A French observational study of botulinum toxin use in the management of children with cerebral palsy: BOTULOSCOPE.

International audienceBACKGROUND: Dystonia and spasticity are common symptoms in children with Cerebral Palsy (CP), whose management is a challenge to overcome in order to enable the harmonized development of motor function during growth. AIM: To describe botulinum toxin A (BTX-A) use and efficacy as a treatment of focal spasticity in CP children in France. METHODS: This prospective observational study included 282 CP children mostly administered according to French standards with BTX-A in lower limbs. Realistic therapeutic objectives were set with parents and children together before treatment initiation and assessed using the Visual Analogue Scale (VAS). Child management was recorded and the efficacy of injections was assessed during a 12-month follow-up period by physicians (Modified Ashworth Scale, joint range of motion, Physician Rating Scale, Gillette Functional Assessment Questionnaire and Gross Motor Function Measure-66) and by patients/parents (Visual Analogue Scale). RESULTS: BTX-A treatment was administered in different muscle localizations at once and at doses higher than those recommended by the French Health Authorities. Children were treated in parallel by physiotherapy, casts and ortheses. Injections reduced spasticity and improved joint range of motion, gait pattern and movement capacity. Pain was reduced after injections. BTX-A administration was safe: no botulism-like case was reported. The log of injected children who were not included in the study suggested that a large population could benefit from BTX-A management. CONCLUSIONS: We showed here the major input of BTX-A injections in the management of spasticity in CP children. The results are in favor of the use of BTX-A as conservative safe and efficient treatment of spasticity in children, which enables functional improvement as well as pain relief

Biosimilars for the Management of Inflammatory Bowel Diseases: Economic Considerations

Biological drugs revolutionized the treatment of inflammatory bowel diseases (IBD) such as Crohn’s disease and ulcerative colitis. However, not all clinically eligible patients have access to biologicals due to significant costs and budget impact. Biosimilars are highly comparable to their originator product in terms of clinical efficacy and safety. Biosimilars are priced 15-75% lower than their reference product, which makes them a less costly alternative and is expected to offer better patients access to biologicals. The total projected cost savings are significant. If the achieved budget savings were used to cover more biological therapy, several additional IBD patients could be treated. Currently, the main barriers to the increasing uptake of biosimilars are the few incentives of the key stakeholders, while physicians’ and patients’ skepticism towards biosimilars seems to be changing. Over the coming years, biosimilars are expected to gain a growing importance in the treatment of IBD, contributing to a better access to treatment, improving population-level health gain and sustainability of health systems. This review summarizes the results of the literature on the economic considerations of biosimilars in IBD and the role of biosimilar infliximab in the treatment of IBD

Optimal use and cost-effectiveness of biologic therapies in inflammatory bowel disease

Inflammatory bowel diseases (IBD), namely Crohn's disease and ulcerative colitis, are burdened by high medical costs which are mostly dependent on hospital inpatient treatment. New biologic therapies, which target specific cytokines in the inflammatory cascade leading to the intestinal lesions, including tumor necrosis factor (TNF)-α, have revolutionized the management of IBD by offering a therapeutic chance to patients in whom conventional therapies failed. However, the relatively high costs of biologic drugs, together with their potential toxicity due to infections and malignancies, have led to debate regarding their indiscriminate use in IBD patients. The purpose of this review is to deal with the optimal use and cost-effectiveness of the two main monoclonal anti-TNF-α agents currently used in the management of IBD patients, i.e. the chimeric human/murine antibody infliximab and the fully human antibody adalimumab