Report from the DIA Pharmacovigilance and Risk Management Strategies Conference, January 23-25, 2017, Washington, DC.

Written by Suzanne Berresford, Product Manager, Pharmacovigilance, Springer Nature

Report author, Suzanne Berresford

The nature of medicinal products is in a rapidly shifting world, with therapeutic innovation becoming a major global trend that is here to stay. The precision medicine approach and use of targeted therapies such as biologicals, gene therapy and stem cell therapy is weaving its way into standard practice but questions still remain about monitoring the long-term safety and efficacy of such advanced therapies. The DIA Pharmacovigilance and Risk Management Strategies Conference set out to discuss some of the challenges and to provide an opportunity for pharmacovigilance professionals to share their experiences in these areas.

In the Keynote Address, Hans-Georg Eichler (Senior Medical Officer at the European Medicines Agency) highlighted some of the differences between advanced therapies and more traditional medicines, such as earlier disease interception, more prolonged effects and individual treatment combinations. Furthermore, Dina Tresnan (Senior Director, Worldwide Safety and Regulatory at Pfizer) informed us in a separate session that many advanced therapies are administered as single doses yet their effects (beneficial or harmful) can persist for long periods of time. So this leads to the question of how long their effects should be monitored for.

Because of such prolonged effects, clinical trials for these types of therapies may not be of sufficient duration to fully realise the benefit-risk profile. Therefore, post-marketing experience is even more vital for advanced therapies. This may be in the typical approach of individual follow-up and observational studies, and importantly, the use of real world data will become increasingly significant. In fact, it was proposed that such data may be used even earlier in the drug development cycle, to the point where real world data may eventually supplement control-arm data in clinical trials. Indeed, the trial design itself could be very close to replicating real world clinical practice, as demonstrated by the Salford Lung Study.

Another important consideration of precision medicine discussed was the smaller target population size, mirrored by clinical trials often being performed in limited number of patients. Even in trials investigating a larger heterogeneous group, subgroup analysis of specific targets is likely to be underpowered. Again, this places a much higher importance on post-marketing data, and not just at a population level but at an individual level. For both gene therapies and biologicals, it is important to capture information on the batch administered to an individual patient. Dina Tresnan made reference to the EMA’s GVP on pharmacovigilance of biological medicinal products, as many of the recommendations in here are also applicable to other advanced therapies.

My take-home message from this conference is that pharmacovigilance is continually evolving, and the uptake of advanced therapies and precision medicine is a current trend likely to be a key influencer on the evolution of pharmacovigilance. Some of the changes we may see in the near future include:

  • The scope of pharmacovigilance expanding to include monitoring of long-term effectiveness as well as safety
  • Real world data being used to support pharmacovigilance activities much earlier in the drug development cycle
  • A shift from population-focused to individual-focused pharmacovigilance

So we, as pharmacovigilance professionals, need to prepare for this brave new world and accept the challenges as further opportunities to improve patient safety.


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