Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)

International audienceAbstractRare diseases are an important public health issue with high unmet need. The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. Despite this advancement, patients do not have universal access to these new medicines. There are many factors that affect OMP uptake, but one of the most important is the difficulty of making pricing and reimbursement (P&R) decisions in rare diseases. Until now, there has been little consensus on the most appropriate assessment criteria, perspective or appraisal process. This paper proposes nine principles to help improve the consistency of OMP P&R assessment in Europe and ensure that value assessment, pricing and funding processes reflect the specificities of rare diseases and contribute to both the sustainability of healthcare systems and the sustainability of innovation in this field. These recommendations are the output of the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL), a collaboration between rare disease experts, patient representatives, academics, health technology assessment (HTA) practitioners, politicians and industry representatives. ORPH-VAL reached its recommendations through careful consideration of existing OMP P&R literature and through a wide consultation with expert stakeholders, including payers, regulators and patients. The principles cover four areas: OMP decision criteria, OMP decision process, OMP sustainable funding systems and European co-ordination. This paper also presents a guide to the core elements of value relevant to OMPs that should be consistently considered in all OMP appraisals. The principles outlined in this paper may be helpful in drawing together an emerging consensus on this topic and identifying areas where consistency in payer approach could be achievable and beneficial. All stakeholders have an obligation to work together to ensure that the promise of OMP’s is realised

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Validation of the Global Allergy and Asthma European Network (GA2LEN) chamber for trials in allergy: Innovation of a mobile allergen exposure chamber

Background Field clinical trials of pollen allergy are affected by the impossibility of predicting and determining individual allergen exposure because of many factors (eg, pollen season, atmospheric variations, pollutants, and lifestyles). Environmental exposure chambers, delivering a fixed amount of allergen in a controlled environmental setting, can overcome these limitations. Environmental exposure chambers are currently already used in phase 2, 3, and even 4 trials. Unfortunately, few chambers exist in the world, and this makes it difficult to perform large, multicenter clinical trials. The new Global Allergy and Asthma European Network (GA2LEN) mobile exposure chamber is a step forward because the mobility of the chamber makes it convenient for patients to participate in clinical testing. Objective This study was made to validate the reproducibility, sensitivity, and specificity of the results obtained in the new GA2LEN chamber. Methods Seventy-two adult patients (19-61 years old) with allergic rhinitis with or without asthma caused by grass pollen were included in different clinical validation tests. Total symptom scores and total nasal symptom scores were recorded at time zero (0) and every 10 minutes during exposures, along with nasal and respiratory parameters. Results Exposure tests confirmed the reproducibility between subsequent runs and the sensitivity (P < .00001 vs patients exposed to placebo) and specificity (very low score in nonallergic subjects) in the GA2LEN chamber. No adverse reactions were recorded during the tests. Conclusions The mobility of the GA2LEN chamber provides a new, potentially effective, and safe way of generating reliable data in allergy multicenter clinical trials. © 2016 The Author

Epidemiology: Extent of the Problem

Peritoneal carcinomatosis (PC) most commonly represents local or regional evolution of an abdominal carcinoma. Sometimes it can be synchronous with the primary tumor (primary carcinomatosis) but more often is present as recurrent disease (metachronous or secondary) after first-line treatment of the originating tumor. Patients with tumors from colon, ovary, and stomach cancer are more likely to present with PC during their clinical course. Less frequently, other abdominal malignancies, such as uterus, pancreas, small bowel, biliary, or urinary tract, can involve the peritoneum. Tumors originating from the peritoneum itself are definitely rarer: mesothelioma, pseudomyxoma peritonei (PMP), primitive peritoneal carcinoma, and desmoplastic small-round-cell tumor. PC from extra-abdominal tumors, such as lung, breast, melanoma, or peritoneal sarcomatosis, is exceptional, and few epidemiological data are available on them. Statistical analysis of worldwide cancer incidence, prevalence, and mortality rate is available on GLOBOCAN 2012 [1]. In Italy, most epidemiological data are available in the reports from the Italian Association of Tumor Registries (AIRTUM) [2], which collects data regarding incidence, prevalence, and mortality rates from all local and regional tumor registries, covering at least 34% of total population. This data is considered a high-quality regional coverage by and international ranking system (GLOBOCAN 2012 rate B)