President Obama Signs Into Law a Bill to Expedite FDA Approval of Drugs for Rare Diseases.

Post date: May 17, 2013 2:53:48 PM

July 11, 2012.

President Obama Signs Into Law a Bill to Expedite FDA Approval of Drugs for Rare Diseases.

President Obama has signed into law legislation that will advance therapy development for rare diseases like Spinal Muscular Atrophy (SMA). The Food and Drug Administration Safety and Innovation Act provides new tools to the Secretary of Health and Human Services (HHS) and the Director of the Food and Drug Administration (FDA) to help expedite the approval process for drugs that treat rare and life-threatening conditions.  Families of SMA is supporting two therapies that currently are in clinical trials and could benefit from this new authority.

Under the provisions of the FDASIA, if a new drug demonstrates the potential to address unmet medical needs for a serious or life-threatening disease or condition, the Secretary of HHS can deem it a “fast track product” and employ more broadly effective processes for the accelerated development and review of the innovative new medicines.  Additionally, if a drug shows early clinical evidence that it could be significantly more effective than existing therapies, the Secretary can declare it a “breakthrough therapy” and expedite the review and development of those drugs. 

The new law also establishes a process whereby the Secretary of HHS can consult with external experts on rare diseases, targeted therapies, and genetic targeting of treatments concerning the severity of and the unmet medical need associated with rare diseases.  The Secretary also may consult with these external experts on the willingness and ability of individuals with a rare disease to participate in clinical trials, an assessment of the benefits and risks of therapies to treat rare diseases, the general design of clinical trials for rare disease populations and subpopulations, and the demographics and the clinical description of patient populations.  This ability of experts in SMA to work formally in a collaborative fashion with HHS and FDA officials is tremendously beneficial and will facilitate the drug approval process.

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Click here for Bill details S.2281 -- ExPERT Act (Introduced in Senate - IS~)