Peter Marks wants drug developers to ask more stupid questions.
It’s part of the top Food and Drug Administration official’s plan to reinvigorate gene therapy, a field that has struggled despite significant technological advances. Some companies are shelving programs or going out of business, even when they have promising data.
The problems are numerous: The diseases are often exceptionally rare, limiting the potential market. Manufacturing at commercial quality is complex and expensive. Proving a drug works can be difficult, because there may be too few patients to run a traditional randomized study.