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Accounting for patient preferences: “Nothing for us, without us”

Taking patient preferences into consideration when developing a therapy would seem common sense. Yet it is only relatively recently that consultation has become formalised. IMI’s PREFER project is now helping to consolidate this process.

08 June 2022
A pile of pieces of yellow, green, blue and pink paper against a blue background. The top piece of paper is bright yellow and has a question mark on it. Image by SNeG17 via Shutterstock.
Image by SNeG17 via Shutterstock.

When developing a new treatment or health intervention, it’s easy to assume that – if successful – it will automatically improve the lives of patients. However, the reality can be more complex. Such improvements can be subjective; often, patients may be prepared to balance the potential benefits of treatment outcomes against, say, the impact of the side effects on their quality of life. It is only the individuals – often along with their family and carers – who really know what matters most to them.

Fortunately, regulators have recognised the importance of capturing these insights to help ensure treatments are truly patient centred. Increasingly, the European Medicines Agency (EMA) requires that manufacturers seek patient input at all stages of medical research and drug development.

However, capturing these insights in a way that makes them useful has proved a challenge in the past. Often, patients lack the vocabulary and experience required to articulate their experiences and preferences in a way that provides structured and quantifiable information that can be used in the development process.

Resolving this challenge has been the focus of the Innovative Medicines Initiative (IMI) project PREFER (Patient Preferences in Benefit–Risk Assessments during the Medical Product Lifecycle). PREFER sought to determine two things: how to undertake studies into patient preferences in order to make better treatments, and when and how to incorporate these insights into the drug development process.

Involving input from patient representatives at every stage of its work, the PREFER project has now determined an approach to these issues. It has developed a series of recommendations for industry, regulators and health technology assessment (HTA) bodies - the organisations that assess the effectiveness of medicines. The recommendations can help develop guidelines on when and how to include the patient perspective on the benefits and risks of their medicines. In addition, PREFER has also developed guidance covering all aspects of how to design and conduct studies into patient preferences, from determining the purpose to interpreting the results.

PREFER published its findings – including the initial qualitative stakeholder studies and the 10 prospective case studies – in more than 40 scientific peer-reviewed publications. Now, the EMA has given its approval to PREFER’s proposed framework for patient preference studies and to its ‘points to consider’ when selecting an appropriate method for selecting the best way to engage with patients. The Qualification Opinion adopted by the Committee for Medicinal Products for Human Use (CHMP) on 2 April 2022 stated that: “The proposed research framework and ‘points to consider’ document is generally endorsed as a comprehensive reference document for planning and conducting patient preference.”

So, given this approval, what comes next? How do the PREFER coordinators see the outputs being used, and what will be the benefits for patients? Conny Berlin and Mats Hansson, PREFER project leaders explained: “We are optimistic that the PREFER recommendations will help encourage the systematic and evidence-based inclusion of patient preferences in medical decision making in both industry and academia.”

PREFER is supported by the Innovative Medicines Initiative, a partnership between the European Union and the European pharmaceutical industry.