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- Heard, J. M., et al.
(författare)
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Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network
- 2020
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Ingår i: Orphanet Journal of Rare Diseases. - : Springer Science and Business Media LLC. - 1750-1172. ; 15:1
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Tidskriftsartikel (refereegranskat)abstract
- Background The European Medicine Agency granted marketing approval to 164 orphan medicinal products for rare diseases, among which 28 products intended for the treatment of hereditary metabolic diseases. Taking advantage of its privileged connection with 69 healthcare centres of excellence in this field, MetabERN, the European Reference Network for hereditary metabolic diseases, performed a survey asking health care providers from 18 European countries whether these products are available on the market, reimbursed and therefore accessible for prescription, and actually delivered in their centre. Results Responses received from 52 centres (75%) concerned the design of treatment plans, the access to marketed products, and the barriers to delivery. Treatment options are always discussed with patients, who are often involved in their treatment plan. Most products (26/28) are available in most countries (15/18). Among the 15 broadly accessible products (88.5% of the centres), 9 are delivered to most patients (mean 70.1%), and the others to only few (16.5%). Among the 10 less accessible products (40.2% of the centres), 6 are delivered to many patients (66.7%), and 4 are rarely used (6.3%). Information was missing for 3 products. Delay between prescription and delivery is on average one month. Beside the lack of availability or accessibility, the most frequent reasons for not prescribing a treatment are patients' clinical status, characteristic, and personal choice. Conclusions Data collected from health care providers in the MetabERN network indicate that two-third of the orphan medicines approved by EMA for the treatment of hereditary metabolic diseases are accessible to treating patients, although often less than one-half of the patients with the relevant conditions actually received the approved product to treat their disease. Thus, in spite of the remarkable achievement of many products, patients concerned by EMA-approved orphan medicinal products have persistent unmet needs, which deserve consideration. The enormous investments made by the companies to develop products, and the high financial burden for the Member States to purchase these products emphasize the importance of a scrupulous appreciation of treatment value involving all stakeholders at early stage of development, before marketing authorization, and during follow up.
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| 2. |
- Cafiso, S., et al.
(författare)
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Zastosowanie i porównanie różnych metod analizy konfliktów ruchowych - Studium przypadku drogi 2+1
- 2021
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Ingår i: Archives of Civil Engineering. - 1230-2945. ; 67:1, s. 619-634
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Tidskriftsartikel (refereegranskat)abstract
- Safety Performance Functions and Crash Modification Factors are statistically-based prediction methods that require significant efforts and long periods in crash data collection. Traffic conflict studies can mitigate this issue using a short time survey to measure the number and severity of traffic conflicts, which are regarded as surrogate safety measures. Unfortunately, they are empirical studies that can be carried out only after the implementation of a treatment. The overall objective of the present research is to investigate the performance of different methods for conflict detection and classification, considering the observed conflicts on 2+1 roads in Poland. Observations were compared with conflicts detected in simulated environments. The latter include either the Agent-Based Microsimulation (ABM) approach, or the virtual reality simulation using a Driving Simulator (DS). Conflicts were detected and classified based on video recording and analysis of vehicle trajectories in the merging area of 2+1 roads. The studies focused only on lane-changing conflicts. Locations, Post Encroachment Time and Time to Collision values of observed conflicts between vehicles were subsequently identified. Observed conflicts were compared with the ones resulting from ABM and DS, to determine whether there is a correlation between them.
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