Incorporating the patient voice in clinical trials ‘mandates time and thoughtful consideration of every stakeholder’
Physician education is one way in which the industry is working to improve patient access to research, with few providers referring their patients to clinical trials – though the numbers are up for debate.
Still, the need remains to develop more physicians into clinical research investigators at a time when the industry is pushing for greater involvement from all stakeholders and focusing on implementing more ‘patient-centric’ approaches.
To further discuss the ways in which this patient centricity movement is shaping the way trials are designed, we caught up with Erem Latif, MBA, MS, director of patient engagement and patient-centered research at Evidera, who goes into detail, offering several examples of these efforts.
OSP: Historically, why has there been a disconnect with physicians as it pertains to clinical trials?
Erem Latif: The Affordable Care Act, passed in 2010, has had an incredible impact on the practice of medicine in the United States. While primarily targeting improved and expanded care for patients, there have been unique impacts on health care algorithms and practice patterns.
The cost and time associated with rigorous referral paradigms, mandated electronic health record (EHR) system implementation, and PQRS quality measure reporting has forced many single practice providers into group or managed care organizations.
Time spent, per patient visit, is tracked as are all lab and procedural orders—which must be pre-authorized for the most part. Ultimately, while attempting to expand access to health care, the ACA has driven medicine to be more machine-driven, with accountable care organizations (ACO’s) and payers seeking efficiency and cost-savings.
This directly impacts the engagement time and discussion between physician and patient… The physician has minimal time to speak to the patient and understand symptoms, quality of life issues, and/or treatment concerns.
Studies have indicated on average, the physician-patient interaction time to be estimated at 17.5 minutes per engagement. This leaves little time for discussion and evaluation of alternative paths of care, including clinical research participation. Many HCP’s balk at the required efficiencies leading to minimized interactions, as it adversely impacts their experience as a health care practitioner.
OSP: How is industry working to educate physicians? And how will this lead to improved patient access to clinical trials?
Latif: Educating providers on the evolution of clinical research to prioritize patient engagement may help reinstate their motivation to connect patients and viable research programs. Regulatory agencies could significantly impact the priority given to discussions on clinical research options by educating HCP’s on revised patient-focused drug development directives. Further, life science organizations can expand their dialogue with HCPs beyond new product launches to new research programs planned for the coming year(s).
Ultimately, active and engaged patients and patient advocacy groups could be their own greatest asset in expanding access to clinical research. Well-informed patients with knowledge of potential studies would stand to have a more meaningful interaction with their provider—despite the aforementioned time limits.
By reiterating their desire to have a greater voice in their care paradigms and options of treatment (to include clinical studies), patients may drive the needle in expanding physician referrals to research programs.
OSP: How is the patient centricity agenda shaping the way trials are designed?
Latif: While patient-centricity has been a buzzword in the health care industry for over ten years, its definition and implementation varies greatly by company and by solution. Software platforms support recruitment and retention, technology solutions to implement virtual trials, and data-mining tools to identify potential patients are all visible patient engagement tools.
From a more scientific perspective, patient-centricity has traditionally focused on the inclusion of patient reported outcomes (PRO) in trials – ensuring that treatment outcomes related to how patients’ feel or function are captured. Any status or insight captured on a patient’s health condition that comes directly from the patient is considered a PRO. PRO data are used to help guide care paradigms from a clinical perspective or inform policy development from a regulatory perspective.
However, we are now seeing patient engagement and involvement in clinical trials beyond standardized PRO assessments including patient input into protocol design to reduce barriers to participation and reduce operational burden; patient experience and patient preference studies to inform clinical program design or interpretation of benefits of treatments meaningful to patients; and qualitative embedded or exit interviews with patients participating in clinical trials to capture information about their experience in the trials, with treatment, and patient perception of meaningful treatment benefit.
Furthermore, patients are becoming more vocal in requesting access to information about clinical trial results and medicines in development or approved, and this is directly creating a focus on dissemination and patient-friendly medical information documents in order to communicate scientific findings to patients.
OSP: What impact does patient involvement in protocol design have and what does this mean for drug manufacturers?
