The CETP was set up last year for the benefit of Gaucher disease patients at high-risk for the development of progressive disease or complications and without access to alternative treatments during the period of Cerezyme shortage
CETP will be reinstated now due to prolonged shortage of Cerezyme supplies and based on a recommendation of the European Medicine Agency (EMA).
Introduction: This program has been set up for the benefit of patients with type I and III Gaucher disease at high-risk for the development of progressive disease or complications and without access to alternative treatments in Europe, the Middle-East and Eurasia during the period of Cerezyme shortage. Although the program had stopped in January 2010, it has been reinstated now due to continued shortage of Cerezyme supplies.
The program is conducted by a Board of Advisors consisting of an independent group of physicians, all members of the European Working Group for Gaucher Disease (EWGGD). The Board of Advisors manages the medical and clinical aspects of the program. Genzyme supports the logistical part of the program: Genzyme provides the product and manages the distribution of product based on decisions of the Board of Advisors.
CETP is not a charitable program. It will run until the stakeholders (EWGGD and Genzyme) conclude that its continuation is no longer necessary. The program is coordinated by the Academic Medical Centre (AMC), Amsterdam, The Netherlands. In order to enable AMC to do this, Genzyme provides financial support to AMC.
Background: A global shortage of imiglucerase occurred as a result of viral contamination of the production facility in June 2009. While striving to improve operations at the facility, inventory levels of Cerezyme remain low. As a consequence of a recent disruption to operations of the facility, the current reduced allocations for Cerezyme are prolonged.
In September 2009, the European Working Group for Gaucher Disease (EWGGD) created an Emergency Treatment Program to allocate Cerezyme to patients at high risk for the development of progressive disease or complications. The program ran until the end of December 2009.
Due to the prolonged reduced allocations and based on a recommendation of the European Medicines Agency (EMA), the CETP has been reinstated. A limited amount of product allocated to the regions mentioned above will be reserved for this program. The reserved enzyme that is not used within a month will immediately flow back to the general pool. This will be assessed by Genzyme on a monthly basis in consultation with the coordinator, to allow optimal use of allocations for these regions.
If there is insufficient enzyme within the allocation designated to the institution/country to treat patients at high risk for the development of progressive disease or complications, the treating physician may apply for additional Cerezyme, for that patient, to the CETP.
Included countries: Albania, Algeria, Austria, Azerbajdian, Belarus, Belgium, Bosnia-Herzogovina, Bulgaria, Croatia, Czech Republic, Denmark, Estonia, Finland, France, Germany,Greece, Hungary, Iceland, Iran*, Jordan*, Ireland, Israel, Italy, Kazakhstan, Kosovo, Kuwait, Lebanon*, Latvia, Lithuania, Luxembourg, Macedonia, Middle East, Montenegro, Morocco, Netherlands,Norway,Oman, Poland, Portugal, Qatar, Romania, Russia, Saudi-Arabia*, Serbia, Slovakia, Slovenia, Spain, Sweden, Switzerland, Tunisia, Turkey, UAE*, UK, Ukraine.
The estimated shipment duration is in general 5 working days, except for countries marked with * (about 1 month) and countries in italic (uncertain shipment duration because of import procedures).
Patients at high risk for the development of progressive disease or complications: The following criteria were developed by EWGGD:
A. Infants, children, adolescents
B. Adult patients (either type I or III) with:
- Exacerbation of disease while on dose reduction/dose interruption
- platelet count < 20.000/μl
- thrombocytopenia and bleeding
- symptomatic anemia
- severe co-morbidity requiring imiglucerase treatment, such as:
- need for chemotherapy
- 2. condition that puts a patient at risk for bleeding, e.g. cirrhosis, major surgery, that cannot be postponed for 3-6 months
- 3. lung disease caused by Gaucher cell infiltration
- 4. new acute bone event during last 12 months
C. Pregnant women with symptomatic Gaucher disease
Recommendations by EWGGD for monitoring during imiglucerase reduction or cessation of enzyme replacement therapy and during the recovery period:
- clinical examination and history at least every two months
- complete blood count at least every two months
- plasma sample for biomarker analysis such as chitotriosidase at least every two months
- skeletal manifestations and organ volumes.
For the assessment of chitotriosidase, it was recommended to employ local laboratory facilities for early evaluations, using percentage increase from baseline as a possible indication of deterioration. For an analysis of absolute values in the entire group, the stored plasma sample can afterwards be assayed at a central facility to correct for differences between labs.
Results of all follow-up studies need to be carefully recorded and all were encouraged to submit these data to the Gaucher Registry of the International Collaborative Gaucher Group (ICGG) and/or national registries.
- The treating physician will submit a request form with details on the patient to the AMC
- Approval / response letters to the physicians will be provided by the Program coordinator as soon as possible but no later than 10 days after applications have been received.
This program does not guarantee that Cerezyme supply will be available for all emergency cases. The CETP board will review and discuss applications on a weekly basis. If necessary, additional information can be requested. After approval, Genzyme will ship the necessary extra Cerezyme as soon as possible, preferably within 1-2 weeks.
For logistical reasons, a prerequisite to the granting of a request is that import of imiglucerase should be feasible within 1 month.
CETP board members:
Carla E. M. Hollak
Department of Endocrinology and Metabolism,
Academic Medical Centre,
Shaare Zedek Medical Centre,
Department of Medicine,
University of Cambridge,
Centro di Coordinamento Regionale per le Malattie Rare Ospedale Universitario S. Maria della Misericordia,
Program coordinator and assistant:
Marieke Biegstraaten and Lydia Veerhuis,
CETP coordinating centre contact details:
Fax: +31 20 691 7682
Websites: www.gaucher.org.uk/index.php www.amc.nl/CETP www.esgld.org