By Barbara Lopez Kunz, global chief executive, DIA
Patients have an irrefutable role in shaping the care they need. We have come a long way since the days when the idea of involving patients in healthcare product development was controversial, if not unheard of. In recent years, the healthcare ecosystem, and the role patients play in it, has evolved in a promising direction, leading to a much deeper understanding of the impact the patient voice can and should have in healthcare.
Across the entire ecosystem, from industry and academia to regulators and health technology assessments (HTAs), the goal to include patients is now on everyone’s agenda. And patient partnerships have flourished as a direct result of the confidence and sense of autonomy patients have gained. Patients, who are experiencing the illness first-hand, have become an invaluable resource, capable of clarifying their priorities and needs long before we embark on discovery and R&D. But how can we derive the most value from this development?
Numerous initiatives by DIA, the Clinical Trial Transformation Initiative (CTTI), and many other organizations, such as Faster Cures, the European Patients Forum (EPF), and the Patient Centered Outcomes Research Institute (PCORI), to name just a few, are providing us with important insights into patient engagement. They are giving us a sense of the type of metrics, operating models, as well as skills and experiences that are best suited to be effective. For example:
In all of these instances we have successfully come together and found common ground. But this is no time to rest on our laurels. We can and must learn from these experiences to tackle areas where we’ve only made modest, if any, progress in providing patients with the active role they deserve.
Patient-Centered Benefit-Risk Assessment
Despite the overall positive development over the past many years and the great advancements we’ve made in patient engagement since the early days, there is still plenty of room for improvement. One area where opinions and interests are still diverging and where we need to come together to focus our efforts is patient-centric benefit-risk assessment.
In benefit-risk assessment, benefits are weighed against the risk of harm when using a specific product as treatment. The benefits, risks, and uncertainties must be appropriately balanced from the patient’s point of view to truly meet the needs of those the product is intended for. But patients may weigh these treatment attributes quite differently compared to physicians, regulators, or sponsors. They may even differ as a group when trading off benefits and risks, or when using the same treatment for different conditions. And, when faced with a life-threatening illness, the patient may be willing to tolerate severe risk and uncertainty for the possibility of a life-saving benefit. In fact, the U.S.-wide Right-to-Try Act (signed into law on May 30, 2018) provides terminally ill patients access to experimental therapies that have passed basic safety testing in Phase 1 trials but are not yet fully approved.
Because patients benefit from effective treatments while also bearing the potential risks, their perspectives and judgements must be at the heart of benefit-risk assessment.
Finding Common Ground: Mission Impossible?
Although industry, regulators, and patient groups are increasingly seeing the value of assessing and incorporating patient preferences in benefit-risk assessment and are exploring ways to do so, there is still no clear consensus on the nature and extent of patient involvement:
Current Patient Roles In Benefit-Risk Assessment
Despite the issues, patient-centered benefit-risk assessment is emerging as the next key aspiration. Stakeholders are realizing that patient perspectives can indeed make us less uncertain about decisions made during product development, regulatory review, and post-market surveillance.
Various organizations have come together to drive research and policy activities that can provide tangible guidance on how to approach patient-centricity in benefit-risk assessment and to systematically incorporate data on patient perspectives into product approval applications. For example:
For patients to be truly integrated into the healthcare product development process and life cycle management, it will require consensus and conscious commitment from all stakeholders to work together to develop shared solutions and map a common path forward. It’s imperative to advance the dialogue, particularly among those who might address the issue from different perspectives. Only then are we able to overcome current barriers.
Some first initiatives have shown that this is possible, despite the cultural changes that need to take place among patients, sponsors, and regulators alike. At DIA we realize that, because each approach will be unique, we need to share our knowledge and experiences to make consistent progress in understanding how patients, regulators, and research sponsors can best work together to improve the medical development process — a topic that will be explored extensively at the upcoming DIA Global Annual Meeting in Boston in June.
We are committed to working with all stakeholders to find scientifically valid metrics to reliably assess patient preferences, establish frameworks for incorporating that information into product development, and examine how patient experience data can be incorporated into structured benefit-risk assessment frameworks in regulatory decision-making.
It won’t be easy, but with the right amount of commitment and passion, I’m confident we will make great strides toward benefit-risk assessment in healthcare product development that is truly patient-centric.
Note: Since the publication of this article, the FDA released a Patient-Focused Drug Development (or PFDD) draft guidance that provides methodological recommendations for collecting and submitting comprehensive and representative input from patients and caregivers. The guidance provides a framework for the inclusion of patient preference data in drug development and regulatory decision making and addresses commonly raised concerns, such as the lack of scientifically validated methods and their application to well-defined patient groups. This draft is the first of four guidances required by the 21st Century Cures Act.
About The Author:
Barbara Lopez Kunz, M.S., is global chief executive of DIA. She is leading the transformation of DIA through developing and implementing a strategy that focuses on engaging the global healthcare community in DIA’s neutral platform, to drive thought leadership and innovation in the development of therapeutics to improve the health of people worldwide.