Azacitidine access program for Belgian patients with myelodysplastic syndromes, acute myeloid leukemia or chronic myelomonocytic leukemia

Abstract

Objective:

Azacitidine (Vidaza) is approved in Europe for treatment of myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) with 20–30% bone marrow (BM) blasts, and chronic myelomonocytic leukemia (CMML) with 10–29% BM blasts and no myeloproliferative syndrome (i.e. <13.000/μL white blood cells). In Belgium, the azacitidine reimbursement process can take several months, and is often delayed at submission for medical assessment by the Belgian National Institute for Health and Disability Insurance of incomplete patient dossiers, due to disease complexity and classification, and administrative burden. We describe the Vidaza Access Program and its application to an initial 175 patients. Individual medical dossiers were reviewed for completeness to facilitate patient access to treatment in Belgium.

Methods:

A standardized anonymized patient information form is completed by the physician and sent for review to the Belgian Celgene Medical Department. The form is reviewed within three working days and, for complete dossiers, Celgene grants a financial guarantee for treatment with azacitidine. The patient can then be treated without the hospital being subjected to financial risk.

Results:

Between January 2013 and June 2014, 63 physicians (53 Belgian hospitals) recruited 175 patients. In total, 163 patient dossiers were approved by Celgene (120 MDS, 36 AML, and 7 CMML), of which 104 dossiers were also approved by the review committee and 49 have been waiting for a final decision for a median of 6 months; no information is currently available for the remaining 10. No dossiers approved by Celgene have been rejected by the review committee.

Conclusions:

The Celgene Vidaza Access Program offers support to healthcare professionals in the appropriate use of azacitidine. By facilitating the assessment of patient dossiers and providing a financial guarantee for prescribers and hospitals, treatment can be initiated more rapidly and patients may better benefit from azacitidine treatment.

Key words::

• Azacitidine
• Belgium
• Myelodysplastic syndromes
• Reimbursement

Transparency

Declaration of funding

Celgene Corporation, Summit, NJ, USA was the funding source and was involved in all stages of the program. Celgene Corporation also took in charge all costs associated with the development and the publishing of the present manuscript. The authors had full access to the data and are fully responsible for all content and editorial decisions for this manuscript.

Declaration of financial/other relationships

P.M. has disclosed that he has no significant relationships with or financial interests in any commercial companies related to this study or article. S.M. has disclosed that he has received honoraria from Celgene and is serving on Celgene’s advisory boards. D.S. has disclosed that he has received honoraria from Celgene, is serving on Celgene’s advisory boards and is a member of Celgene’s speaker’s bureau. He is also a member of the RIZIV/INAMI review committee for Vidaza. A.G., V.V. and H.P. have disclosed that they are employees and stock holders of Celgene.

CMRO peer reviewers on this manuscript have received an honorarium from CMRO for their review work. Peer reviewer 1 has no relevant financial or other relationships to disclose. Peer reviewer 2 is a CMRO Editorial Board member, but has no relevant financial or other relationships to disclose.

Acknowledgments

The authors thank Urszula Miecielica, PhD and Iudit Filip, PhD (XPE Pharma & Science) for providing medical writing services and editorial support in preparing this manuscript.

Notes

*Vidaza is a registered trademark of Celgene Corporation, Summit, NJ, USA

*Vidaza is a registered trademark of Celgene Corporation, Summit, NJ, USA