Current Reimbursement of Enzyme Replacement Therapy for MPS I

Updated Feb 2014
Reimbursed by the Government
All access to ERT is through the Australian Life Saving Drug Programme. Many children
and adults remain untreated. ERT funded through Life Saving Drugs Programme (LSDP)
however criteria regarding data was tightened in 2010 making reimbursement of future
ERTs for the other MPS’s much less certain. MPSI – ERT funded for Hurler/Scheie but not
for patients undergoing transplant. Funding for ERT pre and post-transplant is not covered
by LSDP and historically has been reliant on compassionate funding from pharma.
Currently such compassionate funding is problematic to obtain and is not a viable option
for long term. Society currently advocating for ERT for MPSI patients undergoing
transplant to be funded by LSDP. MPSII – ERT funded for those without neurological
involvement. This has proven particularly challenging for treating very young patients. A
small number of patients with mild neurological involvement are now receiving LSDP
funded ERT following much lobbying from individual families and Society but constant
threat of having ERT withdrawn if neurological decline becomes significant.
MPSVI – ERT funded for all.
One MPSI patient on ERT, one MPSV patient on ERT and 7 MPSII patients on ERT. There
is no official law to get the ERT funded by our government, however all of these are paid.
Additionally we have three MPSIVA patients on the ERT trial and once the drug is licensed
we will have quite a few patients more willing to do the ERT. To this time we have no
commitment from anyone for paying, but hope that it will work like it does in other MPS.
Reimbursed by social security. Number of patients with reimbursement: 13 MPSI, 9 MPSII
& 6 MPSVI.
400 patients on ERT for MPSI, MPSII and MPSVI. Patients have to go to judicial review to
secure reimbursed treatment
In Bulgaria patients with MPSII treated and treatment is reimbursed by the Ministry of
Health. We have one patient with MPSVI, his treatment is covered by the Fund to treat.
Our objective for 2014 is the treatment of MPSVI funded by the Ministry of Health as
ERT not recommended to be funded by Federal Common Drug Review; however, it is
funded on a case-by-case basis in most provinces (funding decisions are made
provincially). Currently there is one patient with MPS I in New Brunswick being denied
funding; all other eligible patients are being funded.
Patients in Shanghai receive $200,000 per year towards the cost of ERT. No other areas of
mainland China offer reimbursed ERT
Reimbursed by the Government
ERT therapy is fully covered for all patients with MPS I, MPS II and MPS VI through the
Croatian Health Insurance System.
ERT in Czech Republic is reimbursed by Helaths Insurance companies through the
goverment selected hospital. Because of limited budget, some adults remain untreated.
ERT therapy is totally covered by the French National Health Insurance
All MPS patients eligible for ERT receive ERT funded by health insurance
Reimbursement of ERT for adults is a problem. 2 adult males with normal spectrum IQ are
denied ERT for MPSII. 2 MPS II & 2 MPS VI on ERT funded by the Government
There is NO reimbursement in INDIA
All patients receive ERT reimbursed by the Department of Health through the hospital. 5
MPSI, 10 MPSII and 1 MPSVI patients are receiving reimbursed ERT
10 MPSI, 100 MPSII and 4 MPSVI are receiving ERT funded jointly by pharma companies
and National Health Insurance / Rare Disease Fund
39 MPS patients diagnosed. 15 are on Government reimbursed ERT and 13 are waiting
for ERT.
ERT for patients up to 18 years of age is reimbursed.
ERT’s for MPS I (milder phenotypes, for MPS I H, SCT is preferred treatment), MPS II and
MPS VI are reimbursed by insurance companies to specific centres of expertise (Erasmus
MC in Rotterdam for MPS II and VI and AMC Amsterdam and Erasmus MC Rotterdam for
MPS I. Insurance companies are in turn reimbursed by the Health ministry. Treatment has
to be coordinated by one of the centres. Treatment can take place at home in most cases.
Since the discussion about reimbursement for Fabry and Pompe disease, start and stop
criteria for the clinical aspects of LSD’s will be deployed. In most cases with severe
neurologic involvement this can lead to discontinuation of treatment, when clinical benefit
does not show (anymore). We hold the position that stopping treatment is a decision the
clinical specialist can only take in consensus with parents/relatives of the patient
1 Hunter patient fully funded 2nd Hunter patient denied access and had bone marrow
transplant with 3 months ERT prior to BMT. One MPSI on compassionate ERT. Six MPSI
patients given 3 months ERT prior to BMT. No other patients funded.
We are currently waiting on a decision for Pompe and anticipate that Pharmac will decline
funding again which will see us taking the ERT issues to court for a Declarity Judgement
and working through International Human rights law. 21 Gaucher patients are also funded.
Regarding reimbursement of ERT for MPSI, MPSII, and MPSVI, Health Insurance covers
the cost for ERT treatment for MPS I, II and MPSVI when clinically indicated.
Reimbursed by the Government, all eligible for ERT patients with MPS I, MPS II, MPS VI
and Pompe disease
MPS-I – 58 patients treated with ERT, 7 patients with no treatment
MPS-II – 60 patients treated with ERT, 30 patients with no treatment
MPS-VI – 11 patients treated with ERT, 13 patients with no treatment
There are different financing options and the availability of treatment varies from region to
reigon of the country.
Over 100 patients on ERT funded by the Government. 40 MPSII patients on Hunterase a
biosimilar ERT to Elaprase. Hunterase is approved by the regulatory authorities in South
Korea to treat MPSII patients.
All patients have treatment reimbursed by the Government for MPS I, II and VI, at the
moment. Sometimes if the patients have a serious neurological problem they are declined
treatment. This depends on the Hospital and the Community of the family.
13 patients with MPSI of which 11 are Bone Marrow transplanted and 2 are on ERT.
12 patients with MPSII of which 10 are on ERT and two will start ERT.
3 patients with MPSIV of which 2 are on ERT and 1 received a BMT.
Until the age of 20 the Federal Insurance covers all ERTs, after that the (mandatory)
insurances take over. It always takes a little time fighting, but so far we’ve been lucky.
ERT is fully covered for all patients with MPS I, MPS II & MPS VI through the Government
Health Insurance System.
ERT for patients with MPSII is reimbursed so long as they meet the government restriction
of a minimum IQ of 50
The Department of Health – NHS England funds ERT for all eligible patients with MPSI,
The Department of Health – NHS Wales does not fund ERT for all eligible patients and
many children and adults remain untreated.
The Department of Health – NHS Scotland does not fund ERT for all eligible patients and
some children and adults remain untreated.
The Department of Health – NHS Northern Ireland funds ERT for all eligible patients.
All MPS patients eligible for ERT receive ERT funded either by private health insurance or
government funded health insurance.