US Drug Negotiations Plan Shifts Focus for Rare Disease Programs

The rare disease community is pushing for changes in the Inflation Reduction Act’s drug price-setting scheme as it leads more drugmakers and investors to reconsider development of drugs to treat small patient populations.

Pharmaceutical companies such as Pfizer Inc., Alnylam Pharmaceuticals Inc., Eli Lilly & Co., and Protagonist Therapeutics said they’re halting or reshaping research into rare disease or small-molecule drugs out of concern they may not recoup their costs under the Medicare Drug Price Negotiation Program.

The program, President Joe Biden‘s signature health initiative to lower prescription drug costs by negotiating prices with manufacturers, exempts ...