Evidence map›Paper›PMID 34931373›Full record

ReviewBritish journal of clinical pharmacology2022

Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project.

Francesco Moretti, Fabrice Ruiz, Fedele Bonifazi, Elena Pizzo, Jenny M Kindblom, c4c HTA expert group

Open access · hybridAbstract readReview
In one paragraph

Review in British journal of clinical pharmacology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
2.7field-weighted citation impact, top 10% of its field
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

8 citing papers in PubMed, 12 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Article
  5. Article
  6. Article
  7. Access to novel drugs and therapeutics for children and youth: Eliciting citizens' values to inform public funding decisions.Health expectations : an international journal of public participation in health care and health policy · 2023
    Article
  8. Review
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 at 5 institutions in 4 countries.

Francesco MorettiArsenàl.IT-Centro Veneto Ricerca e Innovazione per la Sanità digitale, Padova, Italy.ORCID 0000-0003-0947-7860
Fabrice RuizClinSearch, Malakoff, France.
Fedele BonifaziFondazione per la Ricerca Farmacologica Gianni Benzi Onlus, Bari, Italy.
Elena PizzoDepartment of Applied Health Research, University College London, London, UK.
Jenny M KindblomPediatric Clinical Research Center at the Queen Silvia Children's Hospital at Sahlgrenska University Hospital, Göteborg, Sweden.
c4c HTA expert group
Consorzio per la Ricerca Sanitaria · ITGianni Benzi Pharmacological Research Foundation · ITMonal Group (France) · FRSahlgrenska University Hospital · SEUniversity College London · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The medicine development process is complex and requires time and effort to ensure safety, efficacy and quality. In paediatrics, this process is even more challenging, as it involves a subgroup of the population that already faces a considerable gap in the clinical evaluation of medicines and devices compared to the adult population. Moreover, access to therapies is heavily influenced by national health technology assessment (HTA) recommendations, which often form the basis for pricing and reimbursement decisions that affect the availability of effective treatments within the national health systems. Yet performing an HTA to assess the relative effectiveness and cost-effectiveness of a new children's treatment has several non-trivial implications, creating a critical issue for the paediatric population. In addition, the advent of innovative health technologies for children emphasises the need to empower the role of HTAs in paediatrics. This article aims at describing the most relevant elements of the drug development process in the paediatric field by focusing on the HTA. Particular attention will be paid to the factors that influence market access for new paediatric medicines and patients' access to treatment. The article will also highlight some central methodological challenges in conducting HTA in the paediatric field. Finally, the article will provide insight into how initiatives, such as conect4children, may subsequently reinforce HTA awareness in the paediatric community and strengthen collaborations through network mechanisms.

Indexed as

Technology Assessment, BiomedicalChildCost-Benefit AnalysisHumansaccess to treatmentdrug developmenthealth technology assessmentpaediatric

Identifiers

PMID34931373
PMCPMC9786554
OpenAlexW4200159242

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.