Development and Application of a Novel Systematic Method to Identify Patients’ Unmet Needs: A Pilot Study in Inflammatory Bowel Disease

OBJECTIVES: Patients’ unmet needs (PUNs) can be a valuable tool to inform drug research and development. Hence, this study aimed to i) identify current methods used to reveal PUNs, ii) evaluate the applicability of those methods in practice, and iii) develop and validate a novel method to systematically determine PUNs.

METHODS: This mixed-methods study consisted of i) an orienting literature review on methods for PUN identification, ii) the development of a literature-based, systematic method to identify PUNs, iii) a pilot test of the novel method in inflammatory bowel disease (IBD), and iv) the evaluation and validation of the results of the pilot via multi-stakeholder discussions.

RESULTS: There is no systematic method available in literature to determine PUNs. The novel four-step methodology developed consisted of i) a scoping literature review of patient-centered research (such as patient preference studies and quality of life studies among patients) to determine patients’ medical needs in a given disease area, ii) the listing of available therapies authorised by the responsible regulatory authority, iii) the identification of PUNs by comparing patients’ medical needs and available therapies, and iv) a review of ongoing phase III clinical trials to assess whether treatments in development target outcomes that relate to the identified PUNs. Piloting this methodology in IBD revealed that: i) fatigue is a PUN, and ii) only one out of the 24 identified phase III IBD clinical trials explicitly included fatigue as clinical trial endpoint. Discussion with IBD specialists, patients, and patient representatives confirmed these results, being an important validation of the developed method.

CONCLUSIONS: To advance the discussion on the identification of PUNs and enable priority setting by relevant stakeholders such as Health Technology Assessment bodies, payers, and pharmaceutical industry, the methodology developed and applied in this study should undergo further revision and subsequent validation in other disease areas.