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Financing of therapy with highly expensive medicines by the National Health Fund Rafał Zyśk Medicines Management Department National Health Fund Rafał Zyśk Services, under which highly expensive medicines technologies may be financed Therapeutic programmes Non-standard pharmacotherapy Non-standard chemotherapy Rafał Zyśk Assumptions for therapeutic programmes • • • • • • • • intended for most expensive therapies intended for small patient populations intended for indications with clearly defined criteria – inclusions and exclusions of patients monitoring of the number of patients and therapy effectiveness (planned) medical technologies of proven clinical effectiveness medical technologies, which are most cost-effective among the available options taking the public payer budget into account improvement of the reporting and financial settlements system – monitoring of patients in PT in accordance with the rules of clinical register! and verification of the legitimacy of financing technologies after 1-3 years on the grounds of factual data (plans for the future) Rafał Zyśk Features of a good register 1. Maintained on the grounds of a protocol, 2. directed to primary endpoints (survival, recovery/symptoms arduous for patients, quality of life) , 3. collects data regarding important events, which generate costs, 4. covers – if possible – the whole population covered by treatment, 5. monitored / external audit, 6. connected to other similar registers in other countries – COLLECTIVE RESULTS! Rafał Zyśk Within the framework of therapeutic programmes catalogue, NFZ finances two Orphan Medicinal Products officially authorised by the EU: PROGRAMME FOR GAUCHER DISEASE TREATMENT WITH IMIGLUCERASE (56 patients), PROGRAMME FOR HURLER DISEASE TREATMENT WITH LARONIDASE (13 patients) Rafał Zyśk LIST OF RARE DISEASES FINANCED BY THE NFZ UNDER PT 2005 2006 2007 Value of services provided Value of services provided Estimated value of contracted services 573,000,610 618,467,170 640,000,000 26,071,790 30,886,790 4,627,700 TOTAL % of the whole amount Name of PT Amount per PT in the whole country PROGRAMME FOR GAUCHER DISEASE TREATMENT WITH IMIGLUCERASE PROGRAMME FOR HURLER DISEASE TREATMENT WITH LARONIDASE CHANGE dymanics in 2005/2006 CHANGE dymanics in 2006/2007 34,000,000 18.47% 10.08% 8,076,700 9,545,980 74.53% 18.19% 30,699,490 38,963,490 43,545,980 5.36% 6.30% 6.80% Rafał Zyśk In the course of assessment by the Polish HTA Agency: TREATMENT OF POMPE DISEASE WITH MYOZYME MEDICINE TREATMENT OF FABRY DISEASE WITH FABRAZYME MEDICINE TREATMENT OF NEWMANN-PICK DISEASE WITH ZAVESCA MEDICINE Rafał Zyśk Not recommended for public financing by the Polish HTA Agency: TREATMENT OF MUCOPOLYSACCHARIDOSIS TYPE VI WITH NAGLAZYME MEDICINE Rafał Zyśk Therapeutical programs in 2007 1. Treatment of breast cancer with docetaxel 20. Treatment of myocardial infarction with abciximab 2. Treatment of breast cacner with capecitabine 21. Treatment of rheumatoid arthritis with infliximab 3. Treatment of breast cancer with trastuzumab 22. Treatment of sepsis with activated protein C 4. Treatment of colorectal cancer with irinotecan 23. Treatment of anaemia of non-dialysed patients with chronic renal disease 5. Treatment of colorectal cancer with capecitabine 6. Treatment of glioma with temozolomide 7. Treatment of ovarian cancer with topotecan 8. Treatment of chronic myeloid leukaemia with imatinib 9. Treatment of gastrointestinal stromal tumours (GIST ) with imatinib 10. Treatment of malignant lymphoma with rituxmab 24. Enzyme replacement therapy of Gaucher disease with imiglucerase 25.Treatment of dwarfish chldren with somatotropin hypopituitarism with growth hormone 26. Treatment of dwarfish chlidren with Turner syndrome with growth hormone 27. Treatment of dwarfish chlildren with chronic renal disease with growth hormone 28. Enzyme replacement therapy of Hurler disease with laronidase 11. Treatment of ovarian cancer with paclitaxel 29. Treatment of rheumatoid arthritis with leflunomide 12. Treatment of idiopathic thrombocytaemia with anagrelide 13. Treatment of ovarian cancer with paclitaxel 14. Treatment of children with primary immune deficiency with immunoglubulin 15. Treatment of myodystonia with botulin toxin 30. Treatment of rheumatoid arthritis and juvenile arthritis with etanercept 31. Treatment of virus hepatitis C or B with pegylated interferon alpha 32. Treatment of virus hepatitis B or C with natural interferon alpha 33. Treatment of virus hepatitis B or C with recombinant interferon alpha 34. Immunosupressive treatment with mycophenolate mofetil 16. Treatment of infantile cerebral palsy with botulin toxin 35. Immunosupressive treatment with sirolimus 17.Treatment of sclerosis multiplex with glatiramer 36. Immunosupressive treatment with tacrolimus 18. Treatment of sclerosis multiplex with interferon beta 19. Treatment of chronic virus hepatitis type B with lamivudine 37. Treatment of children with Prader Willi syndrome with growth hormone Rafał Zyśk New therapeutical programs in 2008 Breast cancer - adjuvant therapy with trastuzumab or docetaxel Chronic myeloid leukaemia - second line therapy with double dose of imatinib or dasatinb Myocardial infarction - eptifibatide or tirofiban RA and JA - first line therapy with infliximab or adalimumab RA and JA - second line therapy with rituximab or abatacept Gaucher desease – enzyme replacement therapy with miglustat Lamivudine- resistant chronic hepatitis B - therapy with entecavir or adefovir Plasmocytoma - therapy with bortezomib Crohn’s disease in children - therapy with infliximab Crohn’s disease in adults - therapy with budesonide or adalimumab or infliximab Pulmonary arterial hypertension - first line therapy - sildenafil - second line therapy - iloprost