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New strategies for the conduct of clinical trials in paediatric Pulmonary Arterial Hypertension (PAH): Outcome of a multi-stakeholder meeting with patients, academia, industry and regulators held at EMA on Monday 12th June 2017
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- Olivier, Cecille (author)
- European Medicines Agency (EMA)
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- Sun, Haihao (author)
- United States Food and Drug Administration
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- Amchin, Wayne (author)
- United States Food and Drug Administration
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- Beghetti, Maurice (author)
- Geneva University Hospital
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- Berger, Rolf (author)
- University of Groningen
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- Breitenstein, Stefanie (author)
- Bayer Pharma AG
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- Garnett, Christine (author)
- United States Food and Drug Administration
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- Gullberg, Ninna (author)
- European Medicines Agency (EMA)
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- Hassel, Patrik (author)
- PH Sweden
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- Ivy, Dumbar (author)
- University of Colorado School of Medicine
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- Kawut, Steven (author)
- University of Pennsylvania
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- Klein, Agnes (author)
- Public Health Agency of Canada
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- Lesage, Catherine (author)
- Actelion Pharmaceuticals Ltd.
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- Migdal, Marek (author)
- Children’s Memorial Health Institute
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- Nije, Barbara (author)
- Public Health Agency of Canada
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- Odermarsky, Michal (author)
- Lund University,Lunds universitet,Pediatrik, Lund,Sektion V,Institutionen för kliniska vetenskaper, Lund,Medicinska fakulteten,Barnkardiologi,Forskargrupper vid Lunds universitet,Paediatrics (Lund),Section V,Department of Clinical Sciences, Lund,Faculty of Medicine,Children cardiology,Lund University Research Groups,Skåne University Hospital
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- Strait, James (author)
- Merck Sharp & Dohme, Switzerland
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- de Graeff, Pieter (author)
- Medicines Evaluation Board, Utrecht
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- Stockbridge, Norman (author)
- United States Food and Drug Administration
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(creator_code:org_t)
- 2019
- 2019
- English.
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In: Journal of the American Heart Association. - 2047-9980. ; 8:10, s. 1-10
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http://dx.doi.org/10... (free)
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Abstract
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- Aims: Drug development for paediatric pulmonary arterial hypertension (PAH) ispressingly needed. Experts from the US Food and Drug Administration, EuropeanMedicines Agency, Health Canada, key opinion leaders, academia, patients, and industry representatives held a workshop on 12th June 2017 dedicated to addressing challenges and unmet needs. This report summarises the approaches proposed during the meeting to address key issues in extrapolation, trial design, and study endpoints in pediatric drug development.Methods and Results: A pre-workshop stakeholder survey was conducted and showed that most respondents believe the pathophysiology of heritable PAH and some forms of idiopathic PAH is thought to be sufficiently similar in adult and paediatric patients, although the clinical manifestations may differ. In this situation, placebo-controlled trials might not be required to confirm clinical benefit in paediatrics. The study endpoints used to support drug approvals in adults were reviewed to determine if these existing study endpoints can be applied in paediatric PAH efficacy trials. It showed that non-invasive study endpoints, such as the time to clinical worsening, WHO functionalclass, and 6-Minute-Walk-Test could be applicable in paediatric PAH trials, although each presents some limitations in paediatrics.Conclusion: Extrapolation of efficacy from informative adult studies may be appropriate in some forms of PAH. Initial dose-finding studies and exposure-response modelling are warranted in paediatric PAH, followed by an efficacy and safety study to explore the response to treatment and exposure-response relationship. A novel, non-invasive, developmentally-appropriate, and reliable study endpoint needs to be developed.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Kardiologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Cardiac and Cardiovascular Systems (hsv//eng)
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Farmaceutiska vetenskaper (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Pharmaceutical Sciences (hsv//eng)
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Olivier, Cecille
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Sun, Haihao
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Amchin, Wayne
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Beghetti, Mauric ...
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Berger, Rolf
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Breitenstein, St ...
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Garnett, Christi ...
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Gullberg, Ninna
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Hassel, Patrik
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Ivy, Dumbar
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Kawut, Steven
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Klein, Agnes
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Lesage, Catherin ...
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Migdal, Marek
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Nije, Barbara
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Odermarsky, Mich ...
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Strait, James
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de Graeff, Piete ...
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Stockbridge, Nor ...
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MEDICAL AND HEAL ...
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and Basic Medicine
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Lund University