Medicines repurposing gains traction as help is made available to researchers and clinicians

Dr Rick Thompson

Beacon: for rare disease

Blayne Baker

Beacon: for rare disease

Many clinicians are turning to existing medicines to find a treatment for their patients. This type of clinic-led drug repurposing is vital to the rare disease community, but the route to success is often challenging.

While rare disease knowledge within the medical profession is limited, some clinicians manage multiple people living with a rare condition. Many of these conditions have no effective treatment. Rather than settle for the status quo, such clinicians often tenaciously seek out a treatment option: turning to existing medicines that could benefit their patients. This type of clinic-led drug repurposing is vital to the rare disease community, but the route to success is often challenging.

Why innovative therapies are failing to reach patients

Over the past decade, Beacon: for rare diseases (a UK-based charity focused on rare disease patient group support) has seen several promising repurposing ideas hit roadblocks that delay or halt patient access to potentially life-changing treatments.

Some researchers struggle to find funding for a clinical trial or access the test drug from the pharmaceutical industry. Other trials can be delayed for months as they try to source a placebo. Many are blocked entirely as researchers struggle to turn their research hypothesis into an effective treatment that can be made available to patients.

Drug repurposing initiative

REMEDi4ALL is a new EU-funded research initiative that is driving the repurposing of medicines across the UK and Europe for all conditions with high unmet medical needs.

This trailblazing project is uniting those interested — or actively engaged — in drug repurposing to identify the barriers and bottlenecks faced when repurposing an existing medicine.

Through collaboration and ingenuity, the consortium is removing these barriers to bring new treatment options to those living with some of the world’s rarest and most neglected conditions. The project is providing the therapies and hope that were previously inconceivable.

Drug repurposing works to a heightened timeframe, as it can significantly reduce the time, money and resources needed to bring a therapy to patients.

Repurposing medicines work for high unmet medical needs

Time is not a luxury afforded to those living with a rare condition. Drug repurposing works to a heightened timeframe, as it can significantly reduce the time, money and resources needed to bring a therapy to patients.

Researchers are working with a drug they already know, eliminating the uncertainty over a drug’s safety and effectiveness. Academics and researchers can build on what is already known and reposition a drug to treat a new patient population that desperately needs a therapy.

Advances in repurposing medicines across the UK and Europe

REMEDi4ALL is one of many advancements in the drug repurposing space. This brand-new initiative follows the launch of NHS England’s new Medicines Repurposing Programme and LifeArc’s drug repurposing toolkit. These resources are the result of a more open attitude towards repurposing medicines to meet high unmet medical needs.

The support is there to initiate a repurposing project and avoid common pitfalls. REMEDi4ALL, the Medicines Repurposing Programme and LifeArc’s toolkit all streamline the drug repurposing process by providing the connections, expertise, tools and resources needed to succeed at any stage. Place patients at the heart of research and ensure that they are co-creators. Patients are best placed to know the needs, wants and preferences of the target community. Help make repurposing medicines the new norm, so those living with rare and ultra-rare diseases can see a treatment in their lifetime.