Evidence map›Paper›PMID 42200984›Full record

ArticleJournal of market access & health policy2026

Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies.

Oliver Blandy, Pierluigi Lembo, Rebecca Folorunso, Karl-Johan Myren, Helene Chevrou-Severac, Simu K Thomas

Abstract read
In one paragraph

Article in Journal of market access & health policy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Oliver BlandyClarivate, 70 St Mary Axe, London EC3A 8BE, UK.
Pierluigi LemboClarivate, 70 St Mary Axe, London EC3A 8BE, UK.
Rebecca FolorunsoClarivate, 70 St Mary Axe, London EC3A 8BE, UK.
Karl-Johan MyrenHealth Economics and Outcomes Research, Alexion, AstraZeneca Rare Disease, Forskaren, Hagaplan 4, 11368 Stockholm, Sweden.ORCID 0000-0003-2065-2930
Helene Chevrou-SeveracHealth Economics and Outcomes Research, Alexion, AstraZeneca Rare Disease, Neuhofstrasse 34, 6340 Baar, Switzerland.
Simu K ThomasHealth Economics and Outcomes Research, Alexion, AstraZeneca Rare Disease, 121 Seaport Blvd, Boston, MA 02210, USA.ORCID 0009-0009-6710-3841

Funding

Alexion, AstraZeneca Rare Disease N/A
6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Indexed as

health technology assessmentnon-oncology orphan medicinal productsorphan drugorphan medicinerare diseasereal-world datareal-world evidence

Identifiers

PMID42200984
PMCPMC13214738

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.