A Martini

L-Dopa decreases cutaneous nociceptive inhibition of motor activity in Parkinson's disease.

Objectives– To estimate changes in motor inhibitory mechanisms at the spinal level in Parkinsons disease (PD) patients by measuring cutaneous silent responses to nociceptive stimuli in the course of L‐Dopa therapy. Material and methods– Fourteen patients with idiopathic PD (Group 1) and 13 patients with other forms of parkinsonism (Group 2) participated in the study. The cutaneous silent period (CSP) from the hand and clinical scores (UPDRS, part III) were measured “off” therapy (T0), after a single dose of L‐Dopa (T1) and 3 months after the beginning of L‐Dopa daily therapy (T2). Results– At T0 the duration of the CSP was significantly prolonged in Group 1 and Group 2. At T1 and T2 the mean duration of the CSP significantly decreased in Group 1 (P < 0.05) and a significant correlation was found between the shortening of the CSP and the improvement of rigidity and bradikynesia in the upper limb. Conclusions– Our findings show that L‐Dopa decreases the cutaneous nociceptive inhibition of motor activity in PD patients. CSP may be useful to assess L‐Dopa responsiveness during the clinical course of PD.

The RaDiCEA Project: cost of illness (COI) analysis applied to Cryopyrin Associated Periodic Syndromes (CAPS)

When a disease affects only a few individuals in each country (ultra-orphan disease), it can be very hard to establish the costs of illness (COI) and the cost-effectiveness (CE) of treatments (ultra-orphan drugs). Conventional methods for COI and CE of drugs for common conditions do not apply, and additional factors need to be considered. As expensive medications (biologicals) show promising results, it becomes crucial to have detailed information on as many patients as possible.

Cost-effectiveness analysis and prevention effects of ultra-orphan drugs for rare diseases: an in silico model applied to Cryopyrin Associated Periodic Syndromes (CAPS)

8th International Congress of Familial Mediterranean Fever and Systemic Autoinflammatory Diseases

PW02-027 - CAPS and cost-effectiveness analysis project

Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening. Low patient numbers make it difficult for pharmaceutical companies to recoup research and development costs, and consequently these medicines are generally very expensive on a per patient basis. European Union (EU) regulations promote the development of orphan drugs; but to contain costs, EU healthcare systems will increasily need the cost-effectiveness analysis (CEA) of therapies when deciding if they should be funded. Conventional methods for CEA of drugs for common conditions do not apply to ultra-orphan drugs; therefore, additional factors need to be considered.