Compassionate use

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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.

Agency Role

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.

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.

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.

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 opinions

The list of compassionate use opinions includes the CHMP’s recommendations on how a medicine should be used, and the type of patient who should be eligible.

Name of medicineLedipasvir/Sofosbuvir
Active substanceledipasvir, sofosbuvir
Dosage90 mg / 400 mg
Pharmaceutical formFilm coated tablet
Member State notifying the AgencyIreland
CHMP opinion documents

Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring

Summary on compassionate use

Date of opinion20/02/2014
Company contact informationGilead Sciences Limited
Granta Park
Abington
Cambridgeshire
CB21 6GT
United Kingdom
Tel. +44 (0)208 5872206
Fax +44 (0)1223 897233
E-mail: eamemed.info@gilead.com
StatusOngoing
Related documents -

 

Name of medicineDaclatasvir
Active substancedaclatasvir
Dosage30 and 60 mg
Pharmaceutical formFilm coated tablet
Member State notifying the AgencySweden
CHMP opinion documents

Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring

Summary on compassionate use

Date of opinion21/11/2013
Company contact informationBristol-Myers Squibb Pharma EEIG
Uxbridge Business Park
Sanderson Road
Uxbridge UB8 1DH
United Kingdom
Tel. +44 (0)1895 523 740
Fax +44 (0)1895 523 677
E-mail: medical.information@bms.com
StatusOngoing
Related documents -

 

Name of medicineSofosbuvir Gilead
Active substanceSofosbuvir
Dosage400 mg
Pharmaceutical formFilm-coated tablet
Member State notifying the AgencySweden
CHMP opinion documentsConditions of use, conditions for distribution and patients targeted and conditions for safety monitoring
Summary on compassionate use
Date of opinion24/10/2013
Company contact informationGilead Sciences International Ltd
Granta Park, Abington
Cambridgeshire CB21 6GT
United Kingdom
Tel. +44 (0)1223 897496
Fax +44 (0)1223 897233
E-mail: eamemed.info@gilead.com
StatusOngoing
Related documents -

 

Name of medicineIV Zanamivir
Active substanceZanamivir
Dosage10 mg/ml
Pharmaceutical formSolution for infusion
Member State notifying the AgencySweden
CHMP opinion documentsConditions of use, conditions for distribution and patients targeted and conditions for safety monitoring
Summary on compassionate use
Date of opinion18/02/2010
Company contact information

GlaxoSmithKline Research & Development Limited
980 Great West Road, Brentford 
Middlesex, TW8 9GS

United Kingdom
Telephone: 00800 2468 3579 free phone 
or +44(0)2089904855
E-Mail:
GSKClinicalSupportHD@gsk.com

StatusOngoing
Related documents -

 

Name of productTamiflu IV
Active substanceOseltamivir phosphate
Dosage100 mg
Pharmaceutical formPowder for solution for infusion
Member State notifying the AgencyFinland
CHMP opinion documentsConditions of use, conditions for distribution and patients targeted and conditions for safety monitoring
Summary on compassionate use
Date of opinion20/01/2010
Company contact informationF. Hoffmann-La Roche Ltd.
Pharmaceuticals Division
PBMV Bldg 74/3O 104
CH-4070, Basel
Switzerland
Tel. +41 61 688 5522
Fax +41 61 687 2239
E-mail: basel.tamifluquestions@roche.com
StatusClosed
Related documentsPublic statement on Tamiflu IV: Closure of compassionate-use programme in the EU
Tamiflu IV compassionate-use programme EMEA/H/K/002287 - Closure of programme

 

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