Levelling up UK rare disease research

Scientist in lab The UK needs new approaches to developing treatments for rare diseases, according to a new report led by UCL researchers describing how universities, the pharmaceutical industry and the NHS could improve life sciences collaborations. The report authors are calling for an independent British Office for Rare Diseases, comparable to the Office of Rare Disease Research in the US. Report co-author Professor David Taylor (UCL School of Pharmacy) 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. "There is a special problem with developing new treatments for very and ultra-rare disorders, which alone affect some 500,000 people in this country." Over 90 percent of rare diseases still lack satisfactory treatments. The report authors write that innovative ways of funding collaboration between university and researchers and scientists in industry could open new pathways to developing better therapies and secure the UK's position as a global centre for the life sciences.