Repurposing existing drugs to treat rare diseases could improve outcomes, save money

Findacure, a rare diseases charity based in Cambridge, says repurposing existing drugs to treat rare diseases will improve patient outcomes and save the NHS money.

Diseases that affect less than 1 in 2,000 people are known as ‘rare diseases’. They are some of the world’s most complicated conditions, yet they are also the most underfunded and under-researched. Of the 7,000 rare diseases that have been identified to date, only 400 have a licensed treatment – that’s less than 6%.

So few treatments have been researched for rare diseases because it is not profitable for pharmaceutical companies to develop new drugs for such small patient populations. Drugs that are developed often have an astronomical price tag attached to them, meaning they might not be approved by the cost-effectiveness watchdog NICE and patients are still denied access to them.

Findacure is holding a conference to share their solution to these challenges: repurposing existing, off-patent drugs to treat specific rare diseases.

Since early 2016, Findacure has been developing a drug repurposing programme specifically for rare diseases. They hope their research will slow symptom progression, improve patient quality of life, and extend patients’ lives. But Findacure believe their project will also save the NHS money. As the drugs they will be researching are off-patent and already proved to be safe for use in humans. This means that they are much cheaper than newly discovered drugs, and their improvements on patient health could greatly reduce the cost of managing rare diseases to the NHS.

Findacure will be holding a conference on Rare Disease Day, Tuesday 28th February 2017, to share the ideas behind their drug repurposing project. It will take place at the Royal College of Nursing, near Marylebone in London.

The conference will introduce attendees to the concept of drug repurposing, before going on to explore its uses and potential for treating rare diseases. Six talks will be given by a variety of speakers, including clinicians, researchers, pharma reps, regulators, and patients involved in drug repurposing trials. Breaks between talks will be a perfect opportunity for attendees to network with the already equally diverse audience.

The event will also feature the launch of Findacure’s ‘open call’ for submission of drug repurposing projects. The open call will see Findacure representatives touring the country to invite researchers, clinicians, doctors and patient groups to submit and potentially get funding for their ideas.

Findacure would like to welcome any interested persons to join them at the conference.

Comments

The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
Post a new comment
Post

While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. We do not provide medical advice, if you search for medical information you must always consult a medical professional before acting on any information provided.

Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles.

Please do not ask questions that use sensitive or confidential information.

Read the full Terms & Conditions.

You might also like...
GLP1R medications show promise in reducing suicidal ideation among adolescents with obesity