Main characteristics of CUPs in different EU Member States

Timing

 

Typically, Compassionate Use Programmes (CUPs) are initiated late in the developmental process of medicinal products, to ‘bridge’ the time between the end of phase III development, regulatory approval and product launch (which may take 1–2 years). However a CUP can start earlier. There are cases of CUP starting at the end of phase I trials.

Cost: for free, or not for free?
 

Some CUPs are free and some have costs. In some countries, the company is prohibited from charging for a medicinal product in a compassionate use setting (on the contrary, the company needs to pay for running the programme, for obtaining approval from ethics committees, etc,. and consequently decides not to propose a CUP). In other countries, it is left at the discretion of the company whether or not to charge.

Often, when the company does not charge, there can be criticism that the CUP is an attempt to conquer a large market share before obtaining a marketing authorisation, and the company can be accused of using compassionate use as a marketing tactic. On the other hand, when a company does charge,  it is criticised for putting a price on compassion.

Duration
 

Typically a CUP is authorised for one year and can be renewed. But a marketing authorisation application needs to be prepared and submitted sooner or later, as a CUP cannot last for ever. If regulatory authorities are not provided adequate information on the clinical trials and intention to submit a marketing authorisation application, they may reject the demand for a CUP.

Scope
 

To summarise, medicinal products eligible for a CUP aim at treating, preventing or diagnosing a serious or rare disease when there is an unmet medical need (no therapeutic alternative) for some or all patients.

Efficacy Data and claims on benefit
 

There is no consensus on efficacy/benefits data as a prerequisite to authorise a compassionate use. As a CUP is run in parallel to clinical trials, and in particular phase III trials (which aim at demonstrating efficacy), not all data are available when deciding on a CUP. Therefore benefit for the group of patients needs to be plausible, based on earlier trials.

Consent form
 

When receiving a treatment via compassionate use, the patient often needs to read and sign a consent form that explains what is known and not known about the medicine, and which highlights the need to report any responses, both good or bad, that occur when taking the medicine. Responsibility for the prescription remains the healthcare professional’s responsibility.

Type of CUP, procedure and review times for 7 Member States

 

Country

CUP type

Regulatory process

Review times

France

Cohort or named patient

Well defined

Named patient: 24-48h
Cohort: 2-4 months

Denmark

Cohort or named patient

Well defined

Within 3-4 weeks

Germany

Cohort or named patient

Under change

2-3 months

Italy

Cohort or named patient

Well defined but complex

Named patient: 1 month
Cohort: not defined

The Netherlands

Cohort or named patient

Well defined

Named patient: 4 weeks
Cohort: not defined

Spain

Cohort or named patient

Need local expertise

Very variable, case by case

United Kingdom

Named patient

Well defined

4 weeks after receipt of notification of intent to import

Adapted from Helene Sou, Pharm Med 2010; 24(4):1-7

How to import, who pays, legal basis for 7 Member States
 

Country

Import authorisation

Payer

Legal basis

France

Nominative ATU authorisation taken as import permit
Cohort ATU requires import permit

Hospital or national insurance. 100% covered

Code de la Santé Publique (Art. L.5121-12 and R.5121-68 to 76)

Denmark

Imported by pharmacy

Hospital/patient co-payment. Reimbursement as for approved products

Lov om laegemidler no. 1180 as amended 17 June 2008

Germany

Import permit required

Company

14 and 15 ArzneimittelGesetz Novelle

Italy

Ethics committee written approval required

Hospital or national insurance

Decreto 8 maggio 2003; Legge 648/1996

The Netherlands

Import permit required

Patient/company

 Usually not reimbursed

Art. 40.3c Geneesmiddelenwet van 25 Juni 2007. Circular 2002-06-IGZ

Spain

Need to apply for importation to the national agency

Company

Real Decreto 1015/2009; Ley 29/2006 (Art. 24) and Real Decreto 223/2004 (Art. 28)

United Kingdom

Import permit required

Reimbursement case by case, price can be set freely

SI 1994 no. 3144

Adapted from Helene Sou, Pharm Med 2010; 24(4):1-7

Page created: 30/10/2013
Page last updated: 07/11/2014
 
 
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