Transcript Patient Choice Act - Rare Disease Legislative Advocates

Patient Choice Act
….for those you love
Basic Elements
• A drug for deadly diseases that qualifies for
fast track, can seek a provisional approval with
the establishment of Safety
• During provisional term, patients gain access
with informed consent.
• Full approval continues to be sought, during a
provisional term of 5 years (that can be
extended by regulators).
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.
Calls for Provisional Approval
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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
Legislative Action
• Many support letters – ALWAYS NEED
MORE!!
• Set to be introduced in 113th congress
• Lead by Cong. Morgan Griffith (R-VA)