How to address the challenges of evaluating treatment benefits-risks in rare diseases? A convergent mixed methods approach applied within a Merkel cell carcinoma phase 2 clinical trial

Orphanet Journal of Rare Diseases

Volumn 13, Issue 1, 2018, Pages

  Bharmal, Murtuza ^a   Guillemin, Isabelle ^b   Marrel, Alexia ^b   Arnould, Benoit ^b   Lambert, Jérémy ^b   Hennessy, Meliessa ^c   Fofana, Fatoumata ^d  

^a MERCK KGAA   (Germany)

^b MAPI Institute   (France)

^c EMD Serono   (United States)

^d Mapi   (Netherlands)

Author keywords

Merkel cell carcinoma; Mixed methods research; Qualitative outcomes; Quantitative outcomes; Treatment meaningfulness

Indexed keywords

AVELUMAB; MONOCLONAL ANTIBODY;

ADULT; AGED; ARTICLE; CLINICAL ARTICLE; CONTROLLED STUDY; DETERIORATION; DRUG EFFICACY; DRUG SAFETY; FEMALE; HEALTH STATUS; HUMAN; INTERVIEW; LONGITUDINAL STUDY; MALE; MERKEL CELL CARCINOMA; MULTICENTER STUDY; OPEN STUDY; PHASE 2 CLINICAL TRIAL; POPULATION RESEARCH; QUALITATIVE ANALYSIS; QUANTITATIVE ANALYSIS; RISK BENEFIT ANALYSIS; TREATMENT RESPONSE; TREND STUDY; CLINICAL TRIAL; MIDDLE AGED; RARE DISEASE; RISK ASSESSMENT; SKIN TUMOR; VERY ELDERLY;

AGED; AGED, 80 AND OVER; ANTIBODIES, MONOCLONAL; CARCINOMA, MERKEL CELL; FEMALE; HUMANS; LONGITUDINAL STUDIES; MALE; MIDDLE AGED; RARE DISEASES; RISK ASSESSMENT; SKIN NEOPLASMS;

EID: 85048751166     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-018-0835-1     Document Type: Article

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