No one has better knowledge of life with chronic disease than those directly involved—the patients, their families and those who care for them at home. They have good and bad experiences of using treatments, hospitals and clinics, and importantly, are aware of their own preferences. “This unique patient perspective is now considered credible and legitimate. It can be used to inform health policy and decision making,” stated Karen Facey, an independent consultant at the Health Technology Assessment Agency in Scotland. “Doing so will allow policy makers to provide better value for their patients,” Facey added.
Clinical evidence will never tell us exactly how treatments work in every setting. In recent years there has been increasing recognition of the patient voice in evidence-based policy making. “How can patients improve the quality of evidence?” asked Fischer. Identifying priorities for research, making content more accessible and understood by all and ensuring that research outcomes align with public needs are all positive ways that patients can enhance evidence.
The Patient Reported Outcomes, Burdens and Experiences Study (PROBE) is a good example of how people with hemophilia have collected data on outcomes they deem relevant to their lives. The rarity of hemophilia makes it even more important for patients to take part in policy making and influence their disease area.
Barriers to patient participation in health policy development are still present. Technical demands and a lack of time and resources can make patient engagement difficult. Eliminating bias and maintaining scientific integrity can be challenging. Tools to evaluate the impact of patient participation are lacking. Nonetheless, patients will always have the power of lobbying and a strong media appeal to position them for policy making.