ReviewInternational journal of technology assessment in health care2023
Uncertainty management in regulatory and health technology assessment decision-making on drugs: guidance of the HTAi-DIA Working Group.
Review in International journal of technology assessment in health care, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
13 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix.Orphanet journal of rare diseases · 2025Pooled it
- Uncertainty in Economic Evaluation: A Pragmatic Guide for Health Technology Assessment (HTA) Agencies in Resource-Constrained Settings.PharmacoEconomics · 2026Review
- Conflicts of interest as a barrier to the development of health technology assessment in Iran: a qualitative study.Journal of health, population, and nutrition · 2026Article
- Editorial: Regulatory science: addressing uncertainties in medicines development, evaluation and use.Frontiers in medicine · 2026Article
- Shaping the Future of DHT Assessment: Insights on Industry Challenges, Developer Needs, and a Harmonized, European HTA Framework.Journal of market access & health policy · 2025Article
- Evolving Recommendations for Patient Populations Among Oncology Medicines: A Quantitative and Qualitative Analysis.Clinical pharmacology and therapeutics · 2025Article
- Good Practices for Health Technology Assessment Guideline Development: A Report of the Health Technology Assessment International, HTAsiaLink, and ISPOR Special Task Force.International journal of technology assessment in health care · 2025Article
- Early access disparities in innovative therapies across the US, EU, China, and Japan.Frontiers in medicine · 2025Article
- Managed Entry Agreements for High-Cost, One-Off Potentially Curative Therapies: A Framework and Calculation Tool to Determine Their Suitability.PharmacoEconomics · 2025Article
- How can managed entry agreements contribute to coverage decisions in Latin America?International journal of technology assessment in health care · 2024Article
- Assessing the credibility of a drug's effects: identification and judgment of uncertainty by the Dutch Medicines Evaluation Board.Frontiers in medicine · 2024Article
- Response to uncertainty management in regulatory and health technology assessment decision-making on drugs: guidance of the HTAi-DIA Working Group - author's reply.International journal of technology assessment in health care · 2023Article
- What could health technology assessment learn from living clinical practice guidelines?Frontiers in pharmacology · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
23 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesUncertainty is a fundamental component of decision making regarding access to and pricing and reimbursement of drugs. The context-specific interpretation and mitigation of uncertainty remain major challenges for decision makers. Following the 2021 HTAi Global Policy Forum, a cross-sectoral, interdisciplinary HTAi-DIA Working Group (WG) was initiated to develop guidance to support stakeholder deliberation on the systematic identification and mitigation of uncertainties in the regulatory-HTA interface.
methodsSix online discussions among WG members (Dec 2021-Sep 2022) who examined the output of a scoping review, two literature-based case studies and a survey; application of the initial guidance to a real-world case study; and two international conference panel discussions.
resultsThe WG identified key concepts, clustered into twelve building blocks that were collectively perceived to define uncertainty: "unavailable," "inaccurate," "conflicting," "not understandable," "random variation," "information," "prediction," "impact," "risk," "relevance," "context," and "judgment." These were converted into a checklist to explain and define whether any issue constitutes a decision-relevant uncertainty. A taxonomy of domains in which uncertainty may exist within the regulatory-HTA interface was developed to facilitate categorization. The real-world case study was used to demonstrate how the guidance may facilitate deliberation between stakeholders and where additional guidance development may be needed.
conclusionsThe systematic approach taken for the identification of uncertainties in this guidance has the potential to facilitate understanding of uncertainty and its management across different stakeholders involved in drug development and evaluation. This can improve consistency and transparency throughout decision processes. To further support uncertainty management, linkage to suitable mitigation strategies is necessary.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.