Transcript or” non-standard chemotherapy”

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