SynthesisOrphanet journal of rare diseases2025
Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix.
Synthesis in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
Abstract
backgroundReimbursing orphan medicinal products (OMPs) presents both opportunities and challenges for national healthcare payers and health technology assessment (HTA) bodies because of their potential high benefits, large clinical uncertainties and high prices. To support a more structured application of (outcome-based) managed entry agreements (MEAs) intended to mitigate these OMP-related reimbursement challenges, a matrix was developed to facilitate reimbursement negotiations and, ultimately, patient access.
methodsA systematic literature review was performed, searching PubMed, Embase, and grey literature from 1 January 2000 until 1 January 2024 to globally identify reimbursement challenges (clinical-, cost-effectiveness uncertainties, or financial risks) described in relation to MEAs for OMPs. The data retrieved were used to develop a matrix that structures the drivers of managed access agreements to reduce financial risk and reimbursement challenges specific to OMPs.
resultsA total of 77 studies were included in the review, identifying 23 different types of MEAs for OMPs. The results indicated that more commonly known MEAs were designed to mitigate different reimbursement challenges, and more innovative MEAs and combinations thereof have been frequently described in literature. The selected case study of Myozyme
conclusionTo address reimbursement challenges for OMPs along their life cycle, it is valuable to consider both established and innovative, e.g., outcome-based MEAs. Combining reimbursement and payment models has the potential to address multifaceted reimbursement challenges. The developed matrix fills a gap in providing a structure for drivers of MEAs tailored to OMPs, enhancing decision-making processes and ultimate patient access to OMPs targeting high unmet medical needs.
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.