Perspectives for improving the evaluation and access of therapies for rare lung diseases in Europe

Respiratory Medicine

Volumn 106, Issue 6, 2012, Pages 759-768

  Luisetti, Maurizio ^a   Balfour Lynn, Ian M ^b   Johnson, Simon R ^c,d   Miravitlles, Marc ^e   Strange, Charlie ^f   Trapnell, Bruce C ^g   Van Bronswijk, Hans ^h   Vogelmeier, Claus ⁱ  

^a UNIVERSITY OF PAVIA   (Italy)

^b ROYAL BROMPTON AND HAREFIELD NHS FOUNDATION TRUST   (United Kingdom)

^c UNIVERSITY OF NOTTINGHAM   (United Kingdom)

^d National Centre for Lymphangioleiomyomatosis   (United Kingdom)

^e HOSPITAL CLÍNIC   (Spain)

^f MEDICAL UNIVERSITY OF SOUTH CAROLINA   (United States)

^g CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^h PAREXEL INTERNATIONAL   (United States)

ⁱ PHILIPPS UNIVERSITY MARBURG   (Germany)

Author keywords

Clinical trial design; Licensing and reimbursement; Orphan drug; Outcome measure biomarker; Rare lung disease; Registry

Indexed keywords

INTERLEUKIN 5 ANTIBODY; ORPHAN DRUG; RAPAMYCIN; ROFLUMILAST; VASCULOTROPIN D;

ALPHA 1 ANTITRYPSIN DEFICIENCY; ASTHMA; CHRONIC OBSTRUCTIVE LUNG DISEASE; COMPUTER ASSISTED TOMOGRAPHY; CYSTIC FIBROSIS; DISEASE COURSE; DNA POLYMORPHISM; DRUG BIOAVAILABILITY; DRUG LEGISLATION; EUROPE; EVALUATION; FORCED EXPIRATORY VOLUME; GENE MUTATION; HEALTH CARE ACCESS; HEALTH CARE QUALITY; HUMAN; INTERPERSONAL COMMUNICATION; LICENSING; LUNG ALVEOLUS PROTEINOSIS; LYMPHANGIOLEIOMYOMATOSIS; OUTCOME ASSESSMENT; PATIENT ADVOCACY; PHENOTYPE; PRIORITY JOURNAL; QUALITY CONTROL; RANDOMIZED CONTROLLED TRIAL (TOPIC); RARE DISEASE; REGISTER; REIMBURSEMENT; REVIEW; SMOKING; WORKSHOP;

EUROPE; HEALTH SERVICES ACCESSIBILITY; HUMANS; LEGISLATION, DRUG; LUNG DISEASES; ORPHAN DRUG PRODUCTION; RARE DISEASES; REGISTRIES; RESEARCH DESIGN;

EID: 84859790922     PISSN: 09546111     EISSN: 15323064     Source Type: Journal    
DOI: 10.1016/j.rmed.2012.02.016     Document Type: Review

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