Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints

Orphanet Journal of Rare Diseases

Volumn 10, Issue 1, 2015, Pages

  Kakkis, Emil D ^a   O'Donovan, Mary ^b   Cox, Gerald ^c   Hayes, Mark ^d   Goodsaid, Federico ^e   Tandon, P K ^c   Furlong, Pat ^f   Boynton, Susan ^g   Bozic, Mladen ^g   Orfali, May ^h   Thornton, Mark ⁱ  

^a Kakkis EveryLife Foundation for Rare Diseases   (United States)

^b BIOMARIN PHARMACEUTICAL INC   (United States)

^c GENZYME CORPORATION   (United States)

^d Synageva BioPharma Corp   (United States)

^e VERTEX PHARMACEUTICALS   (United States)

^f Parent Project Muscular Dystrophy   (United States)

^g SHIRE   (United States)

^h PFIZER INC   (United States)

ⁱ Sarcoma Foundation of America   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA 1 ANTITRYPSIN; ALPHA 1 ANTITRYPSIN CONCENTRATE; AMMONIA; AUTOANTIBODY; BIOLOGICAL MARKER; DEFERASIROX; ENZYME; GLYCEROL PHENYLBUTYRATE; PHENYLALANINE; PLACEBO; PROTEIN; RNA; SAPROPTERIN;

ACCELERATED APPROVAL; ACQUIRED IMMUNE DEFICIENCY SYNDROME; ADSORPTION DISTRIBUTION METABOLISM AND EXCRETION MODEL; ALPHA 1 ANTITRYPSIN DEFICIENCY; ALPHA DYSTROGLYCAN RELATED MUSCULAR DYSTROPHY; BETA THALASSEMIA; BLOOD TRANSFUSION; CLINICAL ASSESSMENT TOOL; CLINICAL EFFECTIVENESS; COMPARTMENT MODEL; CONCEPTUAL FRAMEWORK; DIET THERAPY; DISEASE COURSE; DISEASE SEVERITY; DOSE RESPONSE; DRUG APPROVAL; DRUG DELIVERY SYSTEM; DRUG DEVELOPMENT TOOL; DRUG LEGISLATION; DRUG MECHANISM; DRUG RESEARCH; DUCHENNE MUSCULAR DYSTROPHY; DYNAMICS; ENDOTHELIUM CELL; FABRY DISEASE; FOOD AND DRUG ADMINISTRATION; GENE EXPRESSION; GENETIC DISORDER; GENETIC MARKER; HEALTH BELIEF; HEALTH CARE ACCESS; HEALTH CARE NEED; HEALTH PROGRAM; HEALTH SERVICE; HUMAN; HURLER SYNDROME; IRON OVERLOAD; MALIGNANT NEOPLASTIC DISEASE; MATHEMATICAL MODEL; MEDICAL INFORMATION; MUSCULAR DYSTROPHY; MYASTHENIA GRAVIS; NONHUMAN; PATHOPHYSIOLOGY; PHENYLKETONURIA; PRACTICE GUIDELINE; PREDICTIVE VALUE; RARE DISEASE; REVIEW; RISK ASSESSMENT; RISK FACTOR; SAFETY; SENSITIVITY ANALYSIS; STANDARD; UNITED STATES; UREA CYCLE DISORDER; DRUG MANUFACTURE; RARE DISEASES;

BIOMARKERS; DRUG APPROVAL; HUMANS; LEGISLATION, DRUG; ORPHAN DRUG PRODUCTION; RARE DISEASES;

EID: 84928751258     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-014-0195-4     Document Type: Review

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