In a significant stride towards healthcare accessibility, medicines for four rare diseases have become markedly more affordable due to the initiation of domestic production by Indian pharmaceutical companies.

The Union Health Ministry confirmed this transformation on Friday, highlighting a substantial shift away from costly imported formulations.

The Health Ministry's concerted efforts, prioritizing action on 13 rare diseases alongside sickle cell anemia, have resulted in the approval and indigenous production of medications for Tyrosinemia Type 1, Gaucher's Disease, Wilson's Disease, and Dravet-Lennox Gastaut Syndrome, PTI reported.

Officials disclosed that four additional drugs for three diseases—Tablet Sapropterin for Phenylketonuria, tab Sodium Phenyl Butyrate, tablet Carglumic Acid for Hyperammonemia, and Capsule Miglustat for Gaucher's disease—are currently undergoing approval processes. These medications are anticipated to be available by April 2024.

This monumental shift towards local production is poised to significantly reduce the annual cost of these medications. For instance, the cost of Nitisinone capsules, previously priced at Rs 2.2 crore annually, will now be accessible for merely Rs 2.5 lakh, a staggering hundredfold reduction from its imported counterpart.

Similarly, the domestically manufactured Eliglustat capsules, formerly valued at Rs 1.8-3.6 crore per annum, will be obtainable for only Rs 3-6 lakh.

The cost of the imported Trientine capsules, prescribed for Wilson's disease, will plummet from Rs 2.2 crore per annum to a mere Rs 2.2 lakh due to indigenous production.

In a noteworthy revelation, the cost of Cannabidiol used in treating Dravet-Lennox Gastaut Syndrome is expected to diminish from Rs 7-34 lakh per annum to a range of Rs 1-5 lakh due to domestic manufacturing.

The commercial supply of Hydroxyurea Syrup, pivotal in treating sickle cell anemia, is anticipated to commence by March 2024 at a tentative price of Rs 405 per bottle, significantly more accessible than the previous cost of 840 USD (Rs 70,000) per 100 ml from abroad.

This paradigm shift in drug availability and affordability follows intensive discussions and collaborations initiated in July 2022.

Engagements with academia, pharmaceutical industries, regulatory bodies, and governmental departments played a pivotal role in prioritizing rare diseases, culminating in approval and cost-reduction strategies.

A rare disease, affecting a small percentage of the population, collectively impacts 6-8 percent of any country's populace. In India alone, it is estimated that there could be 8.4-10 crore cases of such diseases.

These reforms aim to alleviate the burden of exorbitant medical expenses, enhancing accessibility for individuals affected by these rare yet significant health conditions.