Osteogenesis imperfecta patients in Romania
Osteogenesis Imperfecta (OI), also called Brittle Bone Disease, is responsible for varying degrees of skeletal fragility in patients, and minimal trauma is sufficient to cause fractures and bone deformities. It was demonstrated that bisphosphonates can have beneficial effects in children suffering from OI1, (who can have 10 to 100 fractures a year) by dramatically reducing the number of fractures they suffer. Pamidronate (from the bisphosphonates class) is marketed in Europe for the treatment of moderate to severe Paget disease and, in conjunction with antineoplastic therapy, for the treatment of osteolytic bone metastases of breast cancer and osteolytic lesions of multiple Myeloma. Pamidronate is also widely administered to OI children on an off-label basis. In Romania, until the beginning of 2006, Pamidronate (Aredia®) could be prescribed by any family physician; it was available in pharmacies, free of charge, and administered to patients by infusion in hospitals. However, in March 2006, the NHIH (National House of Health Insurances, Romanian Ministry for Health) removed Aredia® from the free drug list and decided that it would only be available in hospitals. 'When we asked authorities why they took this action,' says Florin Dananau, father of a young girl suffering from OI, ‘they said it was because the drug could only be administered by infusion in hospitals, and that no one should get the drug outside healthcare facilities.' The cost of the drug is so high that patients from Romania, where the average monthly salary is 200 euros, could not afford it. Florin Dananau decided to bite the bullet and managed to organise a meeting with representatives from the NHIH; he also filed a complaint with the Ministry for Health and contacted OIFE (Osteogenesis Imperfecta Federation Europe). The response from the NHIH was to reintroduce Aredia® in the free drug list; the Bucharest Health Insurance House (BHIH) also committed to provide the necessary funds to the hospital for OI children from Bucharest (Grigore Alexandrescu Children's Hospital) to buy the drug to be administered to OI children. ‘All children suffering from OI, including new cases, have been treated since,' says Florin Dananau. A great example of the impact patients can have on the healthcare system when they take action!
Ute Wallentin, the President of OIFE, had turned to Eurordis for help. Since the 1999 adoption of the EU Regulation on Orphan Medicinal Products, Eurordis has been advocating for availability and affordability of orphan drugs for all rare disease patients in Europe; it conducts a regular survey to assess and compare availability of orphan drugs to patients in the EU, which shows that the situation of patients in new and candidate Member States is particularly difficult. Eurordis wrote a letter to the Romanian Minister for Health, asking that Aredia® be made available to all patients and be reimbursed.
However, to this day, the situation of OI patients in Romania remains worrisome. ‘We don't know if the BHIH will provide the necessary funds in 2007,' says Florin Dananau. ‘There are also very few hospitals in Romania that have both skilled personnel and the drug to administer it to patients. Without mentioning the general lack of knowledge on OI!' ‘But the situation of OI patients is just as bad in other countries,' says Ute Wallentin. Towards the end of 2006 we were saddened to hear that OI adults from Belgium had lost access to free treatments. And OI children in Germany are in danger of losing access to treatments too if the planned budget cuts are put into effect. Countries like Portugal or France are also facing a difficult situation!'
‘The fact that the drug is administered on an off-label basis makes things worse. A possible strategy suggested by Eurordis to overcome the barriers encountered by OI patients in access to treatments is that pharmaceutical companies interested in developing bisphosphonates for OI seek Orphan Drug designation. The designation is a recognition of the potential benefit of bisphosphonates in treating OI; it provides advantages such as 10 year market exclusivity and free-of-charge access to protocol assistance, which are extremely valuable in conditions with very few patients; and once the marketing authorisation is granted, the orphan drug status at EU level facilitates the drug's inscription in the national reimbursement lists. Meanwhile, patients can benefit from off-label use of the drug, even if the issue of free access still remains. Sharing experience and fighting together for free access to orphan drugs at the European level is the best way forward.
1Pamidronate Treatment of Severe Osteogenesis Imperfecta in Children under 3 Years of Age (H. Plotkin, F. Rauch, N. J. Bishop et al. The Journal of Clinical Endocrinology & Metabolism Vol. 85, No. 5 2000); Beneficial effect of long term intravenous bisphosphonate treatment of osteogenesis imperfecta (E Åström and S Söderhäll. Arch. Dis. Child. 2002;86;356-364); Cyclic administration of pamidronate in children with severe osteogenesis imperfecta. F. Glorieux, N. Bishop, H. Plotkin. N Engl J Med 339 :947-52, 1998
This article was previously published in the March 2007 issue of our newsletter.
Author: Jerome Parisse-Brassens
Photo credits: patient © Carlos/www.ostegeonesis.info; Romania © http://europa.eu