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Table 1 List of all extracted information from EMA and each country matched with the definition of indicators for availability and access

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.