TOPRA: 'Patients help benefit—risk decisions'

Tuesday 6th December 2011

Patient contributions to benefit-risk decisions and the transparency of the process were discussed at the recent joint meeting of The Organisation for Professionals in Regulatory Affairs (TOPRA) and the European Medicines Agency (EMA).

The annual meeting took place in the EMA building in Canary Wharf, London, from November 24 – 25.

Fiona Reekie (Janssen Research& Development) and Xavier Luria Oller (EMA) chaired a session looking at how critical decisions are made on new medicines and the role of patients.

Francesco Pignatti (EMA) summarised work done so far in the EMA project with centralised products in five European regulatory agencies aimed at improving transparency and consistency.  

Decision-making models will be applied to medicines where the benefit-risk is more difficult to determine, while effects tables will become more common, it is believed.

Patients have been contributing to the regulator's benefit–risk decision-making by sharing experiences and advising on the risks that they are prepared to accept; tending to accept more risk than regulators think they will.

Filip Mussen (Janssen Pharmaceutical Companies of Johnson & Johnson) illustrated how some companies are using structured processes, qualitative and quantitative, to facilitate and enhance benefit–risk decision-making.  

Methods used range from NNT/NNH (numbers needed to treat/numbers needed to harm) to, eg, BRAT (Benefit-Risk Action Team) framework forest plots, prior to submission of an MAA (Market Authorisation Application). 

Different methods are resource intensive but help transparency. 

Patients can contribute to a company's benefit–risk decision-making through the use of patient reported outcomes in clinical trials, patient preference methods and through anecdotal evidence.

Isabelle Moulon (EMA) shared results from two surveys on the communication of benefit-risk in the context of an environment of increasing transparency.  

Consumers and health care professionals' information expectations in this area are similar, with a need to understand benefit-risk both at the population and individual level.

Access to high quality information on medicines is key to this. The template for EPARs (electronic Personal & Academic Records) has been revised to address benefits, risks and uncertainties and an SmPC (Summary of Product Characteristics) Advisory Board has been setup.

The EudraSmPC webpage will be made public in 2012. The template for package leaflets has been revised to introduce information on the benefit of the medicine. 

Patients are regularly involved in the review of package leaflets, EPAR summaries and of communication on safety issues. 

The new pharmacovigilance legislation will result in more transparency with, eg, the publication of all conclusions of assessments and decisions.  

The EMA communication strategy and transparency policies are under preparation and will build on the work done to date.

TOPRA

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