The ORPHAN Cures Act is bipartisan legislation that would maintain existing incentives and boost research into new treatments for the 30 million Americans currently suffering from one of more than 7,000 rare diseases. The bill would achieve this by amending the Inflation Reduction Act’s harmful orphan drug exclusion and incentivizing critical follow-on investment into rare disease drug development.

The Inflation Reduction Act (IRA) of 2022 requires biopharmaceutical companies to pay rebates to the government if the price for drugs in Medicare Part B or Part D increase faster than the rate of inflation.

Maximum Fair Price: A manufacturer of a drug selected for negotiation will be required to
offer a “maximum fair price” for the selected drug with respect to Medicare beneficiaries.

Timeline: The first set of drugs will be selected for negotiation in September 2023, with the
negotiation process starting a month later. The first set of negotiated prices taking effect in
“initial price applicability year” 2026.

OVERVIEW MEDICARE PART D BENEFIT RESTRUCTURE

Currently, millions of Medicare beneficiaries reach the catastrophic phase in January or February, which exacerbates the financial hardship of the high prices of OOP costs. The Inflation Reduction Act (IRA) will allow Medicare beneficiaries to spread their out-of-pocket cost over the course of the plan year.

The goal of expedited approval pathways is to provide safe and effective therapies for serious, unmet medical needs as quickly as possible.

Learn how patient advocacy groups can get involved in the federal rulemaking process. 

Resources for emerging growth biotech companies would be better used for science than compliance with Section 404(b), economists find.

Learn about the Hatch-Waxman Act and its role in balancing innovation and drug competition.

PDUFA provides greater consistency, certainty and predictability in the FDA's drug review programs.

Learn about how the Bayh-Dole Act benefits the U.S. economy, consumers & patients.