Biopharma sponsors, medical device companies, and clinical research organizations have earnestly started to rely in part on dialog with patients to help inform study protocols and ensure patient perspectives are included in clinical development plans.
These early (and we hope, often) engagements with patients are not only welcome and encouraged, but are also helping to demonstrate that patients are invaluable—and data-rich—stakeholders with the ability to contribute meaningfully to both regulatory and reimbursement decisions.
Speaking the same language
Although including patient perspectives has become a commonly accepted and overall beneficial practice to help inform clinical trial design (recruiting, selection of therapeutic approaches, determining patient-important endpoints, etc.), standards for measuring, analyzing, and incorporating these data into downstream decision-making are lacking.
We still lack scientifically valid metrics to assess patient perspectives data reliably, and we further still lack a wide breadth of patient-informed benefit-risk frameworks for use by regulators and payers.
This looks to be changing, however, as numerous organizations and agencies globally are working to help establish common metrics and frameworks to assess and implement patient experience data into areas beyond clinical development.
Most encouragingly, the 21st Century Cures Act of 2016 addressed the importance of patients as partners by requiring sponsors to include patient experience data to assist in regulatory decision making. To meet these requirements, the FDA aligned its PDUFA VI commitments to include development of a four-part guidance series on patient-focused drug development.
The first of these guidance documents (released in June 2018) not only recognizes the value of patient experience data, but also emphasizes the need for greater uniformity of data collection and benefit-risk assessment. To echo the public comments from the Friedreich's Ataxia Research Alliance on the first guidance, collecting real-world evidence from the daily lives of trial participants—and considering that evidence in regulatory review and reimbursement decisions—is sufficiently important and new to the patient community.
Similarly, EUPATI—a project of the Innovative Medicines Initiative—also addressed the need for a consistent framework for patient engagement across Europe by developing a series of four guidance documents covering industry-led research and development, ethics committees, regulatory authorities, and health technology assessment (HTA).
Published in 2018, its Guidance for Patient Involvement in Regulatory Processes set out objectives for patient involvement in regulation and recommends concrete suggested working practices such as establishing a network of patient organizations and a forum for dialog and exchange between regulators and patient organizations.
Converging evidence generation
EUPATI’s guidance concerning patient involvement in HTA is most useful for global biopharmaceutical companies in that it further demonstrates the ongoing convergence of regulatory and reimbursement evidentiary requirements, as well as the need for real-world data (RWD) to supplement traditional, approval-driven data obtained from randomized clinical trials.
Beyond clinical development, patient engagement must span the entire lifecycle, and capturing patient experience data can inform a whole host of downstream decisions relevant to regulatory and reimbursement agencies.
Real-world data are considerably important as more and more atypical therapies are being discovered and tested (think: gene therapy, CAR-T cell therapy, among others). To maximize the public health effect of these promising therapies, it’s paramount for the industry to engage with patients, patient advocacy groups and other stakeholders to develop proper frameworks that can reliably measure benefit-risk and contribute to meaningful health technology assessment of these novel treatments. Working with a patient community to develop a natural history database, for example, enhances the ability to understand the benefits, risks, and patient experience with a new therapy in a real-world population.
One such effort germane to stakeholders across the healthcare spectrum is the Medical Device Innovation Consortium’s 2016 patient-centered benefit-risk framework, a document created to assist regulators in reviewing new device applications.
Stressing the notion that patients vary greatly in the degree to which they will accept risk for a given benefit, the framework recommends three key roles for patient preference information: 1) framing benefit-risk issues, 2) identifying subgroups of patients with differences in preferences, and 3) providing information for quantitative benefit-risk modeling. Authored by experts from industry, academia, government, and patient advocacy groups, the MDIC’s framework offers a wonderful example of how patient preference and experience data can be quantified and incorporated into regulatory and reimbursement decision making.
The European Medicine Agency’s 2018 strategy document also acknowledges the convergence of regulatory and reimbursement evidentiary requirements. The agency emphasizes the need to “ensure the evidence needed by HTAs and payers is incorporated early in drug development plans,” and additionally calls for an “information exchange with HTAs to support bridging from benefit-risk to relative effectiveness assessment.” This type of improved evidence generation and the call for common metrics underscore the need to better capture patient experience data during the development and evaluation process, potentially making both clinical development and regulation more cost-effective.
Reducing uncertainty
All of the examples mentioned above—and there are several others—illustrate the ways in which data gleaned from patients can be utilized, are being utilized, and should be utilized across a therapy’s lifecycle.
As reimbursement realities are now as important as regulatory approval, we now must evolve the idea of patient engagement from unstructured inclusion of patient input in clinical research, to one that includes a more holistic view which captures the entire patient experience in an organized and consistent manner.
With a mutual understanding amongst stakeholders concerning the metrics, frameworks, and data that contribute to the full patient experience, we can go a long way toward reducing regulatory and reimbursement uncertainty that often plagues new therapies.
