Charities, researchers and others hoping to repurpose existing medicines for treating other diseases can now find help and support in the form of a new free online toolkit developed by LifeArc and the Medical Research Council (MRC). The new toolkit was developed in consultation with scientific, industrial and regulatory experts and launched today at Beacon’s annual Drug Repurposing for Rare Diseases Conference in London.

Medicines repurposing – hope for patients 

Repurposing – finding new uses for existing treatments – offers a potentially rich source of effective treatments for many different diseases. But taking a promising candidate all the way through to patients remains hugely challenging due to the potential roadblocks on the way.

The process of finding new uses for existing drugs achieved a high profile in the fight against COVID-19 with repurposed medications, such as dexamethasone and rheumatoid arthritis treatment, tocilizumab, used to treat severely ill patients. And it’s easy to see the appeal – repurposing offers a cheaper and faster route to getting potentially life-changing new treatments to patients. And approved medicines also have well-established safety profiles.

But not-for-profit organisations, patient-led groups and researchers can find it challenging to navigate the complex commercial and regulatory landscape when undertaking drug repurposing projects. Unfortunately, this can result in promising medications failing to reach patients.

Last year, we held a workshop to explore opportunities and challenges in the field. It highlighted that there was a need to improve awareness of the barriers that can be faced, and ways to overcome them, to increase the likelihood of potentially life-changing medicines reaching patients.

Our approach – practical guidance

With this in mind, together with the MRC, we drew on expertise from a range of collaborators to create the MRC LifeArc Repurposing Medicines Toolkit. It consolidates and simplifies the complexities of repurposing into a clear and actionable guide, highlighting common activities, issues and considerations at each development stage including:

• research steps for demonstrating safety and efficacy
• the regulatory environment
• patient engagement
• accessing the medicine and existing data
• intellectual property (IP)
• funding sources

The toolkit also includes case studies from people who describe their own experiences of driving projects to repurpose medicines. They share the challenges they have faced and how they have overcome them – and give their advice to others considering embarking on a similar journey.

While the path to patients will be unique for each project, the new toolkit will provide useful guidance anyone embarking on a repurposing journey. It will assist them in navigating the potential roadblocks on the way and find ways to overcome them – helping to improve the likelihood of potentially life-changing medicines the people who need them.

We’re really excited that people can start using this free online resource today and would welcome any feedback so that we can continue to improve it, as well as hear how it is being used.