Evidence map›Paper›PMID 35471559›Full record

ReviewTherapeutic innovation & regulatory science2022

Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases.

Carla Epps, Ralph Bax, Alysha Croker, Dionna Green, Andrea Gropman, Agnes V Klein, Hannah Landry, Anne Pariser, Marc Rosenman, Michiyo Sakiyama and 5 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Therapeutic innovation & regulatory science, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
16citing 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

16 citing papers in PubMed, 1 synthesis or guideline pooled it.

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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

15 authors.

Carla EppsOffice of Pediatric Therapeutics, Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA. carla.epps@fda.hhs.gov.
Ralph BaxPaediatric Medicines, Scientific Evidence Generation Department, European Medicines Agency, Amsterdam, The Netherlands.
Alysha CrokerOffice of Paediatrics and Patient Involvement, Health Products and Food Branch and Director General's Office, Biologic and Radiopharmaceutical Drugs Directorate Health Canada, Ottawa, ON, Canada.
Dionna GreenOffice of Pediatric Therapeutics, Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA.
Andrea GropmanNeurodevelopmental Disabilities and Neurogenetics, Children's National Medical Center, Washington, DC, USA.
Agnes V KleinOffice of Paediatrics and Patient Involvement, Health Products and Food Branch and Director General's Office, Biologic and Radiopharmaceutical Drugs Directorate Health Canada, Ottawa, ON, Canada.
Hannah LandryOffice of Paediatrics and Patient Involvement, Health Products and Food Branch and Director General's Office, Biologic and Radiopharmaceutical Drugs Directorate Health Canada, Ottawa, ON, Canada.
Anne PariserOffice of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA.
Marc RosenmanMary Ann & J. Milburn Smith Child Health Outcomes, Research, and Evaluation Center, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.
Michiyo SakiyamaPediatric Drugs Working Group, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.
Junko SatoPediatric Drugs Working Group, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.
Kuntal SenNeurodevelopmental Disabilities and Neurogenetics, Children's National Medical Center, Washington, DC, USA.
Monique StoneAdvanced Therapies Unit, Prescription Medicines Authorisation Branch, Therapeutic Goods Administration, Department of Health, Woden, ACT, 2606, Australia.
Fumi TakeuchiPediatric Drugs Working Group, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.
Jonathan M DavisDepartment of Pediatrics, Tufts Medical Center and the Tufts Clinical and Translational Science Institute, Boston, MA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The literature thoroughly describes the challenges of pediatric drug development for rare diseases. This includes (1) generating interest from sponsors, (2) small numbers of children affected by a particular disease, (3) difficulties with study design, (4) lack of definitive outcome measures and assessment tools, (5) the need for additional safeguards for children as a vulnerable population, and (6) logistical hurdles to completing trials, especially with the need for longer term follow-up to establish safety and efficacy. There has also been an increasing awareness of the need to engage patients and their families in drug development processes and to address inequities in access to pediatric clinical trials. The year 2020 ushered in yet another challenge-the COVID-19 pandemic. The pediatric drug development ecosystem continues to evolve to meet these challenges. This article will focus on several key factors including recent regulatory approaches and public health policies to facilitate pediatric rare disease drug development, emerging trends in product development (biologics, molecularly targeted therapies), innovations in trial design/endpoints and data collection, and current efforts to increase patient engagement and promote equity. Finally, lessons learned from COVID-19 about building adaptable pediatric rare disease drug development processes will be discussed.

Indexed as

Biological ProductsCOVID-19 Drug TreatmentChildDrug DevelopmentEcosystemHumansPandemicsPublic HealthRare DiseasesBiological ProductsPediatric drug developmentRare diseasesRegulatory policy

Identifiers

PMID35471559
PMCPMC9040360

What OpenQuestion holds

Textmetadata
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.