Professor Faiez Zannad
Faiez Zannad is Professor of Therapeutics and Cardiology at the Centre d’Investigation Clinique Inserm and Université de Lorraine, CHU Nancy, France, and founder of the Global CardioVascular Clinical Trialists (CVCT)
The fundamental purpose of clinical research and new therapeutic development is to deliver treatments that effectively and safely improve health outcomes or well-being and that patients consider meaningful.
Although patients are ultimately the end-users of medical therapies, they have not traditionally been involved in the process of product development. This position is changing; the need to purposefully integrate patients as contributors and decision-makers throughout product life cycles is increasingly recognized by patients, investigators, regulators, payers, sponsors, and medical journals, but it is inconsistently implemented.
Patient engagement connotes a range of activities, from empowering individual patients to participate in healthcare decisions, to seeking patient advisement on various topics, to fully integrating patients as members of product development or clinical research teams. Patient involvement in therapeutic development may permit more rapid recognition of unmet needs and encourage alignment of product development priorities with patient priorities. Clinical trials may be conducted more efficiently when patients actively participate in protocol development and are able to minimize unnecessary or burdensome visits, tests, procedures or other factors that may influence enrollment or retention. Patient involvement can also ensure outcomes of value to patients are integrated in clinical trial designs, even if as secondary or supportive endpoints. This contribution will become increasingly important with the evolution of value-based healthcare and as health technology assessors seek reflection of patient preferences in value-based assessments.
The energy and enthusiasm for actively involving patients in the medical product development process has been growing in recent years. Encouraging progress has been made, with notable successes by regulatory agencies and public-private partnerships.
The FDA has a long-standing history of working to involve patients in aspects of drug development and approval. The first patient representative was included on an FDA Advisory Committee in 1991. Since that time, patient representatives serve as consultants to reviewers during review cycles, vote on advisory boards, and provide direct input to inform FDA decision making. The Patients’ and Consumers’ Working Party (PCWP) was established in 2006 to provide recommendations to the European Medicines Agency EMA on relevant topics of interest to patients involving medicinal products. The scope of tasks assigned to the PCWP involve adapting information appropriately for patients and consumers, contributing to the development of communication tools, increasing patient awareness of medicinal product usage, providing advice to EMA on product-specific matters, or collaborating with other stakeholders on medical therapy issues.
The barriers that limit patients from becoming involved in patient advocacy need to be systematically identified and strategies developed to overcome these hurdles. Leaders in clinical research and development within academia, industry and regulatory agencies should commit to creating an environment where patient involvement is essential to accomplish the common aim of bringing effective and safe products to patients that address relevant unmet needs from the patient’s perspective.