According to data from recent studies from Europe, a large percentage of patients have restricted access to innovative medicines for metastatic melanoma. Melanoma World Society and European Association of Dermato-oncology conducted a Web-based survey on access to first-line recommended treatments for metastatic melanoma by current guidelines (National Comprehensive Center Network, European Society for Medical Oncology [ESMO] and European Organization for Research and Treatment of Cancer/European Association of Dermato-oncology/European dermatology Forum) among melanoma experts from 27 European countries, USA, China, Australia, Argentina, Brazil, Chile and Mexico from September 1st, 2017 to July 1st, 2018. Data on licencing and reimbursement of medicines and the number of patient treated were correlated with the data on health expenditure per capita (HEPC), Mackenbach score of health policy performance, health technology assessment (HTA), ASCO and ESMO Magnitude of clinical benefit scale (ESMO MCBS) scores of clinical benefit and market price of medicines. Regression analysis for evaluation of correlation between the parameters was carried out using SPSS software. The estimated number of patients without access in surveyed countries was 13768. The recommended BRAFi + MEKi combination and anti-PD1 immunotherapy were fully reimbursed/covered in 19 of 34 (55.8%) and 17 of 34 (50%) countries, and combination anti-CTLA4+anti-PD1 in was fully covered in 6 of 34 (17.6%) countries. Median delay in reimbursement was 991 days, and it was in significant correlation with ESMO MCBS (p = 0.02), median market price (p = 0.001), HEPC and Mackenbach scores (p < 0.01). Price negotiations or managed entry agreements (MEAs) with national authorities were necessary for reimbursement. In conclusion, great discrepancy exists in metastatic melanoma treatment globally. Access to innovative medicines is in correlation with economic parameters as well as with healthcare system performance parameters. Patient-oriented drug development, market access and reimbursement pathways must be urgently found.
- MeSH
- compassionate use trials MeSH
- dávkové mechanismy MeSH
- dodržování směrnic MeSH
- hrubý domácí produkt MeSH
- klinické zkoušky jako téma statistika a číselné údaje MeSH
- koupě zacílená na zvýšení kvality a hodnoty péče MeSH
- léky zkušební ekonomika zásobování a distribuce terapeutické užití MeSH
- lidé MeSH
- melanom farmakoterapie ekonomika epidemiologie sekundární MeSH
- náklady na léky MeSH
- předpisy - poplatky MeSH
- průzkumy a dotazníky MeSH
- směrnice pro lékařskou praxi jako téma MeSH
- socioekonomické faktory MeSH
- vývoj člověka MeSH
- zdravotní priority MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
- Geografické názvy
- Evropa MeSH
- Latinská Amerika MeSH
- Rusko MeSH
BACKGROUND: Patients with advanced renal cell carcinoma in routine clinical practice can differ considerably from those in phase III studies. PATIENTS AND METHODS: PREDICT (Patient characteristics in REnal cell carcinoma and Daily practICe Treatment with sorafenib) was a prospective, noninterventional study of open-label sorafenib for the treatment of advanced RCC conducted in 18 countries. Patient characteristics, therapy duration, tumor status, and tolerability were assessed at baseline and during routine follow-up. RESULTS: Overall, 2599 patients were evaluable for safety and 2311 for efficacy. The diverse population included patients with brain metastases (5%), non-clear-cell histologies (17%), high Memorial Sloan-Kettering Cancer Center risk score (11%), poor Eastern Cooperative Oncology Group performance status (PS ≥ 2, 29%), and patients with no previous nephrectomy (16%) or no previous systemic therapy (37%). The median duration of sorafenib therapy was 7.3 months and was similar in clinically relevant subgroups (eg, patients with PS 2, brain metastases, or concomitant hypertension or diabetes [range, 6.7-7.0 months]). The median duration of therapy was shorter for patients with PS 3 or non-clear-cell histologies (4.6 and 4.8 months, respectively). The most common drug-related adverse events were hand-foot skin reaction (20%), diarrhea (17%), and rash (8%). CONCLUSION: Sorafenib was generally well tolerated and provided clinical benefit in a large, diverse population of patients with advanced RCC treated in routine clinical practice.
- MeSH
- ambulantní zařízení MeSH
- antitumorózní látky aplikace a dávkování škodlivé účinky terapeutické užití MeSH
- dospělí MeSH
- fenylmočovinové sloučeniny aplikace a dávkování škodlivé účinky terapeutické užití MeSH
- karcinom z renálních buněk farmakoterapie etnologie patologie MeSH
- lidé středního věku MeSH
- lidé MeSH
- metastázy nádorů MeSH
- mladiství MeSH
- mladý dospělý MeSH
- nádory ledvin farmakoterapie etnologie patologie MeSH
- niacinamid aplikace a dávkování škodlivé účinky analogy a deriváty terapeutické užití MeSH
- prospektivní studie MeSH
- senioři nad 80 let MeSH
- senioři MeSH
- výsledek terapie MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- mladý dospělý MeSH
- mužské pohlaví MeSH
- senioři nad 80 let MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
- práce podpořená grantem MeSH
- MeSH
- fenylalanin farmakologie MeSH
- lidé MeSH
- srdeční arytmie etiologie farmakoterapie MeSH
- syndrom dlouhého QT patofyziologie MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- kongresy MeSH
