Why I “sold out” to big Pharma — a patient story
<p>The more I looked into the research pipeline for my condition, the more hope I had for the next generation, but the worse the news seemed for me personally. Despite over 90 DMD clinical trials active or recruiting worldwide today, almost none of the trials for new substances are open to over 21-year-olds, meaning that if they were successful, licences would be unlikely to include treatment of adults due to a lack of evidence. The question for me was why trials weren’t being run in adults. The answer largely lay in the complex regulatory system that didn’t support innovation in clinical endpoints, and the complexity of clinical trials.</p>
<p>There were a couple of hopefuls in the pipeline, treatments for respiratory failure and cardiomyopathy which offered some promise for me. A few years ago, I presented evidence to the EMA on the patient experience of respiratory failure, and to the MHRA in relation to an application to the Early Access to Medicines Scheme. Unfortunately, development of that medicine was halted due to a lack of clear evidence of effectiveness. It was a disappointing moment but a fascinating insight into drug development.</p>
<p><a href="https://medium.com/@jonhastie/why-i-sold-out-to-big-pharma-a-patient-story-72fe4d5bd37a"><strong>Website</strong></a></p>