Access to novel drugs and therapeutics for children and youth: Eliciting citizens' values to inform public funding decisions

• Published in: Health expectations : an international journal of public participation in health care and health policy Vol. 26; no. 2; pp. 715 - 727
• Main Authors: Gauvreau, Cindy L., Wight, Lisa, Subasri, Mathushan, Palmer, Antonia, Hayeems, Robin, Croker, Alysha, Abelson, Julia, Fraser, Brent, Bombard, Yvonne, Moore Hepburn, Charlotte, Wilson, Michael G., Denburg, Avram
• Format: Journal Article
• Language: English
• Published: England John Wiley & Sons, Inc 01.04.2023; John Wiley and Sons Inc; Wiley
• Subjects: Access; access to novel drugs; Adolescent; Canada; Caregivers; Child; Childhood; Children; Children & youth; children and youth; Childrens health; Citizens; citizens' values; Costs and Cost Analysis; Criteria; Data; deliberative public engagement; Disease; Dissemination; Drugs; Ethical dilemmas; Ethical standards; Funding; Governance; Health care; health technology assessment; Humans; Medical technology; Opportunity costs; Original; Pediatrics; Policy; public funding decisions; Qualitative analysis; Qualitative research; Research methodology; Social media; Social networks; Social organization; Teams; Technology assessment; Technology Assessment, Biomedical; Uniqueness; Values; Youth; Canada; health technology assessment; citizens' values; access to novel drugs; children and youth; public funding decisions; deliberative public engagement
• ISSN: 1369-6513; 1369-7625
• DOI: 10.1111/hex.13697

Introduction The unique evidentiary, economic and ethical challenges associated with health technology assessment (HTA) of precision therapies limit access to novel drugs and therapeutics for children and youth, for whom such challenges are amplified. We elicited citizens' perspectives about values‐based criteria relevant to the assessment of paediatric precision therapies to inform the development of a child‐tailored HTA framework. Methods We held four citizen panels virtually in May–June 2021, informed by a plain‐language citizen brief summarizing global and local evidence about the challenges, policy and programmatic options and implementation strategies related to enhancing access to precision therapies for Canadian children and youth. Panellists were recruited through a nationally representative database, medical/patient networks and social media. We inductively coded and thematically analysed panel transcripts to generate themes and identify priority values. Results The perspectives of panellists (n = 45) coalesced into four overlapping themes, with attendant subthemes, relevant to a child‐tailored HTA framework: (1) Childhood Distinctions: vulnerability, ‘fair innings’, future potential, family impacts; (2) Voice: agency of children and youth; lived versus no lived experience; (3) One versus Many: disease severity, rarity, equity, unmet need and (4) Health System Governance: funding, implementation inequities, effectiveness and safety. Participants broadly agreed that childhood distinctions, particularly family impacts, justify child‐tailored HTA. Dissent arose over whose voice should inform HTA and how such perspectives are best incorporated. Conclusions Citizens can offer unique insights into criteria relevant to the development or revision of HTA frameworks to capture holistic, societally responsive dimensions of value attached to unique contexts or populations, including children. Balancing the hopes and expectations of patients and caregivers for access to expensive but potential life‐altering therapies against the opportunity costs borne by encompassing health systems is a fundamental challenge that will require rigorous methods to elicit, weigh and reconcile varied views. Patient or Public Contribution A patient advocate served on the steering committee of this study and co‐authored this article. Key informants for the Citizen Brief included patient advocates and caregivers; a separate patient advocate reviewed the Brief before dissemination. Qualitative and quantitative data were collected from the general public and caregivers of children, with written consent.