Healthcare stakeholders recognize that there is a clear, ethical rationale to support patient engagement in clinical trials. Setting standards that describe how patient engagement is achieved will lead to research findings that are more pertinent to patients’ concerns and dilemmas.1 However, as there are limited comparative analytic studies to recommend a particular method that improves understanding of patients’ concerns1 and enables comparisons of the methods employed, it is unclear how to best engage patients and how to measure the success of engagement.

Patient engagement is well described by the Clinical Trials Transformation Initiative (CTTI) (figure 1). Sustained engagement with patients offers industry- and academic-led research first-hand insights that can help shape clinical trials to the benefit of all stakeholders in clinical development. The engagement process may improve the credibility of clinical trial results and the relevance to the population, and may have an empowering effect on participants. Several studies have also demonstrated a positive impact on patient enrollment, retention and the dissemination of trial results to participants and the community.3,4

Yet there is little guidance on how to perform this engagement. Regulatory authorities (e.g. the FDA5) paved the way by bringing the voice of patients into clinical development, and the EMA6 has been supporting patient engagement since its conception. Patients can get involved in every aspect7 of the regulatory lifecycle of a medicine, from pre-submission and evaluation through to post-authorization. In recent years, various organizations have taken on the difficult task of defining and determining strategies for patient engagement. A DIA study9, which was conducted in collaboration with researchers at the Tufts Center for the Study of Drug Development (Tufts CSDD), found that half of pharmaceutical and biotech companies surveyed have implemented patient-centric initiatives in drug development, and a similar percentage are piloting or planning to implement initiatives (figure 2). Top implementation barriers cited include difficulty securing buy-in internally and the lack of authority to implement initiatives. The research team found it difficult to quantify the impact of various patient-centric initiatives, as there is limited comparative data available.

Every other year the CISCRP2 Perceptions and Insights study serves as a guide and supports efforts in patient engagement by collecting, analyzing and publishing the preferences and opinions of thousands of patients on clinical trials.

With their more specific investigations, organizations such as Patient-Centered Outcomes Research Institute (PCORI)8 and CTTI9 have aided the development of strategies on how to achieve patient engagement in healthcare and clinical trials. CTTI is working with the FDA on a joint endeavor to create an ongoing, collaborative forum related to increasing patient engagement in medical product development. As a whole, however, 30 years after the FDA established the Patient Representative Program, patient engagement is still fragmented and relatively uncoordinated in the industry. The situation remains that there is insufficient data to evaluate the impact of patient engagement, although engagement is feasible in most clinical trials.

Overall, the major barrier to the evidence-based implementation of specific patient-centric initiatives is the lack of comparability of studies conducted in different organizations, and especially between the research conducted in academic and industry settings. Standardized tools for obtaining patient insights and measuring the effect of patient-centric initiatives will provide industry decision makers with the evidence and confidence to move forward with patient engagement initiatives. Meanwhile, increased comparability of patient engagement research will also enable the industry to identify trends and react to change that offers the potential to develop new medicines more efficiently.

With the advances made and momentum gained in recent years, patient engagement is headed in the right direction, but a paradigm shift towards increased transparency, harmonization and standardization is needed to allow evidence to synergize and further improve our knowledge of patient engagement. At the present time, there are no best practices of patient engagement on the basis of comparative evidence. If progress to identify best practices is to be made, then clarifying and standardizing methodologies would be a good place to start. 

For correspondences, email Emil.Hoeck@INCResearch.com.

References:

1. BMC Health Services Research, 2014|14:89|DOI: 10.1186/1472-6963-14-89
2. CISCRP. www.ciscrp.org/our-programs/research-services/perceptions-insights-studies last accessed 25th May, 2017.
3. Edwards V, Wyatt K, Logan S, Britten N. “Consulting parents about the design of a randomized controlled trial of osteopathy for children with cerebral palsy.” Health Expect. 2011, 14 (4): 429-438.
4. Swartz LJ, Callahan KA, Butz AM, Rand CS, Kanchanaraksa S, Diette GB, Krishnan JA, Breysse PN, Buckley TJ, Mosley AM, Eggleston PA. “Methods and issues in conducting a community-based environmental randomized trial.” Environ Res. 2004, 95 (2): 156-165.
5. Learn about Patient Engagement at the FDA. https://www.fda.gov/ForPatients/PatientEngagement/default.htm last accessed 25th May, 2017
6. Getting involved as a patient. EMA. http://www.ema.europa.eu/ema/index.jsp?curl=pages/partners_and_
   networks/general/general_content_000231.jsp&mid=WC0b01ac0580035bee last accessed 25th May, 2017   
7. Voices of Engagement. PCORI. http://www.pcori.org/get-involved/voices-engagement last accessed 25th May, 2017
8. Clinical Trial Transformational Initiative. www.ctti-clinicaltrials.org last accessed 25th May, 2017
9. “Results of Patient Engagement in Drug Development Study.” DIA/Tufts CSDD http://www.diaglobal.org/en/resources/press-releases/2016/10-31-patient-
   engagement-study