There are calls from different healthcare stakeholders to further enhance patient engagement in medicines development . Patient involvement in clinical trials is the most cited area of collaboration between the pharmaceutical industry and patients. Contributions from patients include input into clinical protocols , the wording of informed consents, patient-reported outcomes tools , benefit-risk discussions and the development of materials to encourage patient recruitment. Beyond clinical research, areas for potential collaborations include improvement of adherence to treatments by applying patient insights to predict reasons for non-adherence.
Another area of partnership between patients and the pharmaceutical industry relates to effecting changes in public policy and advocating on topics for improving general well-being. The topics of patient empowerment, access to therapeutic options and policy-focused advocacy are of high interest to all stakeholders in the health environment. Prevention of cancer, as well as cardiovascular diseases (CVDs), provides examples of impactful advocacy driving changes in public policy. The combined efforts of health agencies, professional societies, patient advocates and not-for-profit organisations helped establish standards for cancer screening [14, 15]. These have significantly improved screening test quality and resulted in reduced mortality from colorectal cancer and female breast and cervical cancer . Globally, CVDs are the number one cause of death, even with the decline in mortality over the years [17, 18]. World Health Organization interventions for reducing CVDs include strategies to reduce harmful use of alcohol, comprehensive tobacco control policies, and taxation to reduce the intake of foods high in fat, sugar and salt . CVD patient communities in the US and Europe have a strong presence in raising awareness of disease risk factors and advocating for policy-related changes and public campaigns [20, 21].
Often, chronic disease management and preventive health programmes focus on promoting informed lifestyle choices, risk-factor modification and active patient self-management . These programmes include providing patient information but, at the same time, require individuals to have a certain level of understanding and engagement. Focusing on research and development, initiatives such as the European Patients’ Academy (EUPATI) aim to educate patients and encourage their involvement in the process of developing new medicines . A pan-European initiative established in February 2012, EUPATI was a 5-year public–private partnership funded by the Innovative Medicines Initiative (IMI). It was patient-led, coordinated by the European Patients’ Forum (EPF), with other public bodies (European AIDS Treatment Group [EATG], Patients Network for Medical Research and Health [EGAN] and EURORDIS—Rare Diseases Europe) in key roles. The strong, multi-stakeholder consortium spanning almost 30 organisations, academia and industry has built competencies and capabilities among patient advocates and the health-interested public.
EUPATI provides objective and up-to-date information to patients and patient advocates. The programme has not only pioneered a paradigm shift towards intensified patient involvement in medicines development but has also fostered a trustful partnership between patient organisations, science and industry. Approximately 96 patient advocates have completed a training course based on a ‘EUPATI Toolbox’ and ‘EUPATI Internet Library’ in seven languages, as well as publicly available educational materials. Patient advocates trained by EUPATI now play influential roles in patient groups and collaborate with the pharmaceutical industry, regulatory authorities and health technology assessment bodies (Table 1). After becoming publicly accessible online, the EUPATI Toolbox on medicines development had more than 181,499 unique users to date. Additionally, EUAPTI has delivered four guidance documents for patient involvement with industry, ethics committees, regulators and HTA bodies. Building on its achievements, EUPATI will continue as an EPF-led multi-stakeholder partnership programme.
Patient Focused Medicines Development (PFMD) a not-for-profit, multinational coalition of patients, patient stakeholders and the pharmaceutical industry, is another initiative aiming to facilitate an integrated approach to medicines development with diverse stakeholders . The PFMD ensures that the patient perspective is the starting point when identifying priorities and developing solutions to meet patients’ needs. Formed as an equal collaboration among patient groups, patients and the pharmaceutical industry, the organisation has adopted a trans-Atlantic setup that reflects its global intent. In December 2016, the IMI in Europe launched a call with many opportunities for patient engagement, including a dedicated topic on patient engagement in the medicines lifecycle . The goal of this topic is to provide guidance on principles and processes for all stakeholders on the best ways to meaningfully engage patients at different stages of the medicines lifecycle.
For all these activities and some emerging guidelines , it should be noted that currently there is no best practice for patient engagement. Essential terminology such as patient involvement and patient engagement are often used interchangeably. However, the initiatives mentioned above should lead to greater alignment and mobilise future patient populations who are interested in gaining additional clinical/epidemiological data related to their treatments. Many pharmaceutical companies have made commitments for the responsible sharing of their clinical trial data. To this end, some pharmaceutical companies publish lay summaries of their clinical trial data on corporate websites. UCB has piloted using lay abstracts, written in plain language, in congress posters and peer-reviewed publications [27,28,29,30].
Metrics on how to measure the success of patient engagement are often lacking or require greater robustness or validation. One potential example includes patient input into protocols, leading in turn to patient-friendly information that can speed recruitment to clinical trials. Nevertheless, the lack of a control group in such circumstances makes it difficult to measure the benefits of patient engagement and continues to pose challenges in obtaining budget or resources and gaining endorsement for similar activities.
The pharmaceutical industry is highly regulated and patients and other participants need to be aware of compliant and ethical rules regarding interactions with companies. As patient-focused alliances become more widespread, clarity and transparency regarding initiatives will be crucial for all contributors. Best practices for patient engagement can define the dos and don’ts of relationships. The skill set and competencies of those wishing to partner is essential in contributing to positive experiences and outcomes of any initiative. Although some high-level industry-wide guidance is available (e.g. from the European Federation of Pharmaceutical Industries and Associations [EFPIA] and the Biotechnology Innovation Organization [31, 32]), practical tips on engagement are often lacking. UCB has developed internal recommendations, which give guidance when engaging with both patients  and patient groups (see box below).