What is the role of Congress on Drug Approval and Patient Access
Posted by: Christine McSherry on January 24, 2013
TITLE IX--DRUG APPROVAL AND PATIENT ACCESS
SEC. 901. ENHANCEMENT OF ACCELERATED PATIENT ACCESS TO NEW MEDICAL TREATMENTS.
(a) Findings; Sense of Congress-
(1) FINDINGS- Congress finds as follows:
(A) The Food and Drug Administration (referred to in this section as the ‘FDA’) serves a critical role in helping to assure that new medicines are safe and effective. Regulatory innovation is 1 element of the Nation’s strategy to address serious and life-threatening diseases or conditions by promoting investment in and development of innovative treatments for unmet medical needs.
(B) During the 2 decades following the establishment of the accelerated approval mechanism, advances in medical sciences, including genomics, molecular biology, and bioinformatics, have provided an unprecedented understanding of the underlying biological mechanism and pathogenesis of disease. A new generation of modern, targeted medicines is under development to treat serious and life-threatening diseases, some applying drug development strategies based on biomarkers or pharmacogenomics, predictive toxicology, clinical trial enrichment techniques, and novel clinical trial designs, such as adaptive clinical trials.
(C) As a result of these remarkable scientific and medical advances, the FDA should be encouraged to implement more broadly effective processes for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life-threatening diseases or conditions, including those for rare diseases or conditions, using a broad range of surrogate or clinical endpoints and modern scientific tools earlier in the drug development cycle when appropriate. This may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation without compromising or altering the high standards of the FDA for the approval of drugs.
(D) Patients benefit from expedited access to safe and effective innovative therapies to treat unmet medical needs for serious or life-threatening diseases or conditions.
(E) For these reasons, the statutory authority in effect on the day before the date of enactment of this Act governing expedited approval of drugs for serious or life-threatening diseases or conditions should be amended in order to enhance the authority of the FDA to consider appropriate scientific data, methods, and tools, and to expedite development and access to novel treatments for patients with a broad range of serious or life-threatening diseases or conditions.
Bookmark the permalink | Tagged dmd, duchenne, eteplirsen, exon skipping, muscular dystrophy, sarepta
pdufa fda patient-directed drug development patient perspectives duchenne muscular dystrophy harrison's fund duchenne allinace duchenne research alliance international invitation meeting duchenne muscular dystophy dmd treatment exon skipping sarepta eteplirsen dmd Permissible Risk Patient Perspective Duchenne Alliance PDUFA-V Fast Track Accelerated Approval Priority Review IND NDA Breakthrough therapy exon-skipping Duchenne muscular dystrophy collaboration rigorous review higher resolution. duchenne advocacy treatments pfizer children's hospital boston srpt $srpt jett foundation gower test biotech cambridge becker forbes healthcare rare disease new york times boston daily news prosensa moms morpholinos team community congress race to yes obama washington dc racetoyes washington white house petition fox business fox news stossel willis report government