Indication Screening: Using Early Health Technology Assessment to Prioritize Potential Target Indications for Platform Health Technologies
Author(s)
Nick Dragojlovic, PhD, Toluwase Akinsoji, MSc, Mark Harrison, PhD, Larry D. Lynd, PhD;
University of British Columbia, Faculty of Pharmaceutical Sciences, Vancouver, BC, Canada
University of British Columbia, Faculty of Pharmaceutical Sciences, Vancouver, BC, Canada
OBJECTIVES: The pairing of reprogrammable payloads (e.g. mRNA/siRNA) with scalable delivery systems like lipid nanoparticles has created the opportunity for drug developers to address a wide variety of diseases using the same technology platform. As a result, a key decision problem for groups developing these capabilities both in academia and industry is choosing which lead indication(s) to focus on at the preclinical stage.
METHODS: In this conceptual paper, we present a framework for a novel type of early health technology assessment that can be used to inform these decisions.
RESULTS: Our Indication Screening framework has four steps. The first involves creating a list of priority indications in close collaboration with the life sciences team. While criteria for defining the initial indication pool will vary depending on the technology, a key step in narrowing the indication set is to rate them for scientific suitability. Next, we define a set of indicators of unmet need, cost-offset potential, and addressable market size that can be compared across the included indications even if they are not in the same disease category - e.g. by avoiding disease-specific outcome measures that are not shared across indications. The third step involves systematically collecting evidence for each indication-indicator pair using a targeted literature review. A key challenge is balancing the breadth and depth of data extraction with budgetary constraints and informational needs at an early stage of product development. Finally, the evidence is iteratively synthesized, ultimately arriving at a high-level summary for each indication that can be used to identify the ones with the highest potential value for patients, payers, and manufacturers.
CONCLUSIONS: In our experience, an Indication Screening study can be conducted in an academic setting for ~$4,500CAD (~60 person-hours) per indication, and can bring the benefits of the “target assessment” practices used by pharmaceutical companies to academic teams and startups.
METHODS: In this conceptual paper, we present a framework for a novel type of early health technology assessment that can be used to inform these decisions.
RESULTS: Our Indication Screening framework has four steps. The first involves creating a list of priority indications in close collaboration with the life sciences team. While criteria for defining the initial indication pool will vary depending on the technology, a key step in narrowing the indication set is to rate them for scientific suitability. Next, we define a set of indicators of unmet need, cost-offset potential, and addressable market size that can be compared across the included indications even if they are not in the same disease category - e.g. by avoiding disease-specific outcome measures that are not shared across indications. The third step involves systematically collecting evidence for each indication-indicator pair using a targeted literature review. A key challenge is balancing the breadth and depth of data extraction with budgetary constraints and informational needs at an early stage of product development. Finally, the evidence is iteratively synthesized, ultimately arriving at a high-level summary for each indication that can be used to identify the ones with the highest potential value for patients, payers, and manufacturers.
CONCLUSIONS: In our experience, an Indication Screening study can be conducted in an academic setting for ~$4,500CAD (~60 person-hours) per indication, and can bring the benefits of the “target assessment” practices used by pharmaceutical companies to academic teams and startups.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
SA76
Topic
Study Approaches
Topic Subcategory
Literature Review & Synthesis
Disease
No Additional Disease & Conditions/Specialized Treatment Areas