1. Patient Choice Act
….for those you love

2. Basic Elements
FDA provisional approval of fast track drug after establishment of Safety
Full approval continues to be sought
Distribution under informed consent
Enables provisional approval of drugs on the market in approved countries for > 4 yrs

3. Benefits
Provides patient rights to early access to investigational therapies under informed consent – giving patients the right to choose their therapies.
Can save thousands of patients’ lives every year who die waiting for FDA approved access to therapies, when all the standard approved therapies run out.
Preserves the existing regulatory system while making it financially viable for the companies.
Promotes innovation and ensures medical advances can reach the marketplace more quickly.
Stops the loss of companies investing in the development of therapies for deadly & rare diseases, and creates jobs, growth and drives American leadership in medical innovation.

4. Calls for Provisional Approval
Manhattan Institute
Milken Institute
Kauffman Foundation
PCAST
NEWDIGS
And many more!
“Such a reform would allow drugs that have been found safe and promising (in Phase I and Phase II clinical trials) to win approval for limited marketing to patients. This would give patients early access to innovative new therapies, while the FDA would retain the ability to collect information confirming the drugs’ safety and effectiveness and to revoke a drug’s marketing authorization later, when appropriate.” Manhattan Institute

5. Legislative Status Introduced in 113th congress on May 22nd
Many support letters – ALWAYS NEED MORE!!
Lead by Cong. Morgan Griffith (R-VA), Cong. Peters (D-VA), Cong. McCaul (R-TX)

6. Activities
Presented legislation at DIA on a panel with National Health Council, Vertex Pharmaceuticals, and Abigail Alliance
Working with Patient and Rare Disease Communities
CHI Dinner with Freshmen Congressmen from CA
Film Festival
Kauffman Foundation, Here.Us.Now
Global Genes and Rare Project, RARE
Fight to Live, Fight To Live