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Regulators tentatively endorse patient preference approach

PREFER’s proposals have been described by the EMA as a “comprehensive reference document for planning and conducting patient preference studies’’

Image by eggeegg via Shutterstock
Image by eggeegg via Shutterstock


A major regulatory milestone has been reached for researchers working on the IMI-funded project PREFER, who have been investigating and testing the best ways to include patients’ voice in medicine development and decision-making. A positive draft opinion from the European Medicines Agency (EMA) on the consortium’s proposed framework for patient preference studies, as well as a document outlining points to consider when selecting methods for structured patient input to medical product decision-making, will now undergo a public consultation before the agency gives its final opinion.

Data that's valuable for pharma, regulators and HTAs

Patients choose medical products based on their personal preferences. For example, people who have been living with a particular disease or condition for a long time might have different preferences about treatment options compared to people who have just been diagnosed.

This subjective information, collected in a structured way, is valuable for the companies that make drugs and other medical products, but it’s also vital data for the regulators that give green light to market a product, and the health technology assessment bodies (HTAs) who compare a medicine to existing alternatives. But the question of how we should go about carrying out studies on patient preferences, and how (and when) the results should be incorporated into the drug development and medical product lifecycle process is only being worked out now.

An important regulatory milestone

The IMI-funded PREFER project has been working on both of these questions, and they have defined solid methodologies having carried out 11 in-depth case studies since the project launch in 2016. They are currently finalising what is intended to be the principal output of the project: a set of recommendations that industry, regulatory authorities and HTA bodies will be able to use to develop guidelines on how and when to include patient perspectives on thebenefits and risks of medicines. 

In parallel to these recommendations, the consortium have worked out a research framework for patient preference studies that covers purpose, objectives, how to design and conduct the studies and how to interpret the results, as well as a document with points to consider when selecting the methods for carrying out a patient preference study.

The project submitted an application for a qualification opinion to the EMA-EUNETHA (the first joint regulator an health technology assessment bodies joint procedure), and the EMA has just released a draft opinion that endorses both the framework and the points to consider document, ‘’as a comprehensive reference document for planning and conducting patient preference studies’’.

The draft opinion is now undergoing a one-month consultation period so that researchers, patients, pharma companies, regulators, HTA bodies or anyone else can contribute their comments before EMA releases its final opinion.

Read more

Find out how to contribute comments on the EMA draft opinion 

PREFER sets out 15 ways to listen to the patient voice



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