Giving patients with Muscular Dystrophy a voice in drug development

2019-02-05

PREFER partner Muscular Dystrophy UK is commited to giving patients a voice in drug development. In PREFER, they work with pharmaceutical companies, academics, patients, healthy technology assessment bodies and regulatory authorities to ensure the rare disease patient perspective is present in the recommendations coming out of the project.

In PREFER, 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).

The study is currently recruiting patients in the UK.

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This text is part of a longer text originally published by Muscular Dystrophy UK at www.musculardystrophyuk.org. Want to know more about PREFER? Sign up to our newsletter!

About MDUK

Muscular Dystrophy UK is the charity for the 70,000 people living with muscle-wasting conditions in the UK. We bring together people affected by more than 60 rare and very rare progressive muscle-weakening and wasting conditions.

Muscle-wasting conditions are very rare, affecting just over one in every 1,000 people. As a result, some health professionals may never come across anyone with a muscle-wasting condition and may not know much about them. But we do.

Since 1959, we’ve been supporting families living with muscle-wasting conditions. We provide vital information, advice, resources and support for people with these conditions, their families and the professionals who work with them.

Muscular Dystrophy UK