RARE Act Retaining Access and Restoring Exclusivity Act
Impact
If passed, the RARE Act would adjust the regulatory landscape for orphan drugs significantly by ensuring that the exclusivity rights granted to manufacturers do not inhibit the introduction of new treatments that pertain to specific approved uses within already existing drugs. This change aims to foster a competitive environment in the pharmaceutical market where more treatment options are available to patients suffering from rare conditions. Additionally, it would discourage monopolization in the market for these drugs, theoretically leading to improved patient access and potentially lower costs due to increased market competition.
Summary
House Bill 7383, known as the Retaining Access and Restoring Exclusivity Act (RARE Act), proposes amendments to the Federal Food, Drug, and Cosmetic Act aimed at limiting the exclusivity periods currently granted for drugs intended to treat rare diseases or conditions. The key change involves redefining the terms around 'exclusive approval' to focus specifically on 'approved use or indication' related to the already approved drugs within the same rare disease category. This amendment seeks to streamline the approval process and increase accessibility for patients needing these specialized medications.
Contention
Despite its aims, the bill has been met with scrutiny and points of contention concerning the potential implications for drug research and development. Critics argue that reducing exclusivity could disincentivize pharmaceutical companies from investing in the development of new orphan drugs because they may not see a sufficient return on investment. Proponents of the bill counter that the current system creates unnecessary barriers to access and fails to incentivize the availability of multiple treatment options for patients in need, thus presenting a complex debate between patient access and pharmaceutical innovation.
Fairness in Orphan Drug Exclusivity Act This bill limits which orphan drugs may be granted a market exclusivity period by the Food and Drug Administration (FDA). (Generally, an orphan drug is one that is not economically viable because of the rarity of the disease that it treats; the sponsor of an FDA-designated orphan drug may be granted various incentives, such as a seven-year period in which the FDA may not grant market approval to a different sponsor for the same drug to treat the same disease.) Under this bill, if a drug is designated as an orphan drug on the basis that there is no reasonable expectation that the sponsor will recover the costs of developing and distributing the drug from U.S. sales, the drug shall be granted the seven-year exclusivity period only if the sponsor demonstrates that there is no reasonable expectation that it will recover such costs within its first 12 years of U.S. sales of the drug. When deciding whether an orphan drug meets this requirement, the FDA shall consider the sales of all drugs from the sponsor that are covered by the same orphan drug designation.