Challenges of developing and conducting clinical trials in rare disorders

American Journal of Medical Genetics, Part A

Volumn 176, Issue 4, 2018, Pages 773-783

  Kempf, Lucas ^a   Goldsmith, Jonathan C ^a   Temple, Robert ^b  

^a Rare Diseases Program   (United States)

^b CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

Food and Drug Administration (FDA); orphan drugs; rare disease; rare disorders; regulation; trial design

Indexed keywords

BIOLOGICAL MARKER; ORPHAN DRUG;

DRUG APPROVAL; ELECTRONIC MEDICAL RECORD; FOOD AND DRUG ADMINISTRATION; GOOD MANUFACTURING PRACTICE; HUMAN; NONHUMAN; PATIENT ADVOCACY; PATIENT PARTICIPATION; POSTMARKETING SURVEILLANCE; PRECLINICAL STUDY; PRIORITY JOURNAL; QUALITY CONTROL; RARE DISEASE; REVIEW; STUDY DESIGN; ANIMAL; CLINICAL TRIAL (TOPIC); DRUG DEVELOPMENT; LEGISLATION AND JURISPRUDENCE; METHODOLOGY; PATIENT SELECTION; PROCEDURES;

ANIMALS; BIOMARKERS; CLINICAL TRIALS AS TOPIC; DRUG DEVELOPMENT; DRUG EVALUATION, PRECLINICAL; HUMANS; PATIENT SELECTION; PRODUCT SURVEILLANCE, POSTMARKETING; RARE DISEASES; RESEARCH DESIGN;

EID: 85029839425     PISSN: 15524825     EISSN: 15524833     Source Type: Journal    
DOI: 10.1002/ajmg.a.38413     Document Type: Review

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