• ExplainersExplainers
    Regulatory NewsRegulatory News

    Regulatory Explainer: Everything You Need to Know About FDA’s Priority Review Vouchers

    Since 2007, the US Food and Drug Administration (FDA) has issued a handful of special "priority review" vouchers (PRVs) which allow its recipient to expedite the review of any one of its new drug products. What are these vouchers, why is FDA issuing them and what benefit might they have for society? Find out in our latest Regulatory Explainer on the Priority Review Voucher system. Last updated on 25 February 2020 to include: Vifor Pharma said on 17 February that it p...
  • Regulatory NewsRegulatory News

    FDA Drafts PRV Guidance for Medical Countermeasures

    The US Food and Drug Administration (FDA) on Wednesday released new draft guidance to help companies understand how the agency will award priority review vouchers (PRVs) for qualifying medical products to diagnose, prevent or treat diseases or conditions associated with chemical, biological, radiological and nuclear (CBRN) threats and emerging infectious diseases. The draft, featuring 29 questions and answers on the new PRV program, is part of FDA’s implementation ...
  • Regulatory NewsRegulatory News

    Have PRVs Incentivized New Rare or Neglected Disease Research? Experts Discuss

    The priority review voucher (PRV) programs, created by Congress with an eye to incentivizing the development of new rare pediatric and neglected tropical disease drugs, have so far rewarded a wide range of small and large biopharma companies and products, though whether PRVs have actually spurred new research remains a question. Andrew Robertson, head of regulatory science and policy at Sanofi, and Rachel Cohen, regional executive director for the Drugs for Neglect...
  • Regulatory NewsRegulatory News

    FDA Approves First Treatment for a Form of Batten Disease

    The US Food and Drug Administration (FDA) on Thursday approved the first-ever treatment for a form of the rare pediatric Batten disease, bringing Biomarin its second lucrative priority review voucher (PRV). FDA approved Brineura (cerliponase alfa) to slow the loss of walking ability in symptomatic pediatric patients three years old and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency. ...
  • Regulatory NewsRegulatory News

    Sarepta Sells Priority Review Voucher for $125M to Gilead

    Sarpeta Therapeutics on Tuesday sold its priority review voucher (PRV) to Gilead for $125 million, speeding up the US Food and Drug Administration (FDA) approval process for any future drug or biologic of Gilead’s choosing from 10 months to six months. The voucher was awarded to Sarepta under the rare pediatric PRV program after winning FDA approval for its Duchenne Muscular Dystrophy (DMD) drug Exondys 51 (eteplirsen) in September 2016. This is the third PRV Gilead ...
  • Regulatory NewsRegulatory News

    Priority Review Voucher Fees to Decline in FY 2017

    The US Food and Drug Administration (FDA) on Thursday unveiled the new user fee rates for the tropical disease and rare pediatric disease priority review voucher (PRV) programs. The additional fees necessary to use the vouchers for both programs are set to decline by about $20,000 when compared to last year. Tropical Disease Priority Review Voucher User Fee Year Voucher Fee FY 2011 $4,582,000 FY 2012 $5,280,000 FY 2013 $3...
  • Regulatory NewsRegulatory News

    Congress Signs Off on Short-Term Extension to Rare Pediatric PRV Program

    The House late Tuesday followed its Senate counterparts and sent to President Barack Obama a re-authorization of the rare pediatric disease priority review voucher (PRV) program that will last through the end of December. A spokeswoman for Sen. Bob Casey (D-PA), a sponsor of the bill, told Focus that between now and 30 December, Congress will discuss ways to amend the program and extend it again. “Our policy changes are in S. 1878, which is headed to the president, so...
  • Regulatory NewsRegulatory News

    House E&C Committee Clears New Priority Review Voucher Program

    The House Energy & Commerce Committee late Wednesday pushed through a bill that will create a new priority review voucher (PRV) program for medical countermeasures that will not sunset. The new PRVs for medical countermeasures, if passed by the full House and Senate, would be the third PRV program in existence (in addition to the rare pediatric disease and neglected tropical disease PRV programs ) and the first to not sunset. Like the other two PRV programs, HR 3299...
  • Regulatory NewsRegulatory News

    House Looks to Reform Tropical Disease Priority Review Voucher Program

    The House Energy and Commerce Committee on Wednesday passed by voice vote an amendment (to an unrelated bill on hospital preparedness for dangerous threats) that would significantly change the priority review voucher (PRV) program for tropical diseases. Background Since 2009, the US Food and Drug Administration (FDA) has issued nine PRVs to reward companies for developing new drugs to treat rare pediatric and tropical diseases with a voucher that can be sold on the op...
  • Regulatory NewsRegulatory News

    Coalition Calls on FDA to Follow EMA’s Lead and Offer Scientific Opinions on Use of Drugs Outside US

    As part of efforts to streamline and strengthen regulatory pathways for global health products, the Global Health Technologies Coalition (GHTC) says in a new report issued Thursday that Congress should direct the US Food and Drug Administration (FDA) to establish a specific mechanism to offer a formal scientific opinion on medical products for their use outside the US. Because FDA is classified as a stringent regulatory authority, the agency’s review of drugs and devices...
  • Regulatory NewsRegulatory News

    Congressional Support for Priority Review Vouchers Presses On Despite FDA Qualms

    Almost a decade into the experiment of offering lucrative priority review vouchers (PRVs) to help speed the development of treatments for neglected tropical and pediatric diseases and it seems Congress’s love of the vouchers has only just begun. And although that love is sure to be a good sign for companies looking to make hefty profits off of the vouchers on the open market (selling for upwards of $350 million), it’s a bad sign for the US Food and Drug Administration (...
  • Regulatory NewsRegulatory News

    Senate HELP Advances Bill to Reauthorize Pediatric PRV Program and Six Other Bills

    The Senate Health, Education, Labor & Pensions committee on Wednesday advanced seven bills, including one that would reauthorize the rare pediatric priority review voucher (PRV) program through 2022, and one bill that would add the Zika virus to the tropical disease PRV program. The advancement of the bill sponsored by Sen. Bob Casey (D-PA) to extend the pediatric PRV program comes as the US Food and Drug Administration (FDA) told the Government Accountability Office ...