The thorny challenge of developing ultra-rare disease drugs; pharma lobbyists face their biggest test in a decade

Taking stock of the drug industry, from the lab to the medicine chest ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ 

STAT+: 'No magic bullet': For drugmakers and the FDA, clinical trials on ultra-rare diseases pose thorny challenges By Ed Silverman Meggan Haller for STAT The FDA's decision not to review an ultra-rare disease drug underscores the challenges in gathering and reviewing limited data. Read More STAT+: 'What can we do?': Pharma lobbyists face their biggest test in a decade, as Democrats barrel toward a vote on drug pricing By Rachel Cohrs Alex Hogan/STAT Pharma’s lobbyists are staring down their biggest test yet, as Democrats barrel toward a vote on drug pricing reform. Read More STAT+: Despite their anger over high drug prices, Americans are giving pharma credit for helping contain Covid-19 By Nicholas Florko Matt Rourke/AP Nearly three quarters of Americans give the pharmaceutical industry credit for helping contain the Covid-19 pandemic. Read More Sponsor content by STAT The next generation of STAT Wunderkinds The search continues! STAT is on the lookout for the most impressive young minds in science, to give them the recognition they deserve. Nominate someone by July 29. STAT+: FDA's decision to review a Biogen ALS drug is met with skepticism and uncertainty on Wall Street By Ed Silverman AP/Biogen The FDA's decision to review Biogen's experimental ALS drug is being met with skepticism and uncertainty on Wall Street. Read More Opinion: Value-based payment has produced little value. It needs a time-out By Kip Sullivan and Ana Malinow and Kay Tillow Adobe Billions of dollars have been spent on value-based payment programs that yielded little improvement in health or lower health care costs. Read More Tuesday, July 26, 2022       1 Exchange Pl, Suite 201, Boston, MA 02109 ©2022, All Rights Reserved. I no longer wish to receive STAT emails Update Email Preferences | Contact Us | View In Browser