Today, scientific developments promise to transform the lives of people with serious conditions, but there is also increasing pressure on health budgets and a recognition that not all new therapies are truly innovative. Policymakers are looking for “valuable innovation” that responds to patients’ health needs while being affordable.
Patients ultimately experience the benefits and risks of new treatments; they provide the information and manage the personal risks attached to participation in a trial. Patients therefore have a moral right to be involved in the aspects of the design and process of clinical research. Besides this moral right, patient involvement in research adds concrete value. Thankfully, this is now widely recognised.
Patients as participants, no longer subjects
A recent series in The Lancet claimed that up to 85% of medical research may be wasted for various reasons. Even if the real figure is half of that estimate, we are still talking about a lot of waste. Much of it is due to patient’s priorities not being respected. Patients’ involvement in priority-setting and study design and implementation can help minimise waste and improve the quality and relevance of research. Patients’ knowledge is key to developing therapies that address their priority unmet needs; as users of the new treatments, patients’ perspectives and judgments about the relative importance of benefits versus risks, for example, are often different from those of research sponsors, medical professionals and regulators. Inclusion of so-called “patient-reported outcomes” (PROs) in study designs is becoming more common, but not yet standard practice; in addition, most existing PROs were not co-designed with patients, and prioritisation of “hard” clinical outcomes is still usually decided by clinicians. A recent study by Levitan et al. (2017) attempted to quantify the financial value of patient involvement, in terms of avoiding protocol amendments, improving enrolment, adherence and retention, and concluded that it can far exceed the investment needed.
The European Patients’ Forum believes the moral and material benefits of patient involvement necessitate a fundamental shift to patient-centred, patient-driven research. Patient involvement should be embedded in all aspects of clinical research as the “new normal”, with full recognition of the specific and unique expertise patients, as experts through lived experience, bring to the process not as “subjects” but as participants and as co-researchers.
The role of patient organisations
There are differences and similarities in the perspective of individual patients and representatives of patient organisations; both have their place in the process. Patient organisations bring the perspective of a wide group of patients, rather than individual experience only; they have grassroots networks they can survey and use to disseminate information effectively and quickly. Their specific role should be recognised and appropriately compensated, given that patient representatives are often volunteers and may live with one or more chronic conditions. Many patient organisations have long played an active role in clinical trials, particularly by identifying research priorities around unmet needs, in initiating research projects relevant for them, and often funding or conducting research themselves.
Clearly there is still much further to go. EPF is keeping a close watch on the implementation of the new EU regulation and its relevant aspects. One of our priorities is to inform and empower our member patient organisations to take action in their own area, including at the national level. Most recently, we produced a factsheet on Clinical Trials that explains the concept in simple terms and discusses the main aspects of the EU framework relevant for patients.
Patient involvement is no longer ‘optional’ as pressure grows to identify value in healthcare and ensure that future treatments provide concrete benefits for patients. EPF’s vision is of a fundamental paradigm shift “from doing research on patients… to doing (better) research with patients.”
 Smith, Hammad, Metcalf et al; (2016) “Patient Engagement at a Tipping Point. The Need for Cultural Change Across Patient, Sponsor, and Regulator Stakeholders: Insights From the DIA Conference, ‘Patient Engagement in Benefit Risk Assessment Throughout the Life Cycle of Medical Products’.” Therapeutic Innovation & Regulatory Science 2016, Vol. 50(5) 546-553. DOI: 10.1177/2168479016662902
 Genetic Alliance UK (2012). New medicines for serious conditions: weighing the risks and benefits. The verdict of a jury of patients. Report published in March 2012. https://www.geneticalliance.org.uk/media/1642/citizens-jury-report2012.pdf
 Levitan et al. “Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project.” Therapeutic Innovation & Regulatory Science. First Published July 17, 2017. https://doi.org/10.1177/2168479017716715
 The added value of patient organisations. Report commissioned by EPF, published November 2017. http://www.eu-patient.eu/globalassets/library/publications/epf_added_value_report_final.pdf