2Basic ElementsFDA provisional approval of fast track drug after establishment of SafetyFull approval continues to be soughtDistribution under informed consentEnables provisional approval of drugs on the market in approved countries for > 4 yrs
3BenefitsProvides 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.
4Calls for Provisional Approval Manhattan InstituteMilken InstituteKauffman FoundationPCASTNEWDIGSAnd 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
5Legislative 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)
6ActivitiesPresented legislation at DIA on a panel with National Health Council, Vertex Pharmaceuticals, and Abigail AllianceWorking with Patient and Rare Disease CommunitiesCHI Dinner with Freshmen Congressmen from CAFilm FestivalKauffman Foundation, Here.Us.NowGlobal Genes and Rare Project, RAREFight to Live, Fight To Live