The FDA is launching a new Rare Disease Innovation Hub to help them to better understand and more effectively advance therapeutics, medical devices and diagnostic tests for very small patient populations (https://lnkd.in/eEZxW-Qt). This is exciting news for the #raredisease patients waiting for treatments and the #biotechs looking to deliver them. MassBio Board Chair Tamar Thompson, a longtime rare disease champion and Head, Corporate Affairs at Alexion Pharmaceuticals, Inc./AstraZeneca Rare Disease: “We applaud the FDA for this significant step forward in creating a more equitable environment for rare disease patients and their families. This achievement is also a testament to the tireless efforts of patient advocacy organizations, clinical-stage and small biotech companies collaborating with the FDA to advance innovation. At MassBio, we recognize the critical role these companies have played in driving the progress that the Rare Disease Innovation Hub represents. “There is much more work to be done, and we look forward to serving as a champion for the biotech community, a partner for rare disease patients, and a resource to the FDA.” #patientdriven #FDA #rarediseases #rarediseasecommunity
This is an incredible initiative by the FDA! It's heartening to see the concerted efforts to advance innovation for rare disease patients. Your leadership and commitment to this cause are truly inspiring.
Director, Takeda Ventures, Resesarch Strategy & External Innovation
2moFantastic news that will hopefully bring down the barriers for developing life-saving therapies for rare disease patients!