Evidence map›Paper›PMID 41152943›Full record

ArticleOrphanet journal of rare diseases2025

The EMA as a potential driver of competitiveness of orphan medicines in Europe: a focus on regulatory incentives and funding opportunities.

Maria Ana Gomez-Ferreria, Virginia Garcia-Muñoz, Alex Zwiers

Abstract readLetter
PubMed Full text at PMC Publisher
In one paragraph

Article 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. Cited by 1 paper, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed, 1 pooled it
–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

1 citing paper in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
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

3 authors.

Maria Ana Gomez-FerreriaZwiers Regulatory Consultancy, A ProductLife Group Company, Amsterdam, Netherlands. mariana.gomez@az-regulatory.com.
Virginia Garcia-MuñozZwiers Regulatory Consultancy, A ProductLife Group Company, Amsterdam, Netherlands.
Alex ZwiersZwiers Regulatory Consultancy, A ProductLife Group Company, Amsterdam, Netherlands.

Funding

Clinical Trials Grants ProgramNatural History Studies Grants ProgramRare Neurodegenerative Disease Grants Program
6 · The paper itself

Abstract

The revision of the European Union (EU) Pharmaceutical Legislation aims for a competitive environment for innovation in the EU, including actions to support the development and approval of orphan medicines. In this article, we look at the Food and Drug Administration (FDA) for references to support this goal and discuss some FDA programs fostering innovation and competitiveness for orphan drugs in the US, including the Priority Review Voucher (PRV) program for Rare Paediatric Diseases, the Orphan Drug Grants to support clinical trials in orphan conditions and the recently launched Rare Disease Evidence Principles (RDEP) process. We further share some thoughts on how similar programs could be implemented in the EU, posing the European Medicines Agency (EMA) as a key figure to improve competitiveness for the development of orphan medicines in Europe.

Indexed as

Orphan Drug ProductionDrug ApprovalEuropeEuropean UnionHumansRare DiseasesUnited StatesUnited States Food and Drug AdministrationClinical trialsEuropean Medicines Agency (EMA)Food and Drug Administration (FDA)Orphan drugsOrphan medicinesPharmaceutical legislationPriority Review Voucher (PRV)Rare Paediatric Disease

Identifiers

PMID41152943
PMCPMC12570519

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.