Table 1 EAP: comparison among the major European Countries
EAP | France | Italy | Spain | UK |
|---|---|---|---|---|
General Label | Early access (ex-ATU and ex-PEC-T) + Others | Early access and off-label use | Availability of medicines under special circumstances | EAMS (Early Access to Medicine Schemes) |
Regulatory reference | LOI n° 2020-1576 du 14 décembre 2020 de financement de la sécurité sociale pour 2021 | 648 List: Law 648/96, Law 79/2014, Decreto 2/8/2019 5% Fund: Law 326/03 | Royal Decree 1015/2009 (under review) | No regulatory references so far |
Named/Cohort | Named or cohort depending on the program | 648 List: cohort 5% Fund: named | Named | Both: named/cohort |
Coverage | Before the completion of the P&R process | Before MA | Foreign medicines: after MA Off-label: no MA | Only before MA |
Process | ||||
Applicant | Industry/physicians—hospitals | 648 List: Patients Associations, Scientific Societies, Health Care Organization, Universities, Clinicians 5% Fund: Health Care Organization (specialised centres), Clinicians | Physicians/hospitals | Industry/physicians—hospitals |
Approval | ANSM (safety and efficacy) HAS (other selection criteria) | AIFA (CTS) | AEMPS; Regional authorities (in special occasions for expensive drugs) | MHRA |
Payers of medicines | Hospitals (after receiving an internal permit) But companies could provide medicine for free | 648 List: Regions 5% Fund: AIFA through 5% Fund fed by the pharma industry | Hospitals via Regions/industries (most compassionate use cases) | Industries (for this reason is not an EAP as we intend) |
Selection criteria | ||||
Target diseases | Severe, rare or disabling diseases, no alternatives, | 648 List: Different types of disease 5% Fund: rare diseases and particular/severe diseases | Severe diseases, no alternatives | Severe and disabling disease, high unmet need |
Medicines | New drugs/ indications in development, off-label drugs, foreign medicines | 648 List: No valid alternatives; cheaper than valid alternatives 5% Fund: Orphans drug/drugs in development not approved yet, which represent "a hope of therapy" | Off-label medicines, Foreign medicines | New Drugs, products already marketed in the UK for other indications (off-label), foreign medicines |
Evidence | Safety, efficacy (based on the results of clinical trials) and presumed to be innovative | 648 List: Phase II/Data that may support their use (pure 'off-label') 5% Fund: Clinical report | Safety and efficacy | Safety and efficacy and presumed to be innovative |
Data collection | Yes (well structured). PUT -RD (patient characteristics, medicine usage, efficacy, quality of life (PROMs etc.…), adverse events | 648 List: in principle data on efficacy and safety profile (from the regions to AIFA) Data are not available 5% Fund: No | Yes. Physicians have to collect data on adverse events/ AEMPS has no obbligation to collect data, but may collect them | No evidence on data availability |
Data on economic impact | No | Yes (but only for the 5% Fund) | No | No |
Dedicated Fund | No | Only for 5% Fund | No | No |
MEA | Financial-based agreements (volumes caps/payback if the awarded price < price charged through early access programs) | No, except in 648 List where they are rarely applied | No | No |
Impact on P&R | Data collection influences ASMR’ evaluation and, therefore, the price of the drug | 648 List (revenue from 648 are considered in the P&R negotiation) | No | No |
Fee | No | No | No | Yes |
Limited duration time and, if any, renewal rules | Duration of early access cannot exceed 1 year. It can be renewed with updated product information. If the HAS opinion is negative, the company is obliged to provide patients with treatment for 1 year before stopping supply | No | No | Scientific opinion could be renewed at least 2 months before expiry |