare disease therapies. While the Union Health Ministry introduced the National Rare Disease Policy (NRDP) in 2021 to aid rare disease patients, drug prices—ranging from Rs. 10 lakhs to Rs. 1 crore annually—restrict the government from offering perpetual support. Although 450 rare diseases are documented in India, only 63 are recognized under NRDP, granting up to Rs. 50 lakhs per patient for treatment. Accessibility to affordable drugs is vital for countless patients battling rare diseases. Presently, few companies produce rare disease medications, as the limited patient base does not present a lucrative market for drug developers. These firms justify high prices as necessary to recover their research and development investments.

Given these circumstances, governmental intervention is critical for the survival of patients suffering from rare diseases. It is imperative that authorities do not falter in exploring every avenue to ensure rare disease medications are within the financial reach of those impacted.