Compassionate use

Compassionate use is a treatment option that allows the use of an unauthorised medicine. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory authorised therapies and who cannot enter clinical trials.

The European Medicines Agency (EMA) provides recommendations through the Committee for Medicinal Products for Human Use (CHMP), but these do not create a legal framework. Compassionate use programmes are coordinated and implemented by Member States, which set their own rules and procedures.

Established by Article 83 of Regulation (EC) No 726/2004, this tool is designed to:

• facilitate and improve access to compassionate use programmes by patients in the EU;
• favour a common approach regarding the conditions of use, the conditions for distribution and the patients targeted for the compassionate use of unauthorised new medicines;
• increase transparency between Member States in terms of treatment availability.

These programmes are only put in place if the medicine is expected to help patients with life-threatening, long-lasting or seriously debilitating illnesses, which cannot be treated satisfactorily with any currently authorised medicine.

The medicine must be undergoing clinical trials or have entered the marketing-authorisation application process and while early studies will generally have been completed, its safety profile and dosage guidelines may not be fully established.

How to request an opinion

National competent authorities can ask EMA for an opinion on how to administer, distribute and use certain medicines for compassionate use. The CHMP also identifies which patients would benefit, and Member States should take note of these recommendations when making decisions.

Manufacturers and marketing-authorisation applicants should not contact EMA to request an opinion, but they may wish to inform the Agency of applications underway at national level. National competent authorities will inform the Agency if they are making a product available to a group of patients for compassionate use.

Comparison to individual basis treatment

Compassionate use should not be confused with 'named-patient basis' treatments, which see doctors obtain medicines directly from manufacturers before authorisation. This is done on an individual basis under the direct responsibility of the doctor, and the Agency does not need to be informed.

In general, medicines that are not yet authorised are first made available through clinical trials and patients should always be considered for inclusion in trials before being offered compassionate use programmes.

Compassionate use recommendations

EMA's recommendations cover how a medicine should be used in compassionate use programmes across the EU, and the type of patient who may benefit from treatment.

EMA does not update its recommendations after a medicine receives marketing authorisation, as all relevant information on the medicine's use is available in its European public assessment report (EPAR). However, compassionate use programmes may continue in certain Member States until the medicine becomes available on the market. 

List of recommendations
 

Name of medicine	Remdesivir Gilead
Active substance	remdesivir
Dosage	100 mg
Pharmaceutical form	Concentrate for solution for infusion Powder for concentrate for solution for infusion
Member State notifying the Agency	Estonia, Romania, The Netherlands and Greece
CHMP opinion documents	Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring addressed to member states for Remdesivir Gilead available for compassionate use Summary on compassionate use for Remdesivir Gilead
Date of opinion	02/04/2020
Date of updated opinion	11/05/2020
Company contact information	Gilead Sciences Ireland UC Carrigtohill County Cork, T45 DP77 Ireland Tel: [phone] Fax: [phone] Email: [e-mail]
Status	Closed
Statement of closure	-
Related content	Veklury: EPAR

 

Name of medicine	Ledipasvir/Sofosbuvir
Active substance	ledipasvir, sofosbuvir
Dosage	90 mg / 400 mg
Pharmaceutical form	Film coated tablet
Member State notifying the Agency	Ireland
CHMP opinion documents	Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring addressed to member states for Ledipasvir/Sofosbuvir available for compassionate use Summary on compassionate use for Ledipasvir/Sofosbuvir
Date of opinion	20/02/2014
Company contact information	Gilead Sciences Limited Granta Park Abington Cambridgeshire CB21 6GT United Kingdom Tel. [phone] Fax [phone] E-mail: [e-mail]
Status	Closed
Statement of closure	-
Related content	Harvoni

 

Name of medicine	Daclatasvir
Active substance	daclatasvir
Dosage	30 and 60 mg
Pharmaceutical form	Film coated tablet
Member State notifying the Agency	Sweden
CHMP opinion documents	Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring addressed to member states for Daclatasvir available for compassionate use Summary on compassionate use for Daclatasvir
Date of opinion	21/11/2013
Company contact information	Bristol-Myers Squibb Pharma EEIG Uxbridge Business Park Sanderson Road Uxbridge UB8 1DH United Kingdom Tel. [phone] Fax [phone] E-mail: [e-mail]
Status	Closed
Statement of closure	-
Related information	Daklinza

 

Name of medicine	Sofosbuvir Gilead
Active substance	sofosbuvir
Dosage	400 mg
Pharmaceutical form	Film-coated tablet
Member State notifying the Agency	Sweden
CHMP opinion documents	Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring addressed to member states for Sofosbuvir Gilead available for compassionate use Summary on compassionate use for Sofosbuvir Gilead
Date of opinion	24/10/2013
Company contact information	Gilead Sciences International Ltd Granta Park, Abington Cambridgeshire CB21 6GT United Kingdom Tel. [phone] Fax [phone] E-mail: [e-mail]
Status	Closed
Statement of closure	-
Related information	Sovaldi

 

Name of medicine	IV Zanamivir
Active substance	Zanamivir
Dosage	10 mg/ml
Pharmaceutical form	Solution for infusion
Member State notifying the Agency	Sweden
CHMP opinion documents	Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring addressed to member states for IV Zanamivir available for compassionate use (Rev. 3) Summary on compassionate use for IV Zanamivir (Rev. 1)
Date of opinion	18/02/2010
Company contact information	GlaxoSmithKline Research & Development Limited 980 Great West Road, Brentford Middlesex, TW8 9GS United Kingdom Telephone: [phone] free phone or [phone] E-Mail: [e-mail]
Status	Closed
Statement of closure	Public statement on IV Zanamivir - Closure of compassionate-use programme in the European Union IV Zanamivir compassionate-use programme - Closure of programme
Related content	EPAR: Dectova

 

Name of medicine	Tamiflu IV
Active substance	Oseltamivir phosphate
Dosage	100 mg
Pharmaceutical form	Powder for solution for infusion
Member State notifying the Agency	Finland
CHMP opinion documents	Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring addressed to Member States for IV Tamiflu available for compassionate use Summary on compassionate use for Tamiflu IV
Date of opinion	20/01/2010
Company contact information	F. Hoffmann-La Roche Ltd. Pharmaceuticals Division PBMV Bldg 74/3O 104 CH-4070, Basel Switzerland Tel. [phone] Fax [phone] E-mail: [e-mail]
Status	Closed
Statement of closure	Public statement on Tamiflu IV - Closure of compassionate-use programme in the European Union Tamiflu IV compassionate-use programme - Closure of programme, EMEA/H/K/002287
Related content	EPAR: Tamiflu