Monday, January 14, 2019

NatureNews Piece on Rare Genetic Disorders and Clinical Trials

NatureNews has this interesting piece about the challenges of balancing the requirement for rigorous scientific research with the moral imperative to foster medical innovation. A sample:

We do need rigorous and robust scientific processes,” says Alastair Kent, former director of Genetic Alliance UK, an umbrella body for more than 200 rare-disease patient groups. “But we also need new ways of proving the quality, safety and efficacy of new drugs.” Nick is trying to ensure that the journey will be smoother for others than it has been for him and his family.

....Around the world, regulators are under pressure to speed up the approval of therapies without sacrificing safety and efficacy assessments. Some of these efforts are controversial — a scheme in Japan to approve stem-cell treatments before they are known to work, for example and ‘right to try’ laws in the United States that allow people who are terminally ill to take unlicensed medicines. Nick co-founded another charity in 2012 to help people with rare diseases and their carers advocate for orphan-drug development.