Rafał Zyśk Financing of therapy under procedure „non-standard pharmacotherapy” or” non-standard chemotherapy” service allowing for the therapy of patients, whose treatment may not be qualified under other items of Hospital treatment catalogue, requirement of obtaining individual authorization of the Director of Voivodeship Branch of NFZ each time financing does not increase the volume of the contract of Voivodeship Branch of NFZ against the hospital Director if the Voivodeship Branch, when issuing authorization, should take account of the financial condition of the applying hospital. Rafał Zyśk Economic development vs. Development of medicine MEDICINE ECONOMY Rafał Zyśk Sense of the application of medical technology Brings more benefit than harm Brings more harm than benefit Effect unknown Rafał Zyśk Market authorization – legal term On label in accordance with the authorization indications, results of Phase III, RCT Off label non-compliant with authorization indications Soft label non-compliant with authorization indications, but there are premises regarding the effectiveness of therapy on the basis of research with methodological restrictions MARKET AUTHORISATION REIMBURSEMENT Rafał Zyśk Compliant with indications or not? AUTHORIZATION STATUS CLINIC REGULATOR’S „PROBLEMS” On label On label Current medicine development Soft label Application in accordance with the current medicine development while there are no authorization indications Off label Medical experiment Off label Rafał Zyśk Position of the Minister of Health of 14.12.2006 1. The „application of a medicine in accordance with the indications of the current medicine development” is the application, which meets one of the following premises: a) therapy is based on medicinal products provided with appropriate indications in the summary of product characteristics, or b) therapy is based on long-term and well grounded clinical practice, described in current pharmacology manuals or manuals of a given medical specialisation, or c) therapy is based on reliable sceintific evidence published in renowned scientific magazines, confirming the effectiveness and legitimacy of the procedure. In case of any doubt regarding the agreement that given application of a medicinal product is „in accordance with the current medicine development” the opinion of voivodeship or national consultant in a given branch od medicine shall have the decisive power. Rafał Zyśk The Helsinki declaration distinguishes between a medical experiment and application of a medicine non-compliantly with the authorization indications In relation to the position of the Ministry of Health and ethical principles covered by the Helsinki Declaration of 1964 (as amended), in the case, where a medicinal product is applied non-compliantly with the authorization indications, the National Health Fund (NFZ) requires the service provider to: 1. confirm prior to the application, whether there are optional procedures with proven clinical effectiveness in a given indication, 2. obtain – prior to application – a written, conscious consent of the patient (pursuant to the specimen provided) to administer treatment non-compliant with authorization indications, 3. monitor the condition of the patient; in all cases new information regarding the effectiveness of a medicine and patient's condition need to be registered. Rafał Zyśk Ethics A modern doctor no longer has a moral right to perceive medicine in terms of „I do not care what I do to save the life and health of the patient”. Each decision is a trade-off – when I save X, I reduce the Y’s chances to be treated (which odes not mean recovery) – pretending longer, escaping the vital dilemma is simply burying your head in the sand. Marek Kokot Rafał Zyśk Cost of therapy has become an issue of ethics! Queue is a factor of proven harmfulness PLN 5 million Restriction of availability 3 children and low health benefit 300 adults with cancer and high health benefit – extension of life by 10 years on average Rafał Zyśk What does the decision-maker want to know (including doctors)? 1. Is it a technology of proven effectiveness? 2. What is its intervention power as compared to other options? (which of the options is most effective and to what extent does it exceed other options?) 3. Which of the options is most cost-effective and how does it exceed other options? 4. Is the financing of technologies from available funds justified? What changes would the introduction of a privilaged market position for a given technology bring? Rafał Zyśk Prescrire International – effectiveness of new medicines Rafał Zyśk 2007/056 NICE guidance on bortezomib (Velcade) is a win-win solution for multiple myeloma patients and the NHS The National Institute for Health and Clinical Excellence (NICE) has published final guidance to the NHS in England and Wales on the use of bortezomib (Velcade) for the treatment of multiple myeloma. The guidance confirms the response-rebate scheme which will allow patients at first relapse who show a full or partial response to bortezomib to carry on with the treatment, fully funded by the NHS, and patients who show no or minimal response to be taken off the drug and the drug costs refunded by the drug’s manufacturer. Andrew Dillon, NICE Chief Executive, said: “Providing expensive new cancer drugs is a challenge for the NHS, particularly when it is not possible to identify those patients who will benefit most from treatment. The scheme proposed by the manufacturer and amended by NICE will ensure that patients at first relapse, who have had one prior therapy and who have had or are unsuitable for a bone marrow transplantation will get the chance to see if the drug works well for them. This is a win-win situation for patients and the NHS.” Rafał Zyśk Thank you for your attention Rafał Zyśk