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- Heard, J. M., et al.
(author)
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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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In: Orphanet Journal of Rare Diseases. - : Springer Science and Business Media LLC. - 1750-1172. ; 15:1
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Journal article (peer-reviewed)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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| 3. |
- Gårdfeldt, Katarina, 1959, et al.
(author)
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Evasion of Mercury from coastal and open waters of the Atlantic ocean and the Mediterranean sea
- 2003
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In: Atmospheric Environment. - 1352-2310 .- 1873-2844. ; 37:Suppl 1
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Journal article (peer-reviewed)abstract
- Dissolved gaseous mercury (DGM) was measured in coastal Atlantic seawater and in the Mediterranean Sea. The Atlantic measurements were performed during September 1999 at the Mace Head Atmospheric Research Station, situated on the Irish west coast. The measurements in the Mediterranean Sea were made along a 6000 km cruise path from 14 July to 9 August 2000 in the framework of the Med-Oceanor project. Total gaseous mercury (TGM) concentrations in air were continuously measured with a 5 min time resolution using an automated mercury analyser (Tekran 2537A) during both expeditions. Paired TGM and DGM samples from all campaigns showed that the surface water was supersaturated with elemental mercury. The mercury evasion was estimated using a gas exchange model (J. Geophys. Res. 97 (1992) 7373), which uses salinity, wind speed and water temperature as independent parameters. The predicted average mercury evasion from the coastal Atlantic water was 2.7 ng m−2 h−1 implying that the concentration of TGM in the Atlantic air is enhanced by mercury evasion from the sea. Measurements in different regions of the Mediterranean Sea showed spatial variations in DGM concentrations. The highest DGM concentration (90 pg l−1) was observed at a location in the Strait of Sicily (37°16N 11°52E). The mercury evasion in the eastern sector of the Mediterranean Sea (area: 32–36°N, 17–28°E) was generally higher (7.9 ng m−2 h−1) than that observed in the Tyrrhenian Sea (4.2 ng m−2 h−1) or in the western sector (2.5 ng m−2 h−1) (areas: 38–42°N, 8–13°E and 38–41°N, 7–8°E, respectively). Estimations of mercury evasion were also made at Mediterranean coastal sites using a dynamic chamber technique. In addition, a newly developed method making continuous in situ DGM measurements possible was tested.
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