A new report is calling on universities, the pharmaceutical industry and the NHS to work together to identify new approaches to developing treatments for rare diseases.

With nearly four million people in the UK directly affected by rare diseases and half a million with very and ultra-rare disorders, UCL researchers say more must be done to ensure effective treatments are available.

Over 90% of rare diseases still lack satisfactory treatments

Currently, over 90% of rare diseases still lack satisfactory treatments. With so many diseases still untreatable and incurable, rare diseases have an enormous impact on the physical and mental health of the UK population.

Indeed, if the distress caused to family members is taken into account, then one person in every five in the UK has their quality of life impaired by a rare disease.

And while report co-author Professor David Taylor says better treatments are becoming more readily available, further funding is needed to ensure progress continues.

He said: “Better treatments are already becoming available, particularly for more common forms of rare disease. The advent of cell, gene and other advanced therapies promises more gains. But progress will depend on funding early stage research in settings like universities and providing sufficient financial incentives for companies to develop and provide safe and effective licensed medicines.”

For this reason, the authors of the report are calling for an independent British Office for Rare Diseases, comparable to the Office of Rare Disease Research in the US. They say this could open new pathways to developing better therapies and secure the UK’s position as a global centre for the life sciences.

A “moral obligation” to develop the best possible medicines

The policy briefing, titled Medicines for the Few and the Many, is being published in coordination with the 2022 UCL School of Pharmacy/Royal Pharmaceutical Society lecture on The Future of the British Pharmaceutical Industry by Charlie Galvin.

Galvin, general manager of Amicus Therapeutics and chair of the UK Industry Rare Diseases Group, said: “We are at the dawn of a new golden age for medicine. At this pivotal moment in its history our industry will soon have the ability to help diagnose, treat and potentially cure many more diseases that have until now been untreatable and incurable. This progress will be based not only on fundamental advances in fields like biology and genetics but also on new understandings of the causes of social inequalities and global health problems.

“The challenges of developing rare and very rare disease medicines highlight the fact that there is a need for a new approach. We have a moral obligation to develop the best possible medicines and ensure that every person who may benefit has access to them.”