Evidence map›Paper›PMID 30610746›Full record

ArticleClinical pharmacology and therapeutics2019

Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice.

Harry P Selker, Hans-Georg Eichler, Norman L Stockbridge, Newell E McElwee, Willard H Dere, Theodora Cohen, John K Erban, Vicki L Seyfert-Margolis, Peter K Honig, Kenneth I Kaitin and 2 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Clinical pharmacology and therapeutics, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing 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

14 citing papers in PubMed.

  1. Trial
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  4. Article
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  9. Article
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  11. Decentralised clinical trials in multiple sclerosis research.Multiple sclerosis (Houndmills, Basingstoke, England) · 2023
    Review
  12. Article
  13. Article
  14. 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

12 authors.

Harry P SelkerTufts Clinical and Translational Science Institute, Tufts University, Boston, Massachusetts, USA.
Hans-Georg EichlerEuropean Medicines Agency, London, UK.
Norman L StockbridgeUS Food and Drug Administration Center for Drug Evaluation and Research, Silver Spring, Maryland, USA.
Newell E McElweeBoehringer Ingelheim, Ridgefield, Connecticut, USA.
Willard H DereDepartment of Internal Medicine, Utah Center for Clinical and Translational Science, University of Utah School of Medicine, Salt Lake City, Utah, USA.
Theodora CohenTufts Clinical and Translational Science Institute, Tufts University, Boston, Massachusetts, USA.
John K ErbanCancer Center, Tufts Medical Center, Boston, Massachusetts, USA.
Vicki L Seyfert-MargolisMy Own Med, Inc., Bethesda, Maryland, USA.
Peter K HonigCollegeville, Pennsylvania, USA.
Kenneth I KaitinTufts Center for the Study of Drug Development, Tufts University, Boston, Massachusetts, USA.
Kenneth A OyeMassachusetts Institute of Technology Political Science and Center for Biomedical Innovation, Cambridge, Massachusetts, USA.
Ralph B D'AgostinoDepartment of Mathematics and Statistics, Boston University, Boston, Massachusetts, USA.

Funding

Johns Hopkins-Tufts Trial Innovation Center HEAL Pain ERN SupplementU24TR001609 · NCATS · JOHNS HOPKINS UNIVERSITY · PI HANLEY, DANIEL F · 2016 to 2022
$36.3M
NCATS NIH HHS U24 TR001609
6 · The paper itself

Abstract

Efficacy trials, designed to gain regulatory marketing approval, evaluate drugs in optimally selected patients under advantageous conditions for relatively short time periods. Effectiveness trials, designed to evaluate use in usual practice, assess treatments among more typical patients in real-world conditions with longer follow-up periods. In "efficacy-to-effectiveness (E2E) trials," if the initial efficacy trial component is positive, the trial seamlessly transitions to an effectiveness trial component to efficiently yield both types of evidence. Yet more time could be saved by simultaneously addressing efficacy and effectiveness in an "efficacy and effectiveness too (EE2) trial." Additionally, hybrids of the E2E and EE2 approaches with differing degrees of overlap of the two components could allow flexibility for specific drug development needs. In planning EE2 trials, each stakeholder's current and future needs, incentives, and perspective must be considered. Although challenging, the ultimate benefits to stakeholders, the health system, and the public should justify this effort.

Indexed as

Clinical Trials as TopicCost-Benefit AnalysisDrug ApprovalDrug DevelopmentHumansMarketingPatient SelectionResearch DesignTreatment Outcome

Identifiers

PMID30610746
PMCPMC6422692

What OpenQuestion holds

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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.