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Medicines used in Compassionate Use programs

Through special programmes like Compassionate Use Program (CUP) medicines still in clinical trials are made available for patients whose treatment options with authorised medicines have depleted and who cannot enter clinical trials (for example if there is no clinical trial running in Estonia or patient is not suitable for the trial).

CUP can also be used to continue treatment of patients who started the treatment in a clinical trial. Medicines used in CUP should have successfully passed at least phase II clinical trials.

Through CUP the medicine is made available for a group of patients or for a single 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 as in the application for marketing authorisation.
  • The medicine is used
    • as the last treatment option for a life-threatening disease when there are no authorised medicines available  or they have not been effective/tolerated for the patients or
    • for patients with chronic or disabling disease, who cannot be treated with authorised medicines.
  • There is no clinical trial running that would be suitable for the patients (no trial running in Estonia, patients are not suitable for the trial or inclusion into the trial has ended).

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  submits an application for starting a new CUP to the State Agency of Medicines and provides the following information via e-mail [email protected]:
    • 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 inclusion criteria 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 unauthorised medicine from State Agency of Medicines (additional information regarding application for using an unauthorised medicine can be found below "Arsti taotlused ühele patsiendile").
  • The programme is conducted according to the approved conditions.

1. Arst koostab retseptikeskuse kaudu digiretsepti

  • märgib lisaks tavapärastele retsepti andmetele müügiloata ravimi kasutamise põhjenduse, mis valitakse etteantud loendist (müügiloata ravimi digiretseptil kohustuslik andmeväli);
  • vajadusel täiendab põhjendust tekstiväljal (nt nimekiri varem kasutatud müügiloaga ravimitest, millele patsiendil on allergia vm);
  • lisab oma e-posti aadressi;
  • teavitab patsienti, et kirjutas välja müügiloata ravimi, annab infot ravimi kohta ning tutvustab patsiendile edasist taotluse menetlusprotsessi.

Arst saab välja kirjutada ühekordse või korduvretsepti (kui ravimil ei ole piirangut korduvretsepti kirjutamiseks).

Ravimi väljakirjutamisel tuleb lisaks arvestada järgmist:

  • Kui vajaliku toimeainega müügiloata ravimit ei ole varem Eestisse toodud ja toimeainet ei ole retseptikeskuse toimeainete loendis, esitab arst taotluse müügiloata ravimi kasutamiseks Ravimiametile kliendiportaali kaudu.
  • Müügiloata ravimi kasutamiseks on vaja Ravimiameti otsust, st enne positiivse otsuse saamist ei saa patsient ravimit apteegist osta.
  1. Digiretsept edastatakse läbi retseptikeskuse Ravimiametisse ja Ravimiameti teeb otsuse

Digiretseptist saab taotlus müügiloata ravimi kasutamiseks. Ravimiamet menetleb taotlust ja teeb otsuse müügiloata ravimi kasutamise lubamiseks/mittelubamiseks enamusel juhtudest kuni 14 päevaga, maksimaalselt võib kuluda 30 päeva.

Juhul kui taotluse menetlemisel tekib Ravimiametil lisaküsimusi, võtab Ravimiamet arstiga ühendust e-kirja teel.

  1. Ravimiamet teavitab oma otsusest arsti, kes teavitab omakorda patsienti

Pärast otsuse tegemist saadab Ravimiamet arstile e-kirja, kasutades digiretseptil olnud e-posti aadressi.

  • Positiivse otsuse puhul muutub retsept aktiivseks, patsient võib minna apteeki ja esitada soovi ravimi väljaostmiseks. Apteek broneerib retsepti, tellib ravimi, teavitab patsienti ravimi saabumisest.
  • Kui arst saab teate, et taotlust ei ole rahuldatud ja retseptikeskuses on negatiivne otsus, siis apteek ei saa selle retsepti alusel ravimit väljastada. Sel juhul tuleb patsienti teavitada ja tema ravi ümber korraldada.

Arstil on võimalik retsepti seisu vaadata retseptikeskusest. Kui retsepti staatus on „otsuse ootel“, ei ole Ravimiamet otsust veel teinud. Positiivse otsuse korral on staatus „otsus positiivne“ ja negatiivse otsuse korral „otsus negatiivne“.

Patsient näeb oma retsepti seisu portaalidest ja

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

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. 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.

Last updated: 30.06.2022