What can patients do to give input into clinical trial design? 

Business and profitability remain unquestionably important in the drug discovery community. However, the industry’s overarching goal of public health and the betterment of society have never been more apparent than in the aftermath of the pandemic and when observing the heights scientists reached through determination and collaboration in the fight against Covid-19. If anything, this period has been a reminder that people are the primary reason we do what we do, and the Patient Access, Healthcare & People track at Advanced Therapies Europe 2022 allowed DDW’s Multimedia Editor Megan Thomas to share several reflections on the topic.  

Chaired by Karin Blumer, Director of Global Patient Engagement at Novartis, the first event in this track, called ‘Patient Engagement: Driving Patient Advocacy to Inform Patient Centric Development’, looked into the multi-layered importance of involving patients in the drug development process, particularly in this case for gene therapy. Blumer said: “I think it’s a fascinating time to be talking about patient engagement. We are hopefully nearing the tail-end of a pandemic that made the whole world become participants in that deep learning experience of what clinical development is. I think never before did we have families sit on the couch and talk about Phase I, II and III clinical trials. Never before did we have families around the table asking, ‘Would I volunteer to be in a trial?’ Never before was the whole world a potential patient. I firmly believe that this will change the ecosystem of engaging patients both in the industry but also publicly and regulatorily. The times when patients were silent bystanders to the development are over.” 

Blumer asked: “What can patients do for other patients once they are empowered and know how to really give input into clinical trial design?” By way of answer, each case study presented in this session, from biologics to advanced therapy products, re-affirmed the importance of co-creating with patients across the life science cycle. Katherine Donigan, the Senior Director of Science & Regulatory at Biotechnology Innovation Organisation (BIO), spoke of the strategic approach to international harmonisation of gene therapy products.  

Donigan explained how cell and gene therapies are often indicative for rare or ultra-rare and serious conditions, that it is estimated that between 300 and 400 million people worldwide live with a rare condition, and yet the patient populations for these rare conditions are often very small. As a result, it is critical to achieve adequate enrolment that sufficiently power your trials. 

Anna Dickinson, the Life Sciences Policy Manager for Public Policy Projects (PPP), re-iterated the value of involving and engaging patients. PPP are an independent policy think-tank, offering practical policy analysis as well as government process analysis and development across a range of industries, including health care. Dickinson explained that it is essential for all voices to be heard in order to influence R&D processes and regulations.  

Simon Chandler, CEO of Rinri Therapeutics, made a case for the value of early patient engagement to build credibility and support for the clinical development of a first-in-class cell therapy for hearing loss. Chandler contextualises why the development of this therapy cannot progress without early patient engagement: “We are the only cell therapy that has ever been developed for hearing loss. There is no precedent, no reference points for a proof-of-concept for a cell-based therapy in hearing loss, and we don’t really understand what the patient appetite for ATMPs is.” 

Before the speakers joined on stage for a panel discussion and Q&A session, Blumer presented a case study on patient engagement and CAR-T, making the analogy of building a plane whilst flying it. To move forward in this industry and continue to develop life-changing therapeutics and healthcare solutions, the patient cannot be separated from the science. When the end-goal is people-centric, why would this not be the case for the development process?  

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