AN OPENING TO RAISE IRA WORRIES — Hundreds of rare disease advocates will descend on Washington next week to lobby Congress and the executive branch to speed potentially life-changing therapies to market — and some may seek tweaks to President Joe Biden’s biggest legislative win to better shield those drugs from Medicare price negotiations. Patient advocates — and the pharma companies behind potential treatments — are pushing for lawmakers to amend the Inflation Reduction Act to soften its impact on orphan drugs — the medicines geared toward rare conditions that are harder for drug companies to profit from because of their small patient populations. Under the landmark law, products designated by the FDA as a rare disease drug and approved for the treatment of one specific disease or condition are exempted from Medicare price negotiations. But the agency can designate the drug for a different rare disease — a move that typically occurs early in the regulatory process and might not lead to additional approvals for other specific conditions. If that happens, the drug is no longer exempt, which advocates worry could discourage biotechs from studying potential treatments for uncommon illnesses. “Having a designation does not enable you to market the product,” said Heidi Ross, vice president of policy and regulatory affairs at the nonprofit National Organization for Rare Disorders. But getting Democrats to sign onto any effort to amend the law, touted as a major Biden administration achievement, will be a tough sell, former congressional aides said in December at a rare disease forum. “It has to be bipartisan; it has to be incremental,” Corey Malmgren, a former Democratic health counsel who now lobbies for drug companies, said at the event. “We have to socialize it.” Other asks: Some groups are focusing on other priorities, such as pushing Congress to reauthorize the FDA’s rare pediatric disease priority review voucher program, which expires Sept. 30. The initiative lets companies that win approval for such drugs redeem vouchers for expedited agency review of a subsequent product application. The week culminates with an all-day conference on March 1 at the FDA. Congress weighs in: The House Energy and Commerce Health Subcommittee has scheduled a hearing for the day before on 18 rare disease-focused bills, including a bipartisan bill to broaden the orphan drug exclusion from Medicare negotiations. IT’S FRIDAY. WELCOME BACK TO PRESCRIPTION PULSE. Your host is back from the great Midwestern city of Indianapolis. Send news and tips to Lauren Gardner (lgardner@politico.com or @Gardner_LM) or David Lim (dlim@politico.com or @davidalim).
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