Latif: As trials become more complex and expensive, the value of engaging patients in protocol design becomes more pronounced. Clinical research data over the last 10 years have highlighted the increasing complexity of clinical trials—from an increase in the number of procedures by 59% to an expansion of planned visits by 25%.1,2
This coincides with declining numbers of patients who are eligible and recruited per site, as studies are not being adequately designed for patient populations that actually exist.2 Thus, many companies experience consistent and costly delays as a third of trials fail to meet their recruitment targets and over half need to be extended to do so.3
High-profile initiatives have advocated for and facilitated patient engagement in research and decision making throughout the drug development process.
Examples include efforts by the Patient-Centered Outcomes Research Institute’s (PCORI’s) patient engagement rubric, which provides a blueprint by which to facilitate patient engagement initiatives throughout the research continuum, Transcelerate’s pivotal patient engagement toolkit, released recently, to guide industry professionals on best practices, and most recently, the FDA’s call for guidance on patient diversity in patient engagement: Enhancing the Diversity of Clinical Trial Populations—Eligibility Criteria, Enrollment Practices, and Trial Designs.4-10
Capturing and leveraging the patient voice has consistently shown to impact and improve timelines. In the Cochrane review “Methods to improve recruitment to randomized controlled trials,” the impact of multiple types of interventions on recruitment timelines were evaluated. Of the interventions considered, the impact of trial design, telephonic reminders, opt-out strategies, and financial incentives were all shown to have a positive outcome on recruitment efficiencies. 3
Another group has developed a financial model that considers the impact of patient engagement activities by leveraging empirical data and subjective parameters—ultimately highlighting nominal investments in patient engagement contribute to minimal protocol amendments and improved recruitment, adherence, and retention positively impacting timelines. 11
However, manufacturers are currently not making as much use of patient input into protocol design as they could. Obstacles include a lack of appreciation for how patient engagement can improve enrollment and retention rates, and development timelines that leave little room for patient engagement.
The evidence referenced above suggest that the time given to patient engagement in the trial design process can be more than offset in a reduction in the time required to deliver the trial.
Incorporating patient insights into a product plan mandates time and thoughtful consideration of every stakeholder, from patient to payer, in order to achieve maximum benefit. However, many life science organizations struggle to understand the true value of these patient engagement activities, unless delineated by regulatory guidelines.
This calls for a paradigm shift across the clinical research industry which stands to benefit every stakeholder in improved timelines, cost savings, and products that help treat patients more comprehensively.1 Getz kA, Campa RA. New Benchmarks Characterizing Growth in Protocol Design Complexity. Ther Innov Regul Sci. 2018 Jan;52(1):22-28. https://doi.org/10.1177/2168479017713039. Epub 2017 Jun 23.2 Tufts Center for the Study of Drug Development. Impact Report. Rising Protocol Complexity is Hindering Study Performance, Cost, and Efficiency. July/August 2018;20(4). Available at: https://csdd.tufts.edu/impact-reports/. Accessed February 20, 2019. 3 https://bmjopen.bmj.com/content/bmjopen/3/2/e002360.full.pdf4 Patient-Centered Outcomes Research Institute (PCORI). National Priorities for Research and Research Agenda. May 21, 2012. Available at: https://www.pcori.org/assets/PCORI-National-Priorities-and-Research-Agenda-2012-05-21-FINAL.pdf. Accessed February 20, 2019.5 US Food and Drug Administration. The Voice of the Patient: A Series of Reports from FDA’s Patient-Focused Drug Development Initiative. Available at: https://www.fda.gov/forindustry/userfees/prescriptiondruguserfee/ucm368342.htm. Accessed February 20, 2019.6 European Patients’ Academy (EUPATI). Available at: https://www.eupati.eu/. Accessed February 20, 2019.7 Congress.gov. H.R.34 – 21st Century Cures Act, 114th Congress (2015-2016). Available at: https://www.congress.gov/bill/114th-congress/house-bill/34/. Accessed February 20, 2019. 8 https://www.pcori.org/files/pcori-engagement-rubric. Accessed September 4, 2019. 9 https://transceleratebiopharmainc.com/patientexperience/patient-protocol-engagement-toolkit/. Accessed September 4, 2019. 10 https://www.regulations.gov/document?D=FDA-2019-D-1264-000111 Levitan B, Getz KA, Eisenstein EL. et al Assessing the Financial Value of Patient Engagement. Therapeutic Innovation & Regulatory Science. 2018, Vol. 52(2) 220-229. https://journals.sagepub.com/doi/pdf/10.1177/2168479017716715