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FDA Orphan Drug Designation for Rare Diseases - What does this mean ?
"An estimated 30 million people in the United States are afflicted with rare (orphan) diseases or conditions; however, the availability of novel treatments for these diseases and conditions has been historically low compared to conditions with a higher prevalence. This is mostly due to the financial risks of recouping the costs of drug development. In 1983, Congress passed the Orphan Drug Act to incentivize development of orphan drugs to meet unmet needs. Since then, many companies have taken advantage of the orphan drug designation and its incentive programs for rare disease drug development.
Orphan Drug Act:
The Orphan Drug Act of 1983 was designed to promote the development of drugs, biologics, devices, or medical foods that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. By definition, a disease or condition is classified as “rare” if it affects fewer than 200,000 people total in the United States, or if the cost of developing a drug and making it available in the United States for such diseases or conditions will exceed any potential profits from its sale.
Since the inception of the Orphan Drug Act, more than 4,500 orphan designation requests have been granted, and over 730 drugs and biologic products have been developed and approved for more than 250 rare diseases. To put that into context, less than 10 orphan drug products were brought to market between a ten-year period from 1973 and 1983.
Incentives of Orphan Drug Designation:
One of the biggest challenges for companies developing drugs for rare diseases is that due to the small target population size, sponsors are unlikely to recoup the cost of research, development, and approval from the orphan drug product. In response to this, the FDA has created multiple incentives to make orphan drug development more financially possible for companies to pursue. Some of the incentives include:
7-year marketing exclusivity to sponsors of approved orphan products.
25% federal tax credit for expenses incurred in conducting clinical research within the United States.
(Tax credits may be applied to prior year or applied over as many as 20 years to future taxes)
Waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs.
(A value of approximately $2.9 million in 2021)
NOTE: This saved Revive Therapeutics almost $3 Million USD
Ability to qualify to compete for research grants from the Office of Orphan Products Development (OOPD) to support clinical studies for orphan drugs.
Eligibility to receive regulatory assistance and guidance from the FDA in the design of an overall drug development plan.
Todays Orphan Drug Designation in the Prevention of Ischemi Reperfusion Injury during Liver Transplantation, has already put the safety aspect firmly in the mindset of the FDA.
This will surely be of valuable advantage to Revive Therapeutics
Even if just based on the Drugs Safety aspects and surely will help open the door for Bucillamine being further approved in the treatment of Covid.