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    FDA recognizes Memorial Sloan Kettering tumor variant database

    The first somatic human variant database has been recognized by the US Food and Drug Administration for inclusion in its Public Human Genetic Variant Database listing. The move opens the door for developers of tumor profiling tests to use the database to support clinical validity claims in regulatory submissions.   A portion of Memorial Sloan Kettering Cancer Center’s Oncology Knowledge Base (OnkoKB) received the recognition under FDA’s database recognition program. ...
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    Regulation of cell and gene therapy products in Japan

    In recent years, new cell and gene therapies have been developed and approved in a number of countries, including Japan. The Japanese government fully supports academia and the pharmaceutical industry in the research and development of these innovative therapies, and in 2019, revised the Act on Securing Quality, Efficacy, and Safety of Products Including Pharmaceuticals and Medical Devices to enact the conditional early approval system for accelerating the development and ...
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    FDA guidance says sponsors should provide 'convincing' proof of concept data to support INDs for individualized medicine

    Recent draft guidance from the US Food and Drug Administration addresses the non-clinical safety data to support investigational new drug applications (INDs) for individualized antisense oligonucleotide (ASO) products, a type of personalized medicine.   The guidance, issued on 26 April, addresses INDs for ASOs that treat a severely debilitating or life-threatening disease caused by a unique genetic variant. Such diseases may be amenable to RNA-directed treatment.   ...
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    Time's up for questionable cell and tissue products, says Marks

    Purveyors of unapproved cellular-derived treatments are about to find out what happens when the US Food and Drug Administration ends its period of enforcement discretion for these regenerative medicine products.   A three-year clock for enforcement discretion began when FDA announced its regenerative medicine policy framework in November 2017; the COVID-19 pandemic prompted a 6-month extension, announced the agency last summer. Time is now up, and no more extensions ...
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    Update on RMAT designations

    This article discusses the scope and purpose of the special designation for regenerative medicine advanced therapies (RMAT) created by the passage of the 21st Century Cures Act. The authors explain the benefits expected to be realized with RMAT and provide a tally of products receiving the special designation to date and a current count, by year, of products for which RMAT designation has been requested.   Introduction Section 3033 of the 21st Century Cures Act, titl...
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    Bespoke therapies – opportunities, challenges, and hope

    This article discusses the advent of bespoke therapies, defined as the tailoring of medical treatment to the individual characteristics or symptoms and responses of a patient during all stages of care and as a new frontier beyond personalized medicine. The author covers the revolutionary genetic tools implementing such therapies and the clinical and nonclinical safety perspectives for bespoke therapies. The author concludes that with bespoke therapies we are entering a new...
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    EMA Begins Publishing Reports on Whether Approved Medicines Still Qualify as Orphans

    The European Medicines Agency (EMA) on Wednesday announced it has begun publishing reports detailing its decision-making on whether newly approved drugs to treat rare diseases still qualify for orphan designation when they receive marketing authorization. In the EU, drugs intended to treat diseases affecting fewer than five in 10,000 people in the EU qualify for orphan designation, which provides authorized medicines with ten years marketing exclusivity and reduced fees....
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    Asia Regulatory Roundup: Australia Bans Transvaginal Mesh Products (5 December 2017)

    Welcome to our Asia Regulatory Roundup, our weekly overview of the top regulatory news in Asia. Australia Bans Transvaginal Mesh Products Following Risk-Benefit Review Australia has banned transvaginal mesh products used in the treatment of pelvic organ prolapse. The Therapeutic Goods Administration (TGA) took the action after concluding the risks of the controversial treatment outweigh the benefits. Patients and healthcare experts around the world have questi...
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    FDA Unveils New Regenerative Medicine Framework

    The US Food and Drug Administration (FDA) on Thursday launched a new policy framework for regenerative medicine, building off a previous framework from 2005, as part of efforts to bring new cell, stem cell and tissue products to patients as efficiently as possible while managing the proliferation of unscrupulous actors hawking unproven therapies. FDA’s announcement included the release of two new draft guidance documents – one on ways to expedite approvals for regen...
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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...