National Academies of Sciences Releases Report on Science of Patient Input, Citing Input from HP&M’s James Valentine

December 18, 2018By Hyman, Phelps & McNamara, P.C.

On December 13, 2018, the National Academies of Sciences, Engineering, and Medicine released the proceedings of a May 2018 expert workshop entitled “Advancing the Science of Patient Input in Medical Product R&D: Towards a Research Agenda.  The workshop, hosted by the Academies’ Forum on Drug Discovery, Development, and Translation focused on approaches to:

  1. Understanding the experience of patients with a disease or a medical condition;
  2. Gaining insights on patient perspectives and preferences regarding the benefits and risks of treatments; and
  3. Considering way that patient input could further continuous improvement of clinical trial development.

Experts across a range of backgrounds, including HP&M’s James Valentine, were convened at the workshop to discuss their experiences with collecting, analyzing, and applying patient input so that those approaches could be applied more broadly across medical product development.  In addition, experts in the development of patient reported outcomes (PROs) were invited to explore lessons learned from that field.  Ultimately, the goal was to explore early and clinical R&D applications for soliciting patient input that can help inform medical product decision-making.

The report documents contributions by speakers and participants alike in a 9-page summary.  Here are some noteworthy excerpts:

  • Theresa Mullin (FDA Center for Drug Evaluation and Research): “It is important to understand what aspects of disease burden and treatment matter most to patients and their families and how to measure them, [and] what aspects of clinical trials can be better tailored to meet the needs and interests of potential clinical trial participants…”
  • Mats Hansson (Uppsala University): “…there is no consensus on the definition and role of patient preferences or on how to conduct patient preference studies []. The literature review identified 32 patient preference exploration and elicitation methods that researchers have used when developing medical products and devices.”
  • Kathryn O’Callaghan (FDA Center for Devices and Radiological Health): discussing how CDRH now used patient preference information (PPI) to assess risk tolerance in a case involving at-home renal devices, “[o]ne finding was that patients expressed a range or a heterogeneity of preferences showing the importance of not oversimplifying with assumptions that they all want the same thing. Another key finding was that a substantial portion of patients was willing to accept risk levels associated with at-home, self-care HHD. Considering the results of this study, FDA cleared a change in the indications for use to remove the care partner requirement.”
  • Lynn Hagger (AstraZeneca): discussing an onsite study simulation involving 18 clinical trial participants, “[a]fter consulting with the mock trial participants, AstraZeneca made 21 changes to its clinical trial protocol… The changes AstraZeneca made increased recruitment and retention in two subsequent trials and produced a net savings of $1.1 million.”
  • Ron Bartek (Friedreich’s Ataxia Research Alliance): “beginning discussions with trial sponsors as soon as they have a therapeutic candidate and before designing their protocols could be beneficial. Trial sponsors could use patient generated natural history data to identify the optimal population for a particular therapeutic candidate and to define their inclusion and exclusion criteria, as well as primary and secondary endpoints…”

HP&M’s Valentine Recommends Qualitative Methods to Solicit Patient Experiences in Clinical Trials

The National Academies of Sciences report includes a suggestion made by HP&M’s James Valentine at the workshop to incorporate patient questionnaires or other qualitative techniques (e.g., patient interviews) to help sponsors and regulators assess the success of clinical trials.  This is an approach that may be particularly useful in rare disease clinical trials, which face methodological challenges of small, heterogeneous patient populations and a lack of validated, disease-specific outcome measures.  Questions can be developed to capture changes that patients experienced during the trial, which may not be of things formally captured by trial endpoints.  When reported in the patients’ own words, this patient experience data can add context to the clinical meaningfulness of clinical trial results.

Valentine, in partnership with a CRO and industry collaborator, has a forthcoming manuscript that will provide a methodologic overview for such an approach – so stay tuned!