PREFER: giving patients a voice in drug development Research strategy

At MDUK, we want to make sure people with muscle-wasting conditions have more of a voice in the drug development process. We're working with pharmaceutical companies, academics, health technology assessment bodies and regulatory authorities, in a five-year international project called PREFER.


The difference PREFER will make

The overall aim of the PREFER project is to give people living with long-term conditions, including muscular dystrophies, more of a voice in drug development by creating a set of recommendations on how and when to include their views in the process. This will help to ensure the drugs will strike the optimum balance between benefits and risk, providing the improvements to health people want while trying to reduce the side-effects they would rather avoid. This will help people living with muscular dystrophies (and other health conditions) have the best possible quality of life. 


Drugs are developed for the benefit of patients, so it is essential we incorporate their views and preferences in the process. These views could be useful at all stages of drug development, from the early stages of drug discovery, right up to approval and beyond (see below)

outline of a drug life-cycle

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.

For example, pharmaceutical companies need to know what people value, so they can develop drugs that are suitable for their needs. Regulatory agencies also need to understand people’s preferences, so that they can make informed decisions on whether to make new drugs available. Similarly, these views are helpful to health technology assessment (HTA) bodies such as NICE, who decide whether drugs should be funded by healthcare systems like the NHS. Everyone agrees that it’s important to include the perspectives of people living with health conditions during drug development. However, there hasn’t been agreement on exactly how to gather these views at the different stages of the drug development process. This could lead to inconsistencies in how people’s views are included in the development of drugs, if at all.

The PREFER project

The PREFER project is looking to develop a ‘gold-standard’ process for including the views of people living with health conditions during drug development. PREFER is a consortium of universities, pharmaceutical companies, and patient organisations from across Europe. Watch this YouTube video to find out more.

MDUK is one of the patient organisations taking part, helping to make the views of people with muscle-wasting conditions are included in the project. People with muscle-wasting conditions (and other health conditions) have been involved in the PREFER project throughout, from helping to design the project and analyse the results, to sharing their views. MDUK has also helped recruit people with muscle-wasting conditions to share their views as part of a patient preference study (see ‘Progress to date’ below).

The overall aim of PREFER is to provide evidence-based recommendations on how and when patient preferences should be gathered and used to support decision-making. Organisations that will benefit from these recommendations include the pharmaceutical industry, regulatory authorities, HTA bodies, and academic research institutes such as universities. The PREFER project is due to release its final recommendations in 2022.

Progress to date

There are lots of different methods for collecting the views of patients (called ‘patient preference studies’), some of which will be more suitable than others in different situations. When the project started in 2016, the PREFER team spent a lot of time reviewing these different methods. Some of these methods were then taken forward to be assessed in patient preference studies in three health areas: rheumatoid arthritis, cancer, and muscle-wasting conditions.

Researchers from Newcastle University led the study that focussed on myotonic dystrophy type 1 and mitochondrial conditions which affect the muscles. MDUK helped to recruit people to take part.

The team was interested to know what adults with these conditions and their carers thought about different treatments (real treatments and hypothetical ones) and what trade-offs they were 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 treatments and clinical trials that are more focussed on the needs of people with these conditions.

Results from the patient preference study for muscle-wasting conditions were published in February 2021. A total of 52 people took part in the study, including people with myotonic dystrophy and mitochondrial diseases, as well as their carers. The study found that, of all the aspects of the conditions that the participants wanted to change, “muscle strength” and “energy and endurance” were most often mentioned. Patients said that they would prefer treatments which could provide improved balance, cognition, and gut function, while the most concerning risks were damage to the liver, kidneys, or eyes.

People’s attitudes to risks were also influenced by whether they had previously experienced a particular side-effect, and whether the side-effect would be temporary or permanent. The researchers also found that people living with the conditions sometimes had different preferences from those of their carers.

This research has provided new insights into the preferences of people with muscle-wasting conditions, which will assist the development of new treatments. It has also set an example of how best to carry out patient preference studies with people living with all kinds of long-term conditions.

Cecilia Jiminez-Moreno discussing the PREFER project

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.