USING THE DELPHI METHOD TO IDENTIFY EVIDENCE GAPS IN SUPPORT OF HTA SUBMISSIONS FOR RARE PEDIATRIC CONDITIONS: A CASE STUDY IN NON-DYSTROPHIC MYOTONIAS
Author(s)
Kacper Swierk, MBiol1, Annette Beiderbeck, MSc, PharmD, PhD2, Alla ZOZULYA WEIDENFELLER, PhD2, Rosa OLIVEIRA, EMBA2, Mariska Van Aswegen, MBA3, H Lee, MSc4, Charlie Hewitt, MSc1.
1Remap Consulting UK Ltd, Cheshire, United Kingdom, 2Lupin Neurosciences, Zug, Switzerland, 3Lupin Neurosciences, Naples, FL, USA, 4Lupin Neurosciences, Barnet, United Kingdom.
1Remap Consulting UK Ltd, Cheshire, United Kingdom, 2Lupin Neurosciences, Zug, Switzerland, 3Lupin Neurosciences, Naples, FL, USA, 4Lupin Neurosciences, Barnet, United Kingdom.
OBJECTIVES: Robust evidence generation in rare pediatric disorders is challenging due to the scarcity of patients, the disease severity and ethical considerations regarding patients’ participation in a prolonged trial. Often, the evidence package includes gaps and is not satisfactory to support HTA submissions or inform clinicians' prescribing decisions. The opportunities for addressing evidence gaps are limited due to practical constraints; thus, there is a need for a structured methodology to reliably address uncertainty introduced by them.
Here we present how a clinician Delphi panel, supported by proxy-payer interviews, can help manufacturers identify and address evidence gaps in rare pediatric disorders.
METHODS: Data from pediatric patients with NDM enrolled in the MEX-NM-301 and MEX-NM-303 mexiletine trials were used as a case study. Interviews with 10 European proxy-payers identified HTA-relevant evidence gaps in these clinical trials. Subsequently, a two-round clinician Delphi panel was conducted. In round 1, 12 European clinicians independently identified evidence gaps, and assessed the importance of those highlighted by proxy-payers; in round 2, clinicians revised their responses based on the consolidated round 1 results to achieve consensus and rated possible approaches to address the gaps.
RESULTS: Both proxy-payers and clinicians identified small sample size and lack of long-term data as the most significant evidence gaps in the examined rare pediatric condition trials. Extrapolating adult data to adolescents and a consolidated case series study, were evidence generation strategies that would support HTA submissions and increase clinicians' confidence in prescribing mexiletine in pediatric patients.
CONCLUSIONS: A clinician Delphi panel, supported by proxy-payer interviews, reliably identified evidence gaps in the case study trials. Evidence gaps identified by proxy-payers and clinicians often overlap in such rare pediatric indications; thus, addressing them can reduce uncertainties during HTA appraisal and clinician prescribing. The Delphi Method is likely broadly applicable to identify evidence gaps in other rare pediatric conditions.
Here we present how a clinician Delphi panel, supported by proxy-payer interviews, can help manufacturers identify and address evidence gaps in rare pediatric disorders.
METHODS: Data from pediatric patients with NDM enrolled in the MEX-NM-301 and MEX-NM-303 mexiletine trials were used as a case study. Interviews with 10 European proxy-payers identified HTA-relevant evidence gaps in these clinical trials. Subsequently, a two-round clinician Delphi panel was conducted. In round 1, 12 European clinicians independently identified evidence gaps, and assessed the importance of those highlighted by proxy-payers; in round 2, clinicians revised their responses based on the consolidated round 1 results to achieve consensus and rated possible approaches to address the gaps.
RESULTS: Both proxy-payers and clinicians identified small sample size and lack of long-term data as the most significant evidence gaps in the examined rare pediatric condition trials. Extrapolating adult data to adolescents and a consolidated case series study, were evidence generation strategies that would support HTA submissions and increase clinicians' confidence in prescribing mexiletine in pediatric patients.
CONCLUSIONS: A clinician Delphi panel, supported by proxy-payer interviews, reliably identified evidence gaps in the case study trials. Evidence gaps identified by proxy-payers and clinicians often overlap in such rare pediatric indications; thus, addressing them can reduce uncertainties during HTA appraisal and clinician prescribing. The Delphi Method is likely broadly applicable to identify evidence gaps in other rare pediatric conditions.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HTA48
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
SDC: Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), SDC: Pediatrics, SDC: Rare & Orphan Diseases