Rafał Zyśk

Financing of therapy with highly expensive medicinFinancing of therapy with highly expensive medicineses by the by the

National Health FundNational Health Fund

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Medicines Management DepartmentNational Health Fund

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Services, under which highly expensive medicines technologies may be financed

Therapeutic programmes Non-standard pharmacotherapy Non-standard chemotherapy

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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)

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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!

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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)

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LIST OF RARE DISEASES FINANCED BY THE NFZ UNDER PT

2005 2006 2007

Name of PT Value of services provided

Value of services provided

Estimated value of

contracted services

Amount per PT in the whole country

573,000,610 618,467,170 640,000,000

CHANGE dymanics in 2005/2006

CHANGE dymanics

in 2006/2007

PROGRAMME FOR GAUCHER DISEASE TREATMENT WITH IMIGLUCERASE

26,071,790 30,886,790 34,000,000 18.47% 10.08%

PROGRAMME FOR HURLER DISEASE TREATMENT WITH LARONIDASE

4,627,700 8,076,700 9,545,980 74.53% 18.19%

TOTAL 30,699,490 38,963,490 43,545,980

% of the whole amount 5.36% 6.30% 6.80%

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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

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Not recommended for public financing

by the Polish HTA Agency:

TREATMENT OF MUCOPOLYSACCHARIDOSIS TYPE VI WITH NAGLAZYME MEDICINE

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Therapeutical programs in 2007 1. Treatment of breast cancer with docetaxel

2. Treatment of breast cacner with capecitabine

3. Treatment of breast cancer with trastuzumab

4. Treatment of colorectal cancer with irinotecan

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

11. Treatment of ovarian cancer with paclitaxel

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

16. Treatment of infantile cerebral palsy with botulin toxin

17.Treatment of sclerosis multiplex with glatiramer

18. Treatment of sclerosis multiplex with interferon beta19. Treatment of chronic virus hepatitis type B with lamivudine

20. Treatment of myocardial infarction with abciximab

21. Treatment of rheumatoid arthritis with infliximab

22. Treatment of sepsis with activated protein C

23. Treatment of anaemia of non-dialysed patients with chronic renal disease

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

29. Treatment of rheumatoid arthritis with leflunomide

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

35. Immunosupressive treatment with sirolimus

36. Immunosupressive treatment with tacrolimus

37. Treatment of children with Prader Willi syndrome with growth hormone

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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

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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.

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Economic development vs. Development of medicine

ECONOMY

MEDICINE

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Sense of the application of medical technology

Brings more benefit than harm

Brings more harm than benefit

Effect unknownEffect unknown

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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

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Compliant with indications or not?

AUTHORIZATION STATUS CLINIC REGULATOR’S „PROBLEMS”

On label On label Current medicine development

Off label

Soft labelApplication in accordance with the

current medicine development while there are no authorization indications

Off label Medical experiment

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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.

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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.

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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

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Cost of therapy has become an issue of ethics!

PLN 5 million

3 children and low health benefit

300 adults with cancer and high health benefit – extension of life by 10 years

on average

Queue is a factor of proven harmfulness

Restriction of availability

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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?

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Prescrire International

– effectiveness of new medicines

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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.”

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Thank you for your attention