FDA Law Blog: Rare Disease Week Recap: HP&M’s Frank Sasinowski Briefs the Rare Disease Congressional Caucus

FDA Law Blog: Rare Disease Week Recap: HP&M’s Frank Sasinowski Briefs the Rare Disease Congressional Caucus

Rare Disease Week Recap: HP&M’s Frank Sasinowski Briefs the Rare Disease Congressional Caucus

Posted: 05 Mar 2017 01:36 PM PST

By James E. Valentine –

Every year, hundreds of rare disease patients and caregivers descend on Capitol Hill to participate in Rare Disease Week to learn about federal legislative issues, meet other advocates, and share their unique stories with legislators. As part of this, on March 2, 2017, Hyman, Phelps & McNamara, P.C.’s Frank J. Sasinowski participated in the Rare Disease Congressional Caucus briefing where he spoke to Congressional staff and rare disease advocates about the implementation of the 21^st Century Cures Act. This speech covered the following issues that are important for the rare disease community:

• Patient experience data;
• Qualification of drug development tools;
• Priority review vouchers;
• Regenerative advanced therapies; and
• Targeted drugs for rare diseases.

Slides from Frank Sasinowski’s presentation are available here.

The caucus briefing was organized by the Rare Disease Legislative Advocates in coordination with the Rare Disease Congressional Caucus. The briefing, “Advancing Rare Disease Treatments in the Era of Cures and Health Care Reform,” covered other important topics that were discussed by other speakers:

• The PDUFA reauthorization process in 2017;
• The Affordable Care Act repeal and replacement;
• New models for rare disease drug development; and
• The role of incentives in the development of orphan therapies.

More information about the briefing is available here.

Frank Sasinowski had previously appeared before the House Energy and Commerce’s Subcommittee on Health to present testimony at the first hearing on the 21st Century Cures Initiative (see previous coverage here).