Compassionate Early Access Programs for Medicines: A Path to New Cancer Treatments

For life-threatening diseases with challenging treatment courses, such as cancer, access to new treatment options is especially important for patients whose disease progresses despite standard therapies. In this context, Compassionate Early Access Programs for Medicines, coordinated by the Ministry of Health in Turkey, offer a promising option. These programs provide free access, through manufacturers, to medicines that have not yet been approved in Turkey but are approved in other countries. The programs cover various disease groups, not only oncology. Details of the ongoing oncology programs are provided below.

Compassionate Early Access Programs in Cancer

Current List and Details as of November 2024

1. Program Code: CU 201

• Sponsoring Company: Bristol Myers Squibb İlaçları Inc.
• Active Ingredient (Brand Name): Repotrectinib (Augtyro)
• Diagnosis: Locally advanced and metastatic non-small cell lung cancer in ROS-1-positive patients who have previously received ROS-1 TKI therapy, or NTRK-positive patients who have previously received NTRK TKI therapy.
• Remaining Quota: 14 patients.

2. Program Code: CU 203

• Sponsoring Company: Pfizer PFE İlaçları A.Ş.
• Active Ingredient (Brand Name): Enzalutamide (Xtandi) and talazoparib (Talzenna)
• Diagnosis: Metastatic castration-resistant prostate cancer with homologous recombination repair gene mutations (HRRm).
• Remaining Quota: 123 patients.

3. Program Code: CU 211

• Sponsoring Company: AstraZeneca İlaç San. Tic. Ltd. Şti.
• Active Ingredient: Durvalumab (Imfinzi) and olaparib (Lynparza)
• Diagnosis: First-line durvalumab, or durvalumab together with olaparib, for patients with newly diagnosed advanced or recurrent endometrial cancer, particularly according to dMMR and pMMR status.
• In the pMMR group, patients will receive maintenance durvalumab + olaparib after chemotherapy + durvalumab. In the dMMR group, maintenance treatment after chemotherapy + durvalumab will consist of durvalumab alone.
• Remaining Quota: 29 patients.

What Are dMMR and pMMR?

dMMR and pMMR concern the DNA repair mechanisms in tumors of patients with endometrial cancer. MMR stands for "Mismatch Repair," a DNA repair mechanism that corrects mismatched base pairs.

dMMR (Mismatch Repair-deficient)  tumors are tumors in which this mechanism has lost its function and cannot correct mismatched base pairs in DNA. This leads to a genetic alteration called high microsatellite instability (MSI-H). These tumors generally tend to be more aggressive and more susceptible to the immune system, so they may respond better to immunotherapy.

pMMR (Mismatch Repair-proficient)  tumors, by contrast, have a properly functioning MMR mechanism that can correct mismatched base pairs in DNA. These tumors are called microsatellite stable (MSS) and tend to show a lower immune response. It is therefore important to understand how immunotherapy works in these tumors.

4. Program Code: CU 216

• Sponsoring Company: AstraZeneca İlaç San. Tic. Ltd. Şti.
• Active Ingredient: Durvalumab (Imfinzi)
• Diagnosis: Limited-stage small cell lung cancer without progression following platinum-based chemoradiotherapy.
• Remaining Quota: 20 patients.

5. Program Code: CU 220

• Sponsoring Company: GlaxoSmithKline e Research & Development Ltd.
• Active Ingredient: Dostarlimab (Jemperli)
• Diagnosis: Newly diagnosed, recurrent, or advanced endometrial cancer with mismatch repair deficiency/high microsatellite instability (MSI-H).
• Remaining Quota: 50 patients.

Turkey's Compassionate Early Access Program for Medicines (İAEEP) aims to provide individuals with serious or life-threatening diseases, for whom available treatment options in Turkey have been exhausted or proved inadequate, with access to medicines that are not yet licensed in Turkey but are approved in other countries. By enabling early access to treatment, the program aims to improve patients' quality of life.

Application Process:

1. Patient Assessment:

   • Patients with treatment-resistant disease or whose available treatment options have been exhausted should discuss applying to the İAEEP with their oncologists.

2. Preparing the Required Documents:

   • Patient Medical Summary: The patient's medical history, diagnosis, treatments received, and current condition should be documented in detail.
   • Treatment Rationale: A report explaining the medical rationale for using the medicine and its expected benefits should be prepared.
   • Patient Consent Form: A signed consent form should be obtained, showing that the patient has been informed about the proposed treatment and potential risks and agrees to treatment.

3. Submitting the Application:

   • The prepared documents should be submitted by the physician responsible for the patient's treatment to the Turkish Medicines and Medical Devices Agency (TİTCK) (see the TİTCK Guideline on the Compassionate Early Access Program for Medicines). Information such as the company supplying the medicine and the duration of its use should also be provided with the application.

4. TİTCK Assessment:

   • TİTCK evaluates the application from medical, ethical, and legal perspectives. If it is deemed appropriate, the necessary permissions for importing and using the medicine are granted.

5. Supplying and Administering the Medicine:

   • Following approval, the medicine is supplied by the relevant company and the patient's treatment begins. During treatment, the patient is monitored regularly by their physician, and the necessary reports are submitted.

Important Considerations:

• The application process should be managed by the physician responsible for the patient's treatment.
• The manufacturer or authorized distributor of the relevant medicine should be contacted for information about obtaining it.
• The application process should comply with the guidelines and forms specified by TİTCK.

Conclusion and Recommendations

Compassionate early access programs for medicines open a vital door for patients who seem to have exhausted their available cancer treatment options. Patients wishing to benefit from these programs need to communicate in detail with the oncologists managing their treatment and follow the Ministry of Health's approval processes in Turkey.