Medicinal products available for compassionate use are medicinal products that have entered the marketing authorisation application process at the European Medicines Agency (EMA) or are undergoing clinical research and are available for compassionate use to patient groups with chronic or seriously debilitating disease or life-threatening disease that cannot be satisfactorily treated by authorised medicinal products. Medicinal products for compassionate use may be distributed only free of charge. Compassionate use of medicinal products is intended to facilitate the availability to patients of new treatment options under development, for example, to treat HIV/AIDS, autoimmune disease, etc. Medicinal products for compassionate use must fall into at least one of the categories laid down in Article 3(1) and 3(2) of the Regulation (EC) No. 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency:
1) Medicinal product for human use containing a new active substance that has not been authorised in the Community until 20 May 2004 and for which the therapeutic indication is treatment of any of the following diseases:
- Autoimmune diseases and other disfunctions of the immune system
- Neurodegenerative disorders
- Acquired immune deficiency syndrome (HIV/AIDS)
- Cancer and oncological diseases
- Viral diseases
2) Medicinal products that are designated as orphan medicinal products pursuant to Regulation (EC) No 141/2000 for treatment of rare diseases for which there are no satisfactory diagnostic, preventative and treatment methods approved within the Community or, if such exist, the medicinal product may provide substantial benefit to the people affected by this disease:
- affecting not more than five in 10 thousand persons in the Community
- due to economic reasons it is not possible to release the medicinal product on to the market via any other procedure
3) Medicinal products developed by means of one of the following biotechnological processes:
- Recombinant DNA technology,
- Controlled expression of genes coding for biologically active proteins in prokaryotes and eukaryotes including transformed mammalian cells,
- Hybridoma and monoclonal antibody methods.
4) Medicinal products for veterinary use intended primarily for use as performance enhancers in order to promote the growth of treated animals or to increase yields from treated animals.
Normative acts lay down the procedure how a marketing authorisation applicant or clinical trial sponsor, or an authorised person may request and how a healthcare institution may receive permit for compassionate use of a medicinal product to treat patients. Prior to distribution of medicinal products for compassionate use, a healthcare institution in Latvia must receive an appropriate permit issued by the State Agency of Medicines, i.e., permit for distribution of individually supplied unauthorised medicinal products (Annex 6 of the Cabinet of Ministers Regulation No. 416 of 26 June 2007 “Procedures Regarding Distribution and Quality Control of Medicinal Products” (hereinafter – Regulation No. 416)”, issued to the healthcare institution that submits a substantiated justification for the use of such a medicinal product.
Please note that if the therapeutic indication of an unauthorised medicinal product is different than the one indicated in the summary of product characteristics it is not considered as medicinal product for compassionate use (such use of medicinal product is considered as “off-label use”).
In order to receive permit for distribution of medicinal product for compassionate use, an application must be submitted to SAM to receive permit for distribution of an individually supplied unauthorised medicinal product (Annex 7 of regulation No. 416) by the person who intends to authorise the medicinal product in the centralised authorisation procedure in accordance with Regulation (EC) No. 726/2004 and who has submitted the marketing authorisation application to EMA or whose name is on the clinical trial authorisation.
Documents to be attached to the application
1. Statement from the authorisation applicant that a marketing authorisation application for the medicinal product has been submitted to SAM or that the medicinal product is undergoing a clinical trial.
2. Program for medicinal product use prepared by the authorisation applicant. The program must include all of the information relevant to a healthcare professional. The description of said program must include information regarding the relevant medicinal product and its use:
- Draft summary of product characteristics, label and package leaflet in the state language
- Information for patient, including sample informed consent form in accordance with the Law on the Right of Patients
- Description of patient group, including the estimated number of patients in Latvia, information on whether the relevant medicinal product is being used in other countries, estimated duration of use and number of packagings
- Responsibilities of the healthcare professional and person receiving authorisation, including patient monitoring, data collection, surveillance of adverse drug reactions, procedure for reporting of adverse drug reactions by a healthcare professional, current and planned clinical research (indicating sponsor, clinical trials sites in Latvia and other countries)
3. A substantiated justification by a healthcare institution for the use of medicinal product, including medical or epidemiological data regarding the patient or patient group (special treatment program) in compliance with the criteria stipulated by Article 83(2) of the Regulation No. 726/2004: :
- A chronically or seriously debilitating disease or a disease that is considered to be life-threatening.
- A disease that cannot be treated satisfactorily by a medicinal product authorised via the national authorisation procedure in Latvia, mutual recognition or decentralised procedure, or centralised authorisation procedure in the European Union.
4. A copy of the marketing authorisation application submitted to EMA and supplementary data and documents, if necessary.
5. Opinion of the EMA Committee for Medicinal Products for Human Use (if such exists) or opinion of a competent authority in any other European Union member state regarding the relevant medicinal product.
Patient rights and surveillance of adverse reactions
The medicinal product may be issued only upon request from a healthcare professional for specific patients in a healthcare institution, strictly following the requirements of the aforementioned documents. Taking into account that compassionate use of medicinal products bears a high risk for the patient, the State Agency of Medicines recommends conducting a patient informed consent procedure in all cases and signing of the appropriate documentation. Special attention should be paid to the surveillance of adverse drug reactions.
SAM should be informed about the progress of the program every quarter, and information regarding the planned or premature completion of program should also be submitted to SAM. Adverse drug reactions should be reported in accordance with Articles 17.1, 17.2 and 17.3 of Regulation No. 47.
Forms for receipt of permit for distribution of unauthorised medicinal products:
Application for permit for distribution of individually supplied unauthorised medicinal products (permit requested by a medicinal product wholesaler or medicinal product intended for compassionate use).
Payment for the evaluation of application documentation for the permit for distribution of individually supplied unauthorised medicinal products should be made in accordance with Article 15 and 16 of the SAM paid service pricelist. An invoice will be sent to the client after receipt of application. Upon making payment, the invoice number and date, as well as the name, surname or title of the payer must be indicated in the “purpose of payment”.
Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency
Cabinet of Ministers Regulation No. 416 of 26 June 2007 “Procedures Regarding Distribution and Quality Control of Medicinal Products”
Cabinet of Ministers Regulation No. 47 of 22 January 2013 “Pharmacovigilance Procedures”
Information of compassionate use of medicinal products on the European Medicines Agency website