The Patient Partnership Value Chain:

When patients help define the priorities and shape the agenda, trials are better designed and targeted, endpoints are more clinically meaningful, time to market shortens, and access and outcomes improve. How are patients redefining the innovation agenda, for whom, and according to whose definition of unmet need? How has an enhanced role for patients throughout the Innovation Value Chain demonstrated how science moves faster when patient perspectives are built into the process?  What must the next decade look like if it is truly guided by those it seeks to heal? How can private enterprise, philanthropy, public institutions, and all stakeholders act not as competitors, but as stewards of shared responsibility with patients? How do we ensure that breakthroughs reach the bedsides of those who wait?  What will it take to make patient partnership the default?

Every protocol design decision is simultaneously a commercial and a patient matter. Experience shows that leveraging patients, AI, and other increasingly key factors in protocol design is a rational business decision that improves trial success rates, timelines, and avoids costly regulatory missteps. It is not just an ethical concession. Similarly, trial execution that leverages organized patient communities, AI, and decentralized clinical trials improves the scientific inputs and outcomes of research. Patients have proven value at every stage of the chain. But the field still counts meetings, not results. How do we move from inputs (number of interactions, percentage of trials with patient input) to a real measure of impact? How is a Patient-First approach improving endpoints, inclusion/exclusion criteria, patient-reported outcomes in trial design? In this era of digital precision and novel therapies, are we listening to those whose lives are at stake?  How are advocates, AI, and other tools further supporting decentralized trials, including enabling recruitment that reflects “real-world” populations, improving retention through remote monitoring and otherwise, and enhancing the representativeness and relevance of the data produced?

Even when the scientific and regulatory value have been proven, therapies costing thousands per month (GLP-1s) or millions overall (gene therapies) are not accessible to all who need them. The patients who contributed their data and their trust to build this medicine are often left behind. Access is not a post-innovation problem – it is an innovation design failure. What lessons can be shared from the ALS innovation and patient engagement experience? How can markets where patients are meaningfully included in pricing and coverage decisions produce different access outcomes? What does the ALS experience tell us about the future of pricing policies?  How is the patient’s role in market launch, price setting, coverage decisions, and ensuring access at the last mile changing?