title: [HTA-Perspective: Challenges in the early assessment of new oncological drugs].
abstract: Oncologic drug therapies have gained wide attention in the context of health policy priority setting for serious and socially significant diseases with high human and monetary costs. Due to uncertainties and scepticism about the actual therapeutic importance of newly approved oncology products, an early assessment programme was already established in Austria in 2007. The assessment of new oncology products is thereby faced with special challenges, since study populations are frequently not representative or the study design is laid out in such a manner that a definitive assessment of patient-relevant endpoints is not possible (cross-overs after interim assessments, surrogate parameters as primary endpoints, uncontrolled studies or those with unrealistic comparators, invalidated post-hoc identified biomarkers). On account of these major uncertainties, even the European Medicines Agency (EMA) is already contemplating multi-stage, "adaptive" approvals, and national reimbursement institutions are increasingly working with outcome-oriented, conditional reimbursement. (As supplied by publisher).
authors.affiliation: Ludwig Boltzmann Institute for Health Technology Assessment, Vienna, Austria. claudia.wild@hta.lbg.ac.at
authors.forename: Claudia
authors.initials: C
authors.lastname: Wild
authors.forename: Anna
authors.initials: A
authors.lastname: Nachtnebel
mesh_terms: D001317:Austria; D002986:Clinical Trials as Topic / Q000191:economics / Q000331:legislation & jurisprudence; D003362:Cost-Benefit Analysis / Q000191:economics / Q000331:legislation & jurisprudence; D018572:Disease-Free Survival; D017277:Drug Approval / Q000191:economics / Q000331:legislation & jurisprudence*; D015507:Drugs, Investigational / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D023381:Endpoint Determination / Q000191:economics; D005062:European Union; D019317:Evidence-Based Medicine / Q000191:economics / Q000331:legislation & jurisprudence; D006292:Health Priorities / Q000191:economics / Q000331:legislation & jurisprudence; D006801:Humans; D009313:National Health Programs / Q000191:economics / Q000331:legislation & jurisprudence*; D009369:Neoplasms / Q000188:drug therapy* / Q000191:economics / Q000401:mortality; D012051:Reimbursement Mechanisms / Q000191:economics / Q000331:legislation & jurisprudence
chemical_list: D015507:Drugs, Investigational