Journal article
Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare Diseases
Chronic obstructive pulmonary diseases, Vol.2(2), pp.177-190
04/28/2015
DOI: 10.15326/jcopdf.2.2.2015.0132
PMCID: PMC5556970
PMID: 28848840
Abstract
Clinical research in rare diseases, including alpha-1 antitrypsin deficiency (AATD), faces challenges not shared by common disease research. These challenges may include the limited number of patient volunteers available for research, lack of natural history studies on which to base many clinical trial interventions, an urgency for the development of drug therapies given the often poor prognosis of rare diseases and uncertainties about appropriate biomarkers and clinical outcomes critical to clinical trial design. To address these challenges and initiate formal discussions among key stakeholders—patients, researchers, industry, federal regulators—the Alpha-1 Foundation hosted the
Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare Diseases
conference February 3-4, 2014 in Bethesda, Maryland. Discussions at the conference led to the conclusions that 1) adaptive designs should be considered for rare disease clinical trials yet more dialogue and study is needed to make these designs feasible for smaller trials and to address current limitations; 2) natural history studies, including the identification of appropriate biomarkers are critically needed and precompetitive collaborations may offer a means of creating these costly studies; and 3) patient registries and databases within the rare disease community need to be more publicly available and integrated, particularly for AATD. This report summarizes the discussions leading to these conclusions.
Details
- Title: Subtitle
- Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare Diseases
- Creators
- Adam Wanner - University of MiamiStephen C Groft - National Institutes of HealthJ. Russell TeagardenJeffrey Krischer - University of South FloridaBarry R Davis - The University of Texas Health Science Center at HoustonChristopher S Coffey - University of IowaDavid H Hickam - Patient-Centered Outcomes Research InstituteJeffrey Teckman - Saint Louis UniversityDavid R Nelson - University of FloridaMichael L McCalebRohit Loomba - University of California, San DiegoCharlie Strange - Medical University of South CarolinaRobert A Sandhaus - National Jewish HealthMark Brantly - University of FloridaJonathan M Edelman - CSLAlbert Farrugia - University of Western Australia
- Resource Type
- Journal article
- Publication Details
- Chronic obstructive pulmonary diseases, Vol.2(2), pp.177-190
- Publisher
- COPD Foundation, Inc
- DOI
- 10.15326/jcopdf.2.2.2015.0132
- PMID
- 28848840
- PMCID
- PMC5556970
- ISSN
- 2372-952X
- eISSN
- 2372-952X
- Language
- English
- Date published
- 04/28/2015
- Academic Unit
- Biostatistics
- Record Identifier
- 9984226815302771
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