Evidence map›Paper›PMID 32883327›Full record

ArticleOrphanet journal of rare diseases2020

FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development.

Kathleen L Miller, Christine Mueller, Gumei Liu, Katherine I Miller Needleman, Janet Maynard

Abstract read
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In one paragraph

Article in Orphanet journal of rare diseases, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing 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

7 citing papers in PubMed.

  1. Article
  2. Review
  3. Article
  4. Review
  5. Natural History Studies and Clinical Trial Readiness for Genetic Developmental and Epileptic Encephalopathies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2021
    Review
  6. Article
  7. Article
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

5 authors.

Kathleen L MillerUS Food and Drug Administration, Office of the Commissioner, Office of Orphan Products Development, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA. Kathleen.miller@fda.hhs.gov.
Christine MuellerUS Food and Drug Administration, Office of the Commissioner, Office of Orphan Products Development, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA.
Gumei LiuUS Food and Drug Administration, Office of the Commissioner, Office of Orphan Products Development, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA.
Katherine I Miller NeedlemanUS Food and Drug Administration, Office of the Commissioner, Office of Orphan Products Development, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA.
Janet MaynardUS Food and Drug Administration, Office of the Commissioner, Office of Orphan Products Development, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThe Office of Orphan Products Development (OOPD) of the United States (U.S.) Food and Drug Administration (FDA) has awarded over 700 grants to conduct clinical trials of medicals products for rare diseases since 1983, leading to over 70 marketing approvals. However, despite recent progress in rare disease product development, thousands of rare diseases still have no approved treatments. An assessment of this clinical trial grants program was undertaken to provide an in-depth analysis of the characteristics and outcomes of the program. Results of this analysis will be used to inform future goals of the program, as well as internal data collection to continue to maximize the program's impact in supporting rare disease product development.

resultsBetween fiscal years 2007-2011, OOPD funded 85 clinical trial grants. These grants spanned 18 therapeutic areas, included all pre-approval phases (Phases 1-3), and approximately 75% of the grants studied small molecule drugs. Nine (11%) product approvals, of seven drugs and two devices, were at least partially supported by grants funded within this 5-year timeframe. Four of the seven drugs approved were new molecular entities (NMEs). The average time from funding to approval was seven years. We also found a suggested association between collaboration with multiple types of stakeholders and the success of grants, where we defined success as either positive or negative study findings or a future marketing approval.

conclusionsThe clinical trials funded by OOPD provided valuable information for future product development, and there were a notable number of approvals that occurred using the support of the grants program. There was a suggested association between collaboration and successful outcomes. Efficient and innovative trial designs and collaboration among stakeholders appear vital to continue to effectively bring products to rare disease patients. Ongoing program assessments will ensure that the funding continues to be used to optimally meet the treatment needs of the rare disease community.

Indexed as

Orphan Drug ProductionRare DiseasesDrug ApprovalHumansProgram EvaluationUnited StatesUnited States Food and Drug AdministrationClinical trialsGrantsOrphan drug actOrphan product grants programRare disease medical product developmentRare diseasesUS Food and Drug Administration

Identifiers

PMID32883327
PMCPMC7469294

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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.