In September, You Testified Before Congress On The Inflation Reduction Act's (IRA) Real-World Impact On Rare Disease R&D And Small Molecule Therapies. Can You Summarize The Key Points?

THE IRA MAKES MAJOR DRUG DISCOVERIES LESS LIKELY, especially from smaller emerging biotech companies, which are the innovation engine for the biopharmaceutical sector broadly. It applies government price controls to a range of drugs, which is guaranteed to reduce investment in finding new treatments and cures. There are two specific areas of great concern that I highlighted for the congressional committee: the IRA’s negative effects on research for rare disease, and the disincentive to invest in small molecule drugs.

In rare diseases, policymakers have done a U-turn from the successes of the Orphan Drug Act and are pursuing policies that punish, rather than reward, companies trying to find novel treatments for rare diseases. The law’s authors exempted orphan drugs from price controls if they treat a single rare disease. But medicines that treat multiple rare diseases don’t qualify for the exemption. That’s a big problem. Drug makers routinely investigate whether a drug already approved to treat one rare disease could possibly treat another. The IRA is already forcing some drug companies to freeze efforts to find additional applications for existing rare disease drugs.

In disincentivizing small molecule drugs, which account for about 90% of approved medicines, the IRA slashes the value of any new small molecule drug. In my company’s quest to treat Fabry disease, it took 13 years of continuous clinical trials – and half a billion dollars – before the FDA approved our small molecule treatment in 2018. Investors supported our research because they knew if we were successful, they could earn a return. They wouldn’t have backed us for so long if they thought price controls could take effect less than a decade after the drug’s release.

My own children survived because of scientific research and an entrepreneurial ecosystem that enables breakthrough discoveries. Today, the IRA has made this kind of success story impossible.

JOHN CROWLEY is executive chairman, Amicus Therapeutics.