Review and comparison of clinical evidence submitted to support European Medicines Agency market authorization of orphan-designated oncological treatments

Orphanet Journal of Rare Diseases

Volumn 10, Issue 1, 2015, Pages

  Winstone, Julie ^a   Chadda, Shkun ^a   Ralston, Stephen ^a   Sajosi, Peter ^b  

^a SIRIUS Market Access ^*  (United Kingdom)

^b Takeda Pharmaceuticals International GmbH   (Switzerland)

Author keywords

Clinical trials; European Medicines Agency; Mifamurtide; Orphan disorders; Orphan treatments; Osteosarcoma

Indexed keywords

AMINOLEVULINIC ACID; ANTINEOPLASTIC AGENT; AZACITIDINE; DECITABINE; HISTAMINE; IMATINIB; IMMUNOMODULATING AGENT; LENALIDOMIDE; MIFAMURTIDE; NILOTINIB; PIRFENIDONE; POMALIDOMIDE; RUXOLITINIB; SORAFENIB; TEMSIROLIMUS; THALIDOMIDE; TRABECTEDIN;

ACUTE MYELOBLASTIC LEUKEMIA; ARTICLE; CANCER THERAPY; CHRONIC DISEASE; CHRONIC MYELOID LEUKEMIA; DRUG APPROVAL; DRUG MARKETING; EUROPEAN MEDICINES AGENCY; FIBROSING ALVEOLITIS; FOLLOW UP; GLIOBLASTOMA; HUMAN; KIDNEY CARCINOMA; LIFE THREAT; LIVER CELL CARCINOMA; MANTLE CELL LYMPHOMA; MULTIPLE MYELOMA; MYELODYSPLASTIC SYNDROME; MYELOID METAPLASIA; OSTEOSARCOMA; OVERALL SURVIVAL; PHASE 3 CLINICAL TRIAL (TOPIC); POPULATION SIZE; PREVALENCE; RANDOMIZED CONTROLLED TRIAL (TOPIC); RARE DISEASE; SOFT TISSUE SARCOMA; SPLENOMEGALY; TREATMENT DURATION; ANIMAL; CLINICAL TRIAL (TOPIC); COMPARATIVE STUDY; DRUG MANUFACTURE; ECONOMICS; EUROPE; LEGISLATION AND JURISPRUDENCE; RARE DISEASES;

ANIMALS; ANTINEOPLASTIC AGENTS; CLINICAL TRIALS AS TOPIC; DRUG APPROVAL; EUROPE; HUMANS; ORPHAN DRUG PRODUCTION; RARE DISEASES;

EID: 84945303100     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-015-0349-z     Document Type: Article

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