The Rare Disease Company Coalition

How do we choose the appropriate controls for #raredisease trials? At a workshop with the Duke-Margolis Institute for Health Policy and the U.S. Food and Drug Administration, our Executive Director Stacey Frisk highlighted the importance of using a “totality of evidence” approach — considering all available data to find the best model for patient safety and efficacy. Watch to see her remarks!

Price controls come with a cost. A new analysis from Charles River Associates compares recent novel approvals across the U.S. and other high-income countries and finds that while orphan drugs were more likely than nonorphan drugs to gain regulatory approval abroad, they were less likely to be reimbursed or launched and faced longer delays to patient access. International price controls - like a Most Favored Nation model - could jeopardize U.S. patient access to rare disease treatments, and threaten U.S. leadership in rare disease innovation. Thanks to landmark policies like the Orphan Drug Act, the U.S. leads the way in rare disease innovation and access. We must protect that leadership so that patients can continue to receive access to the treatments they need. Read the full analysis: https://lnkd.in/ginye7ZA

Thank you to everyone who joined us at our briefing, “America Leads: Sustaining U.S. Innovation in Rare Disease.” Leaders across the rare disease ecosystem discussed the U.S.’s role at the forefront of rare disease treatment, exploring the policies that will shape the future of that leadership. Watch the full briefing: https://lnkd.in/gADm4q6B

Passionate about rare disease policy and advocacy? The RDCC is hiring a Manager/Senior Manager of Government Affairs to help advance legislative and regulatory solutions that improve the lives of millions of Americans living with a rare disease. Join us and represent 33 of the most innovative life sciences companies—working across federal and state policy to drive change and deliver real impact for rare diseases patients. Interested candidates should submit a resume and optional cover letter to careers@rarecoalition.com. More details are below - we look forward to hearing from you! https://lnkd.in/ewxYV9Wv #RareDisease #GovernmentAffairs #HiringNow #Policy #Advocacy #RDCC

We're #hiring a new Manager/Senior Manager, Government Affairs in Washington, District of Columbia. Apply today or share this post with your network.

The RDCC brought #OneRareVoice to Capitol Hill! Leaders from the RDCC spent the day meeting with our nation’s policymakers. We highlighted vital legislation like the Give Kids a Chance Act, which would reauthorize the Priority Review Voucher program and deliver life-changing therapies for the rare disease community. We remain committed to advancing policies that drive progress for patients, families, and innovators. Together, we’re working to spur innovation and expand treatment options for those who need them most.

The House Energy & Commerce Committee has unanimously passed the #GiveKidsAChanceAct! We are grateful to Representatives Gus Bilirakis, Debbie Dingell, and Michael McCaul for sponsoring this crucial legislation, which would bolster young #raredisease patients in their fight for life. Sick children don’t have any time to lose — we urge Congress to bring the Give Kids a Chance Act to the House floor for a vote.

Happening today! Tune in for our fall webinar briefing, “America Leads: Sustaining U.S. Innovation in Rare Disease.” Join to hear from experts from across the rare disease community. It's not too late to register: https://lnkd.in/gf4fsrM7

Reminder that the RDCC’s Rare Reception is tonight! We're excited for an evening of conversation, community, and connection with rare disease stakeholders. See you soon! RSVP here: https://lnkd.in/g6YpkEmB 

Join us on Wednesday, September 17 for our fall webinar briefing, “America Leads: Sustaining U.S. Innovation in Rare Disease.” Panelists across the rare disease ecosystem will discuss how the U.S. became a leader in rare disease treatment and what policies will shape the future of that leadership. Register here: https://lnkd.in/gf4fsrM7