By Debra Michaels, senior scientist, DIA Americas
Earlier this year, DIA Global Chief Executive Barbara Lopez Kunz authored a piece for Clinical Leader: Trial Return On Engagement: Quantifying Benefits Of Patient-Centric Initiatives. In that piece, Ms. Lopez Kunz revealed preliminary results of an ongoing collaborative Study of Patient-Centric Initiatives in Drug Development between DIA and the Tufts Center for the Study of Drug Development (CSDD), which found that while organizations across the entire healthcare product continuum aspire to be more patient-centric, there is a need for tangible guidance on how to approach patient-centricity as well as how to quantify the benefits of PCIs (patient-centric initiatives) and measure return on engagement (ROE).
At the recent DIA 2017 Global Annual Meeting, the session Quantitative Metrics to Capture the Value of Patient Engagement provided insights from recent work to define impact metrics for PCIs.
Mapping research sponsor and CRO-implemented and planned PCIs to measures of their impact in four domains of drug development produced the framework for a metrics “toolkit” in the recently completed first phase of the DIA-Tufts CSDD study. Measures, both quantitative and qualitative, that had been used to assess the impact of implemented PCIs were identified in 121 unique cases. The measures were categorized as evaluating impact on trial cost, speed, or quality, or on the patient. They were then further grouped by ability to measure impact within four domains in medical product development: the long-term drug development portfolio (e.g., development timelines, program success rates), internal and external reach (e.g., number of study volunteers, number of trials), study volunteer feedback (e.g., number of changes, number of missed assessments), and trial performance (e.g., number of protocol amendments, changes in protocol complexity, trial cycle time and length). The resulting toolkit, which will undergo further refinement in the second phase of the study, can be used to identify metrics for expressing return on engagement of specific PCIs in specific applications. Together with data quantifying the impact of the PCIs, these metrics provide an early look at approaches that are being successfully used to measure impact.
Metrics Clarify Impact Of Patient-Centric Initiatives (PCIs)
The study examined the value of specific metrics for determining ROE in the context of ease of collecting the data and found a wide variation in that relationship. For example, overall patient satisfaction with a study was considered a useful metric by 91 percent of the study respondents, but only 20 percent of respondents deemed it relatively easy to collect that data. In comparison, 82 percent felt that measuring the number of dropouts not due to serious adverse events (SAEs) or adverse events (AEs) was useful, and 67 percent believed such data relatively easy to collect. Here, models or standardized methods of gauging overall patient satisfaction would facilitate the collection of useful patient engagement impact data. In another example, 100 percent of respondents believed that measuring impact on overall development cycle time would be a useful metric, with only 27 percent feeling that data would be easy to collect. Measuring the study conduct duration based on first patient in to last patient out was cited as a useful metric by 80 percent of respondents, with 78 percent seeing these data as easy to collect. In strategic planning for measuring the ROE of patient engagement, it may thus be useful to begin by assessing impact in more focused areas.
The DIA-Tufts CSDD study also compared the cost and relative ease of conducting the PCIs with their reported impact. Results varied widely, as one would expect. Innovative partnerships tended to have the highest impact; in particular, advocacy group support and involvement, and patient advisory panels and focus groups had relatively high impact with relatively low cost and difficulty to implement. PCIs such as adaptive trial designs also had a relatively high impact but a greater cost and complexity of implementation. PCIs such as open trial design and crowdsourcing were relatively low cost to conduct, but implementation was considered more difficult, and reported impact was lower than for the PCIs previously discussed. E-consent, a much-discussed PCI, was considered to be moderately costly to conduct, moderately difficult to implement, and to have less impact relative to other PCIs. The ROE of a given PCI is dependent on the objectives set forth by the involved stakeholders, but comparisons of the relative cost, ease, and impact are a necessary component of the ROE assessment.
Quantitative Approach Assesses Financial Value Of Engagement
A quantitative approach to pharmaceutical project valuation, aimed at reducing concern about potential financial risks of patient engagement initiatives for clinical trials, has been developed by the Clinical Trials Transformation Initiative (CTTI). A public/private partnership co-founded by Duke University and the FDA, CTTI’s mission is to develop and advance the adoption of practices and tools to improve the quality and efficiency of trials. The approach addresses a contributing factor to one of three main barriers to the implementation of PCIs: regulatory and legal uncertainties around patient-sponsor interactions, lack of communication with patients and in the pre-competitive space among sponsors, and company cultures that are not patient-oriented due to multiple factors such as the fear of potentially associated financial risks.
Project valuation methodology concisely defines paths of success or failure in projects and assesses the costs, revenue, risks, and timing associated with each separately to calculate an expected net present value (ENPV) for each path. In the CTTI approach, the impact of patient input on successes and failures within each path are added, and the resulting changes in ENPV are calculated. A detailed description of the methodology can be found here. The project has demonstrated that such risk-adjusted financial models can assess the impact of patient engagement, thus reducing uncertainty around PCI investment decisions. A combination of empirical data and subjective parameter estimates showed that engagement activities with the potential to avoid protocol amendments and/or improve enrollment, adherence, and retention may add considerable financial value.
Mapping Tool Provides Framework For Patient Engagement
Similar to the lack of impact metrics for the rapidly growing number of patient-centric approaches and methods, the slow rate of diffusion or sharing of this knowledge raises concerns that precious resources may not be used optimally to the benefit of patients, if researchers are expending effort to “reinvent the wheel.” A Patient Engagement Global Landscape Mapping and Networking tool has recently been developed by Patient Focused Medicines Development (PFMD), an open, independent, multinational coalition to develop and drive implementation of patient engagement. The tool serves as a virtual hub to connect and cross-pollinate previously disconnected patient engagement initiatives, enabling stakeholders to share experiences and learn from one another. The mapping tool accepts submissions and is open to all for use, regardless of coalition membership, in an effort to develop a comprehensive view of the patient engagement landscape. Initiatives are categorized by broad focus area (e.g., efforts in research, education, advocacy, access), and the tool may be searched to yield reports based on medical scope, life cycle phase, geographic coverage, maturity, and other characteristics of the initiatives. One benefit of the PFMD mapping tool in terms of patient engagement metrics is that, as it becomes more complete, it can allow identification of potential tools to measure the impact of patient engagement.
Patient-centricity and PCIs are emerging as high-value and high-priority industry goals. The three different approaches discussed in this article can help capture and quantify the benefits of a wide range of patient-centric initiatives in healthcare product development and health research.
What approaches is your organization using to measure the value of patient-centric initiatives? Please share them in the Comments section below.
About The Author
Debra Michaels, MS, RD, serves as senior scientist for DIA Americas. Ms. Michaels is a lead for DIA’s patient engagement thought leadership efforts and brings extensive experience in biopharmaceutical development, public health, and clinical practice to her work in this area.
DIA is a global, multidisciplinary membership organization for professionals working in all aspects of the health care product life cycle. DIA’s upcoming Metrics in Patient-Centered Drug Development, (Oct. 18-19) will examine the core principles of measuring the ROE of patient engagement and demonstrate how current and emerging approaches are being applied by industry colleagues to measure engagement impact on outcomes of importance to patient, industry, regulatory, and payer stakeholders.