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PREFER: giving patients a voice in drug development

5 February 2019

In our commitment to giving patients a voice in drug development, Muscular Dystrophy UK is working with pharmaceutical companies, academics, patients, healthy technology assessment bodies and regulatory authorities in a five-year international project called PREFER.

Drugs are developed for patients, so it is essential that their views and preferences be incorporated into the drug life cycle (see below). For example, pharmaceutical companies need to know what patients value, in order to develop drugs that are suitable for patients’ needs. Regulatory agencies also need to understand patients’ preferences, so that they can make informed decisions on whether to make new drugs available. Similarly, patient preference information is helpful to health technology assessment (HTA) bodies, who decide whether drugs should be funded by healthcare systems like the NHS.

A drug goes through several stages in its ‘life cycle’. The PREFER project looks at how and when it is best to perform and include patient preferences in decision making during this life cycle.

A study collecting this sort of information is called a ‘patient preference study’. At the moment, there is little guidance on the use of these studies during the drug life cycle. The PREFER project will address this by producing evidence-based recommendations. These will advise on how patients’ preferences should be collected and how this information should be used by decision-makers.

Since the project started in 2016, the PREFER team have spent a lot of time studying the different ways of collecting patient preferences. These methods have been reviewed and some are now going to be assessed in patient preference studies in three clinical areas: rheumatoid arthritis, cancer and neuromuscular conditions.

Muscular Dystrophy UK is helping to lead the neuromuscular patient preference study together with Newcastle University. The study will focus on myotonic dystrophy type 1 and mitochondrial disease. We are interested to know what adults with these conditions and their caregivers think about different treatments (real treatments and hypothetical ones) and what trade-offs they are willing to make. A trade-off is the act of balancing a negative against a positive, for example the side-effects or risk of a treatment against the benefits. This sort of information will help pharmaceutical companies to design more patient-centric treatments and clinical trials.

The study will consist of two parts:

  1. interviews and focus groups (March – April 2019)
  2. online survey (September – December 2019).

If you’re interested in being interviewed or taking part in a focus group, please get in touch with Dr Cecilia Jimenez-Moreno (

You can also find out more about PREFER by visiting

Disclaimer: This news item and its contents reflects the PREFER project’s view and not the view of IMI, the European Union or EFPIA.

Acknowledgement: The Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 115966. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.

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