title: [Cancer: Is it really so different? Particularities of oncologic drugs from the perspective of the pharmaceutical regulatory agency].
abstract: For innovative oncological medicines the centralised procedure at the European Medicines Agency is mandatory for a marketing authorisation application for the European Union. As with other medical drugs, the marketing authorisation decision is based on the assessment of its efficacy, safety and pharmaceutical quality but does not consider price or reimbursement. More sophisticated diagnostic methods drive an increasing stratification of cancer into a multitude of different diseases. Regardless of their different pathogenesis and therapeutic options the most relevant clinical endpoints remain cure, overall survival and progression free survival. These endpoints include both efficacy and safety, as patient survival reflects the sum of the beneficial anti-tumour effects (increasing survival) AND the adverse effects (decreasing survival). The benefit of an anticancer medicine should be evident from both overall survival and progression free survival (e.g. used as primary and secondary endpoints). Mature data on overall survival may not be needed for marketing authorisation if a clear increase in progression free survival convincingly predicts a beneficial effect on overall survival. In these exceptional cases treatment of patients with an obviously beneficial medicine must not be delayed - possibly for years - until the exact size of the benefit has been established. The continued stratification of the disease cancer results in a lower prevalence for each of the newly distinguished disease entities and an ever increasing number of orphan designations for medicines for rare diseases. Incentives for the development of orphan medicines include market exclusivity for up to ten years. In specific circumstances, however, the orphan legislation may restrict the authorisation and marketing of competing generic products even beyond these ten years. Conditional approval and approval under exceptional circumstances may accelerate patients' access to a new medicine. Both postulate that the extent of the benefit cannot be determined with sufficient certainty at the time of marketing authorisation. This uncertainty may have a negative impact on price and reimbursement as these decisions may consider data or assessments from the marketing authorisation procedure. Therefore, marketing authorisation applications and subsequent pricing and reimbursement negotiations should not be regarded as completely independent processes, but be included in an overall strategy for the development of oncologic drugs. (As supplied by publisher).
authors.affiliation: Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn. harald.enzmann@bfarm.de
authors.forename: Harald
authors.initials: H
authors.lastname: Enzmann
authors.forename: Karl
authors.initials: K
authors.lastname: Broich
mesh_terms: D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D018572:Disease-Free Survival; D017277:Drug Approval / Q000331:legislation & jurisprudence*; D015507:Drugs, Investigational / Q000009:adverse effects / Q000627:therapeutic use*; D023381:Endpoint Determination; D005062:European Union; D005858:Germany; D006801:Humans; D040541:Marketing / Q000331:legislation & jurisprudence; D009313:National Health Programs / Q000331:legislation & jurisprudence*; D009369:Neoplasms / Q000188:drug therapy* / Q000401:mortality; D009965:Orphan Drug Production
chemical_list: D000970:Antineoplastic Agents; D015507:Drugs, Investigational