Development and Use of New Therapeutics for Rare Diseases: Views from Patients, Caregivers, and Advocates

Patient

Volumn 8, Issue 1, 2015, Pages 75-84

  Kesselheim, Aaron S ^a   McGraw, Sarah ^b   Thompson, Lauren ^a   O’Keefe, Kelly ^a   Gagne, Joshua J ^a  

^a BRIGHAM AND WOMEN S HOSPITAL   (United States)

^b HASTINGS CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

NEW DRUG; ORPHAN DRUG; PLACEBO;

ARTICLE; CAREGIVER; CLINICAL ARTICLE; CODING; CONTROLLED STUDY; DATA ANALYSIS SOFTWARE; DECISION MAKING; DRUG DESIGN; DRUG EFFICACY; DRUG INDUSTRY; DRUG SCREENING; FOOD AND DRUG ADMINISTRATION; GENETIC DISORDER; HEALTH CARE SYSTEM; HEMOPHILIA; HUMAN; INFORMATION PROCESSING; LEARNING; MULTICENTER STUDY; OFF LABEL DRUG USE; OUTCOME ASSESSMENT; PATIENT ADVOCACY; PATIENT CARE; PHELAN MCDERMID SYNDROME; PRIORITY JOURNAL; PULMONARY HYPERTENSION; QUALITATIVE ANALYSIS; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL; RARE DISEASE; TUBEROUS SCLEROSIS; UNITED STATES; ATTITUDE TO HEALTH; DRUG DEVELOPMENT; FEMALE; MALE; PSYCHOLOGY; RARE DISEASES;

CAREGIVERS; DRUG DISCOVERY; FEMALE; FOCUS GROUPS; HEALTH KNOWLEDGE, ATTITUDES, PRACTICE; HUMANS; MALE; PATIENT ADVOCACY; QUALITY OF LIFE; RARE DISEASES; UNITED STATES;

EID: 84925496850     PISSN: 11781653     EISSN: 11781661     Source Type: Journal    
DOI: 10.1007/s40271-014-0096-6     Document Type: Article

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