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    Study Finds FDA Action on Unapproved Drugs Led to Higher Prices, Longer Shortages

    A new study finds that the US Food and Drug Administration's (FDA) 2006 initiative to get manufacturers selling unapproved drugs to submit the drugs for approval or remove them from the market led to higher prices and longer shortages for those drugs. Background Today, drugs must be reviewed by FDA for safety and efficacy or in some cases conform to an over-the-counter (OTC) monograph before they can be marketed in the US. However, in the years between the 1938 Food, D...
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    Preparing for Market: Reimbursement Strategies for Cell and Gene Therapies

    • 08 September 2017
    In this interview with Regulatory Focus , Ted Slocomb and Michael Werner, two leaders from the Alliance for Regenerative Medicine (ARM), provide insight into future challenges and opportunities for ensuring market access and value-based reimbursement for gene and cell therapies. Cell and gene therapies represent a new, transformative era in medicine. For many indications, these products offer the promise of durable - even potentially curative – treatments and in many c...
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    FDA to Unveil New Regulatory Framework for Stem Cell Therapies

    The US Food and Drug Administration's (FDA) Commissioner Scott Gottlieb said Monday the agency will advance a new framework to better regulate stem cell therapies this fall. "This comprehensive policy will establish clearer lines around when these regenerative medicine products have sufficient complexity to fall under the agency's current authority, and then define an efficient process for how these products should be evaluated for safety and effectiveness," Gottli...
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    EMA Lays out Plans for Companion Diagnostic Guideline

    The European Medicines Agency (EMA) on Friday released a concept paper for public consultation outlining issues it plans to address in an upcoming guideline on the challenges in developing companion diagnostics for personalized medicines. Specifically, EMA says its guideline will address the development of companion diagnostics for precision medicines in the clinical development and post-approval phases, and will include a glossary defining terms used in the guideline as...
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    Conditional Approvals for Early Access to New Medications

    This article provides insight into Conditional Approvals (CAs) and Conditional Marketing Approvals (CMAs) as developed across several jurisdictions to potentially provide earlier access to new medicines. The author explains the specifics regarding regulatory requirements for obtaining CAs in the European Union (EU), Japan and the US. Introduction Before a medicinal product for human use is authorized to enter the market, it must undergo extensive study to ensure its s...
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    European Regulatory Roundup: Commission Begins Investigation Into Cancer Drug Prices (18 May 2017)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. European Commission Starts Formal Investigation into Aspen’s Cancer Drug Pricing The European Commission has opened a formal investigation into the way Aspen Pharma priced five cancer drugs. Officials began the probe after receiving reports indicating Aspen had imposed “very significant and unjustified price increases” on five off-patent cancer medicines it acquired ...
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    CBER Director Focuses on Flexibility to Advance Regenerative Medicines

    Peter Marks, director of the US Food and Drug Administration's (FDA) Center for Biologics Evaluation and Research (CBER) on Thursday said his office is equipped to support the development of cutting edge treatments, such as cell and gene therapies. Speaking at the Food and Drug Law Institute's annual conference, Marks said the 21st Century Cures Act added tools to FDA's regulatory arsenal, including the regenerative medicine advanced therapy (RMAT) designation to provi...
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    Humacyte Receives First of FDA’s Regenerative Medicine Designations

    The US Food and Drug Administration (FDA) on Monday offered one of its first Regenerative Medicine Advanced Therapy (RMAT) designations to Humacyte’s investigational human acellular vessel (HAV) Humacyl. “Being one of the first companies to receive the Regenerative Medicine Advanced Therapy Designation from the FDA is an honor and a testament to the significant potential for Humacyl to address a great unmet medical need for patients who are undergoing dialysis,” Ca...
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    CTTI Offers Recommendations for Pediatric Clinical Trials for Antibiotics

    The Clinical Trials Transformation Initiative (CTTI) has released a set of recommendations it says will improve the use of antibiotics in children. According to CTTI, a public-private partnership aimed at improving the quality and efficacy of clinical trials, challenges in enrolling and completing pediatric clinical trials for antibiotics leads to a situation where such drugs are used in children despite lacking adequate labeling for pediatric populations. Furthermore, ...
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    PMDA Head Talks Priorities for 'Rational Medicine'

    The head of Japan's Pharmaceuticals and Medical Devices Agency (PMDA), Tatsuya Kondo, on Thursday released a paper discussing his vision for a patient-centric, regulatory science-driven and evidence-based system for medicine in Japan. "'Rational Medicine' is the idea that a patient-centric system should be created—a system under which optimal medical care from the patient's point of view, which is based on the latest scientific knowledge, is provided—from the perinatal t...
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    International Medical Device Regulatory Update: News and Views From San Jose

    Several sessions at the 2016 RAPS Regulatory Convergence, held in San Jose, California in September, focused on medical device regulations and regulatory changes worldwide. These included: FDA's establishment of a national evaluation system for medical devices, China's agreement to give imported medical devices the same treatment as those manufactured or developed domestically, Japan's reduced review time for medical devices over six years, Asia/Pacific's expected medica...
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    Senate Overwhelmingly Passes 21st Century Cures Act Despite Patient Safety Concerns

    The Senate on Wednesday passed the 21st Century Cures Act by a vote of 94-5. President Barack Obama praised the bipartisan efforts and said he will sign the bill. The bill’s path to success, following the House’s positive vote last week, took a unique route, with the House also passing a previous iteration back in July 2015, and then the Senate trying to craft its own package of bills, which stalled and looked to be dead as recently as September. The wide-ranging, nea...