Grumpy-XL schreef op 18 december 2022 18:11:
www.thepharmaletter.com/article/orpha...Challenges facing the orphan drug Industry
While there is much to celebrate in orphan drug development, there are still some concerns among stakeholders in the sector. The most debated issues are the perceived lax standards for clinical evidence used in obtaining regulatory approval and the sustainability of orphan drug prices.
Since rare diseases affect a tiny subset of the population, there is often limited understanding of the natural histories of the diseases with little regulatory precedent for companies to rely on. It is also challenging enrolling enough participants to collect statistically significant data on the efficacy of the therapies being trialed. These factors are often implicated in concerns about the quality of trial evidence presented by companies, which the FDA relies on for drug approvals.One of the ways the FDA has tried to improve these concerns is via patient-focused drug development meetings, where the FDA facilitates the collection of patient experience data from patients, family members, caregivers, advocacy groups and foundations to help the drug developers determine relevant endpoints for clinical trials.
There have also been persistent concerns about the high prices of orphan drugs once they hit the market, especially with the increasing number of drug approvals. These therapies are launched at high prices which sometimes does not reflect the clinical benefit.In addition, the prices do not decrease when additional indications are added. The Orphan Drug Act of 1983 allows drug developers to obtain a 7-year exclusivity on orphan drugs. However, a loophole in the Act allows manufacturers to maintain exclusivity for diseases that affect 200,000 or more people by ‘piggybacking’ on an older orphan drug designation, even if they can easily recoup R&D costs on the newer version.If passed by the Senate, the Fairness in Orphan Drug Exclusivity Act is expected to close the loophole by requiring manufacturers of such drugs to show that there would be no expectation of recovering their R&D costs through US sales.