Dr Rick Thompson
CEO, Beacon: for rare diseases
For most rare patients, a lack of disease understanding means a treatment for their condition is unimaginable.
Consequently, much of the limited investment into rare diseases is in early-stage understanding of disease biology and identifying treatment concepts.
A challenging route to patients
Once a potential therapeutic is identified, getting treatments to market is more challenging. Success traditionally requires the skills, experience and funding of the pharmaceutical industry, which needs a viable business case to develop a drug. Unfortunately, tiny patient populations are only economically viable when treated with an exceptionally high-priced drug that the NHS struggles to fund. Thus, even patients with viable routes to treatment may be left with nothing.
An academic solution
To solve this problem, we must drive drug development from academia. We need to develop promising therapeutic ideas within universities and hospitals, without relying on market returns to fuel success. This is a significant challenge as researchers are trained and incentivised to test hypotheses, not develop drug products.
Recent innovations suggest
that policymakers and funders
recognise the need for change.
Investing in the development pathway
New projects are creating hope for academic drug development. The Rare Disease Research UK project encompasses 11 research nodes. Alongside the four new LifeArc Translational Centres for Rare Diseases, they represent an investment of over £50 million into the UK’s rare research. Similarly, EU-funded projects, such as REMEDi4ALL (focused on finding new uses for existing medicines) are helping academics deliver meaningful treatments to patients.
There are also early signs of innovation in the regulation and delivery of drugs to patients. The NHS Medicines Repurposing Programme potentially provides new routes to patients outside of the traditional model. Its initial success in the field of cancer prevention is promising for rare disease.
Hope for rare diseases
While a world where everyone living with a rare disease has access to treatment remains beyond our reach, recent innovations suggest that policymakers and funders recognise the need for change. With the correct support and incentives, we could be witnessing the first signs of a new academic drug development engine, able to complement the pharmaceutical industry and tackle the huge challenge of delivering new therapies to the world’s rarest patients.
