THE GLOBAL GUIDE
TO COMPASSIONATE
USE PROGRAMS

Given the recent catastrophic earthquake in Turkey, we are making available for humanitarian need the requirements for the Turkish Ministry of Health for distributing medicines for compassionate use.  Using this document, any manufacturer or sponsor can distribute medication to those in need. Please contact us should you need any more information.

Compassionate Use Programs in Turkey

Regulatory Mechanism Available?

Yes

Type of Mechanism Available

Compassionate Use Program (CUP) for either groups or individual patients

 Named Patient is possible only after the product has one active marketing authorization.

Regulatory Agency

Ministry of Health (Turkish)

Turkish Medicines and Medical Devices Agency (Turkish)

Guidance Document

Ministry of Health General Directorate of Pharmaceuticals and Pharmacy, October – 2005 (İnsani Amaçlı İlaca Erken Erişim Programı Kılavuzu)

Legal Basis for Program

The legislative basis of these programs is based on article 27 and 40 of the decree on the Organization and Duties of the Ministry of Health and its Affiliates dated 11/10/2011 with number 663 and additional article 7 of the law on pharmaceuticals and medical preparations dated 14/5/1928 with number 1262. Based on the legislation described above, there is a guideline published by TITCK describing the details of the process.

Program Description for Compassionate Use

This program is designed for patients who have a serious or life-threatening disease that have been treated with medicinal products that are licensed and accessible by the Turkish Ministry of Health and who do not qualify for a clinical trial in Turkey. The drug must be provided free of charge by the developing company on humanitarian grounds.

Initiating the Process

The Sponsor or licensed Turkish physician may apply on behalf of a patient who meets the qualifications under compassionate use.

Role of the Sponsor or CUP Holder

Sponsors that require a therapy to be included in the Early Access to Humanitarian Program must first apply to the “Clinical Research Department of the General Directorate of Pharmaceuticals and Pharmacy (IEGM)” for inclusion. The Local Ethics Committee can be applied simultaneously. The required information depends on the type of request:

For single patient compassionate use application:

• the requested medicine
• the reason for the request by a licensed physician for a particular patient
• the amount of medication to be used
• a signed Patient Consent Form
• a local Ethics Committee Decision
• a signed letter from IEGM indicating the responsibilities of the treating physician
• a signed letter informing the Ministry of Health at the conclusion of therapy

Patient Group compassionate use application after IEGM approval:

• drug information
• recommended dose
• rationale for the use of a specified drug in a cohort of patients
• drug formula
• mechanism of action
• drug-drug interactions
• preclinical and clinical studies
• safety profile
• information about the program, number of patients, sites and all case report forms used
• a signed letter from IEGM indicating the responsibilities of the treating physician
• every 3 months, a report must be filed on the effectiveness and safety of the therapy for the designation patients
• A final report to the Ministry at the conclusion of the program

If this application is deemed appropriate by the Ministry of Health the Sponsor applies for importation permit with the amount of drug to be imported into Turkey.

Alternative Regulatory Mechanism for Access 

Named Patient import is possible only after the product has one active marketing authorization. This regulatory pathway falls under ‘non-authorised medicinal product for individual use from abroad’.

Non-authorised medicinal product for individual use from abroad

If the product is not included on the list of foreign medicinal products and which are prescribed for the first time for a patient, a prescription including the information of the patient and his/her physician and Health Board Report or Drug Usage Report is sufficient for the first application to be made to the TITCK, the Turkish health authority. When import is approved by the TITCK, drug product would then be added to the List of Foreign Medicinal Products.

The physician must get approval from TITCK before prescribing the medicine. TITCK may require additional information depending on the drug in question.

Application documents are as follows:

• application form
• scientific committee or medicinal product use report
• informed consent form signed by the patient
• literature for unlisted products in the guideline
• medical report including previous treatments, response of patient to these treatments and current laboratory values about diagnostics as applicable

An application must be submitted with the documents referenced above but additional documents and/or test results may be requested.

If a product is not commercialized in Turkey, a site in Turkey can only obtain/import it via the Union of Turkish Pharmacies (TEB), which is a public institution. The manufacturer (or their designee can contact TEB, who then support the import process and deliver the drug to the site or another party, such as a wholesaler in the country.

Previously Authorized CUP

Heidenreich A, Bracarda S, Mason M, et al. Safety of cabazitaxel in senior adults with metastatic castration-resistant prostate cancer: results of the European compassionate-use programme. Eur J Cancer. 2014;50:1090-9.

Chargeable Component?

A sponsor or CUP holder cannot charge for the medicine under a CUP (either single patient or groups of patients). Charging for the medicine under “Non-authorised medicinal product for individual use from abroad”, (named patient), is possible once the drug is added to the list of ‘Non-authorised medicinal products for individual use from abroad’. It is the responsibility of the physician to obtain the funding to purchase the medicine. 

Pharmacovigilance Requirements

The Turkish Ministry of Health (T.C. Sağlik Bakanliği) is division of Turkish Medicines and Medical Device Agency (Türkiye Ílaç ve Tikki Cíhaz Kurumu TITCK) which oversees the Turkish Compassionate Use Program, referred to as İnsani Amaçlı İlaca Erken Erişim Programı Kılavuzu.

The İlaç ve Eczacilik Genel Müdürlüğü, or General Directorate of Pharmaceuticals and Pharmacy, requires that all events, whether related to the medicine or the expanded access program and all serious events and deaths be reported within eight (8) days to the Directorate.

The reporting form can be downloaded in Word format at Advers reaksiyon bildirim formu and sent via email to tufam@titck.gov.tr. The completed form can also be faxed to TITCK at 0312 218 35 99 or called in at 0 312 218 30 00 (central number).

Please note that there are specific forms to report adverse events involving hepatotoxicity,  nephrotoxicity, or a known side-effect.

All documents must be completed in Turkish.

Labeling Requirements for Compassionate Use

Label requirements for drug in a group program would be agreed upon with the Ministry of Health (MOH) as part of the approval for the program.

Labeling Requirements for Non-Authorized Medicinal Product for Individual Use from Abroad (Named Patient Import)

As named patient import is only possible after first marketing authorization, a bona fide commercial pack is usually acceptable, i.e., US commercial pack or EU commercial pack.