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    Medicines used in CUP and NPP programs

    22.09.2017
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    Through special programmes like Compassionate Use Programme (CUP) and Named Patient Programme (NPP) medicines still in clinical trials are made available for patients whose treatment options with authorised medicines have depleted and who cannot enter a clinical trials (for example if there is no clinical trial running in Estonia or patient is not suitable for the trial). CUP and NPP can also be used to continue treatment of patients who started that treatment in a clinical trial. Medicines used in CUP/NPP should have successfully passed at least phase II clinical trials.

    Through CUP the medicine is made available for a group of patients and through NPP for a single named patient.

     

    Programmes can be started if:

    • The medicine is subject of an application for a centralised marketing authorisation from the European Commission (clinical trial is still running or the application is still in the proceedings) or the medicine is already authorised, but the programme is run for other indications than authorised.
    • The medicine is used for the same indications as in the running clinical trial or the application is still under the evaluation.
    • The medicine is used as the last treatment option for a life-threatening disease when there are no alternatives or alternatives cannot be used due to the condition of the patient.
    • The medicine is used for patients with chronic or disabling disease, who cannot be treated with authorised medicines or who cannot take part in a clinical trial (patients not suitable for the trial or the inclusion has ended).

    When there is a suitable clinical trial running in Estonia the patients should always be considered for inclusion in the trial.

     

    How to start a programme?

    • The medicine has passed successfully phase II clinical trials.
    • Initiative for starting a programme can come from the physician or from the company.  
    • Physician/healthcare institution and the company sign a contract in which among others, they agree upon collection and reporting of safety data.
    • Company notifies the State Agency of Medicines about starting a new programme and provides the following information via e-mail info@ravimiamet.ee:
    • The current summary of clinical trials (safety and efficacy).
    • Indications of the medicine to be used in the programme.
    • Number of patients to be included in the programme and criteria of inclusion agreed upon with the physician/healthcare institution.
    • List of healthcare institutions participating in the programme.
    • Information about how the company is planning to provide the medicine after it has been granted a marketing authorisation, but before it has been reimbursed by the Estonian Health Insurance Fund.
    • Physician applies for a permit to use an unlicensed medicine from State Agency of Medicines (additional information regarding unlicensed medicine application: http://ravimiamet.ee/myygiloata-ravimite-info-arstile#ARSTI TAOTLUSED ÜHELE PATSIENDILE).
    • The programme is conducted according to the approved conditions.

     

    During the programme the company reports to the State Agency of Medicines about:

    • Any changes in the programme e.g. plan to include new patients, a marketing authorisation granted by the European Commission, reimbursement granted by the Estonian Health Insurance Fund and ways of providing medicine for patients not applicable for reimbursement.
    • Ending of the programme.

     

    Solicited reporting during CUP/NPP

    The company/Marketing Authorisation Holder (MAH):

    • has to guarantee that a physician records and documents complete and comprehensive case information on adverse reactions* and serious adverse events (GVP IV.C.I.2 and VI.C.I.2.2) and transmits the data to the contact person of the company/MAH given in the contract signed between company/MAH and physician or healthcare institution.

    * According to the Medicinal Products Act § 786 section 4 a person qualified to prescribe medicinal products is required to inform the State Agency of Medicines of all serious adverse reactions. According to the Medicinal Products Act § 786 section 6 the MAH may be informed of an adverse reaction. It is acceptable that all safety data collected from the programme is reported to the company/MAH for further assessment and collection of the follow-up information.

    • has to assess the risks associated to the adverse events. A method of causality assessment should be applied for assessing the causal role of the medicinal products (= adverse reaction) in the solicited adverse events (for example, the WHO-UMC system for standardised case causality assessment).
    • Only valid ICSRs of adverse reactions should be reported to EVDAS - ’Post-marketing’ – ’Report from study’ – ’Other studies’ (CUP) or 'Individual patient use’ (NPP) (GVP IV.B.2, VI.C.3., VI.C.4. and VI.C.6.2.3.7). Adverse reactions should be presented and assessed in the periodic safety update report.
    • In situations where adverse reactions are suspected to be related to other medicinal products, these reports should be managed, classified and reported as spontaneous ICSRs (Medicinal Products Act §786 and Regulation of the Minister of Social Affairs no 26 § 2.