RE:How does Accelerated Approval work ONCY's pelareorep has been granted fast track and orphan drug (rare disease) status in both pancreatic cancer and advanced/metatstatic breast cancer.
In the rare disease policy space, all roads invariably lead back to the Orphan Drug Act’s (ODA) enactment. In 1983, Congress passed the ODA to amend the Food, Drug, and Cosmetic Act — the U.S. law governing the approval of medicines — to include a definition of a rare disease or condition and to establish financial incentives to encourage the development of rare disease treatments, also known as orphan products.
The ODA defines a rare disease as any disease or condition that Orphan Drug Act “...affects less than 200,000 people in the United States, or affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug.”
In addition to defining a rare disease in statute, policymakers purposefully built financial incentives into the ODA to enable access to these medicines, recognizing the challenges and risks in developing treatments for small patient populations. In particular, products developed for rare disease indications are eligible to receive an Orphan Drug Designation from FDA. This designation provides the drug developer, or company that submits an application for FDA review, with several important incentives:
-- ORPHAN DRUG TAX CREDIT.
The sponsor company receives a 25% tax credit to offset research and development costs (qualified clinical testing). It is intended to incentivize biotechnology companies to invest in the development of treatments that are not otherwise economically viable.
-- USER FEE WAIVER. The application fee that is required for an FDA review (over $3.1 million,10 depending on the type of clinical data required, in 2022) is waived for sponsors.
-- MARKET EXCLUSIVITY. The orphan drug designation grants the sponsor company seven years of market exclusivity. With the passing of the Inflation Reduction Act ton 2022, the FDA had correspondingly granted "biological" drug candidates with 13 years of market exclusivity commencing from the time of drug approval. The FDA's accelerated approval process allows for the marketing authorization of an orphan drug based on surrogate endpoints (biomarkers) while the sponsor initiates a post-accelerated approval confirmatory trial for the subsequent acquisition of full marketing authorization. Here the FDA converts the acceleratred approval to a full, traditional approval of the drug.
-- RESEARCH GRANTS. Sponsors may be eligible for grants via FDA’s Office of Orphan Products Development (OOPD), which determines whether medical products meet the criteria for certain incentives and provides funding through grants for research on rare diseases. These grants are funded through Congressional appropriations and are used for clinical trials and natural history studies that advance rare disease medical product development and to develop nonprofit consortia to facilitate pediatric medical device development. In 2021, FDA awarded 11 different grants totaling $25 million to “meet the future and current health needs of those who suffer from a rare disease.”