From: Comparing access to orphan medicinal products in Europe
Country | Indicators | Data sources (listed in Appendix) | Indicator definition |
---|---|---|---|
EC | Central marketing authorisation | European Medicines Agency | • Benchmark of potentially available OMPs. • Central marketing authorisation date used to define time to access indicator in each country. |
France | Early access schemes | ANSM – Autorisations temporaires d’utilisation | Reported number of OMPs to complement indicator of availability. |
Launch | Base de données publique des medicaments | Availability = Number of OMPs. | |
HTA | Base de données publique des medicaments HAS | Reported number of OMPs with HTA evaluation and decision to complement indicator of access. | |
Reimbursement | Base de données publique des medicaments Base Vidal HAS | • Access = Number of OMPs reimbursed. • Time to access = reimbursement date (normally after HTA publication date, except for some pre-license OMPs) - central authorisation date. | |
Germany | National registry/authorisation | Lauer-Taxe database | • Availability = Number of OMPs. • Access = Number of OMPs. • Time to access = 0 |
HTA | IQWiG G-BA | Reported to complement and interpret P&R policies. | |
Italy | Early access schemes | AIFA list of drugs | Reported and included in availability indicator. |
National registry/authorisation | AIFA list of drugs (includes pre-licensed OMPs) | Availability = Number of OMPs. | |
Reimbursement | Manual consultación of the “Gazzetta Ufficiale” and other relevant laws such as Law 648/1996 | • Access = Number of OMPs reimbursed. • Time to access = reimbursement date (publication Gazetta Ufficiale) – central authorisation date. | |
Spain | National registry/authorisation | Consejo General de Colegios Oficiales de Farmacéuticos | Availability = Number of OMPs. |
Reimbursement | Consejo General de Colegios Oficiales de Farmacéuticos | • Access = Number of OMPs. • Time to access = reimbursement date (date enter in reimbursement registry) – central authorisation date. | |
England | HTA Other routine funding | NICE (Technology Appraisals and Highly Specialised Technologies) NHS England (reimbursement date not available) CDF (reimbursement date not available) | • Availability = Number of OMPs (publication of NICE guidance or other routine funding list). • Access = Number of OMPs (positive HTA decision (including treatment recommended for use, optimised), or commissioning policy in other routine funding list). • Time to access = HTA publication date – central authorisation date. |
Scotland | HTA | SMC | • Availability = Number of OMPs with HTA evaluation. • Access = Number of OMPs with positive HTA decision (including treatment recommended for use, optimised). • Time to access = HTA publication date – central authorisation date. |
Wales | HTA | NICE AWMSG | • Availability = Number of OMPs with HTA evaluation. • Access = Number of OMPs with positive HTA decision. • Time to access = HTA publication date – central authorisation date. |