Whilst the Inflation Relief Act (IRA) objectives to decrease the costs of prescription drugs, on the identical time it exempts medication treating uncommon sicknesses (i.e., orphan medication) from drug worth negotiation. On the other hand, this exemption is best legitimate if the orphan medication has a unmarried authorized indication. Thus, a key query is how steadily orphan medication are later evolved for different indications. If this is the case, IRA may just may just de-incentivize R&D investments in new indications for orphan medication.
A brand new paper by means of Chambers et al. (2023) in JAMA Community Open FDA approval database between 2003 and 2022 to look how commonplace new indications are for orphan medication. They to find that:
FDA authorized 282 novel orphan medication from 2003 to 2022…General, the FDA authorized 152 separate follow-on indications; 92 (61%) of those follow-on indications have been additionally for orphan drug prerequisites. The imply (SD) time from novel orphan drug approval to follow-on indication was once 53 (43) months…FDA integrated 58 (38%) follow-on indications in a single expedited assessment program, 46 (30%) in 2 methods, and 17 (11%) in 3 methods; none have been integrated in all 4 methods.
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