Hot Topics in Drug Development: The EMA perspective

Dr. Hans-Georg Eichler spoke to an audience of 55 senior physician executives from the Boston CMO Network on Sep 17, 2013. The event was hosted by Genyme Corporation in Cambridge, MA.

Dr. Eichler discussed four topics in his prepared remarks:

  • Flexible Pathways to Market Authorization
  • The Regulatory – Health Technology Assessment interface
  • Clinical trial data transparency
  • Global harmonization of evidence standards
  • Flexible Pathways to Market Authorization

    Dr. Eichler described the current model of approval which relies on a “magic moment”, where sufficient knowledge about a product has been gained to grant a license to market.  Unfortunately, in the current scenario, while the treatment population grows rapidly, that treatment experience does not contribute a great deal of evidence generation.  He asked the question: “Can we replace the current model with something else, such as Adaptive Licensing ?”

    He suggests the answer is yes, under certain conditions:

  • the unmet medical need is significant enough to make regulatory authorities willing to accept uncertainty
  • the post-licensing treatment exposure is via a measured roll-out with surveillance.
  • the growth of the product is managed to allow gradual access to larger populations.

  • He noted that the concept of Adaptive Licensing goes beyond Accelerated Approval, which is limited to life-threatening diseases.  He mentioned that it would be important for Adaptive Licensing to have a mechanism to capture real-world observational data.

    Regulatory – Health Technology Assessment Interface

    A challenge we all face is how to avoid conflicts between regulators and payers on the path to market access.  A concept that EMA has been exploring is to convene a meeting with sponsors, regulators and payers together during which both the regulators and payers discuss their evidence requirements.  The process is voluntary and private and 20 drugs have used it.  Payers and Regulators have also exchanged guidelines as part of these activities.

    Dr. Eichler believes that there is no question but that post-licensing research requirements will continue to grow.  Regulators will move toward adaptive licensing and payers will move toward allowing coverage, but with requirements for additional evidence development.

    Clinical Trial Data Transparency

    Dr. Eichler believes that clinical trial data transparency will, in the long run, provide benefits to the industry that will outweigh the risks.  Transparency should allow companies to learn from the mistakes of others.  Nonetheless, he acknowledged some of the risks, including the challenge of a level playing field for industry and the use of clinical trial data by academicians for meta-analyses.

    However, despite the risks, clinical trial data transparency is here to stay. This is not something that EMA can resist.

    Global Harmonization of Evidence Standards

    Much progress has been made via ICH on non-clinical and CMC issues, but this process won’t work for harmonizing clinical standards.  Currently, the agencies exchange guidelines and confer of a frequent basis, but some differences of opinion will continue to exist.  Dr. Eichler noted that sponsors can seek to get parallel scientific advice from FDA & EMA, but that this has been infrequent.  Most sponsors continue to approach FDA & EMA separately.

    Dr Eichler’s prepared remarks were followed by a lively Q&A session.

    About the Boston CMO Network

    The Boston CMO Network includes senior physician executives in the greater Boston area who are active in the biotech and pharmaceutical industry. The Network sponsors events for Boston-area physician executives to meet, interact and learn from each other.

    A Steering Committee of physician executives plans the group’s events, which are hosted at local biotech companies.

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