{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,5,13]],"date-time":"2025-05-13T15:48:15Z","timestamp":1747151295770,"version":"3.40.5"},"reference-count":20,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2019,10,31]],"date-time":"2019-10-31T00:00:00Z","timestamp":1572480000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pharmacoepidemiology and Drug"],"published-print":{"date-parts":[[2020,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>The alglucosidase alfa (Myozyme\u00ae) Safety Information Packet (\u201cprevious SIP\u201d) was updated to improve readability and content (\u201cupdated SIP\u201d). We compared the previous and updated SIPs.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A two\u2010wave pre\u2010post multicountry survey was conducted among health care professionals (HCPs) who prescribed or monitored patients on alglucosidase alfa in the largest European Union (\u201cEU5\u201d) countries and Poland. Wave (W) 2 started 15\u00a0months after completion of W1 and the implementation of the updated SIP. Changes between the waves were analysed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Forty\u2010six HCPs (34 physicians\/12 nurses) participated in W1 and 52 in W2 (42 physicians\/10 nurses); 22 participated in both waves. Nonsignificant differences were observed between waves 1 and 2 for awareness (75.6% in W1 and 82.4% in W2) and receipt (77.7% in W1 and 74.5% in W2) of the SIP, reading (88.6% in W1 and 89.5% in W2) and usage (88.2% in W1 and 89.5% in W2) among receivers of the SIP, or the overall knowledge about immunological testing (61.1% in W1 vs 55.1% in W2). Frequency of performance of immunological testing was significantly higher in W2 than in W1 (50.3% vs 34.4%; <jats:italic>P<\/jats:italic>\u00a0=\u00a0.024) with a tendency for increases in the appropriate performance of all types of testing in W2.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Both versions of the SIP showed relatively high awareness, receipt, reading, and usage, with an overall trend for most measures to improve numerically in W2. The updated SIP did not require further changes.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pds.4905","type":"journal-article","created":{"date-parts":[[2019,10,31]],"date-time":"2019-10-31T09:46:44Z","timestamp":1572515204000},"page":"103-110","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Pre\/post effectiveness evaluation of updated additional risk minimisation measures for an orphan disease: Myozyme (alglucosidase alfa) Safety Information Packet"],"prefix":"10.1002","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4294-6462","authenticated-orcid":false,"given":"Esther","family":"Artime","sequence":"first","affiliation":[{"name":"Pharmacoepidemiology Oxon Epidemiology SL  Madrid Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Irene","family":"Shui","sequence":"additional","affiliation":[{"name":"Epidemiology and Benefit\u2010risk Evaluation Sanofi  Bridgewater Township NJ USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ignacio","family":"Mendez","sequence":"additional","affiliation":[{"name":"Epidemiology Oxon Epidemiology SL  Madrid Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stephanie","family":"Tcherny\u2010Lessenot","sequence":"additional","affiliation":[{"name":"Global Pharmacovigilance and Epidemiology Sanofi  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pierre","family":"D'Arbigny","sequence":"additional","affiliation":[{"name":"Risk Management unit, Global Pharmacovigilance Sanofi  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Noelia","family":"Alfaro","sequence":"additional","affiliation":[{"name":"Qualitative Research Oxon Epidemiology SL  Madrid Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6950-2322","authenticated-orcid":false,"given":"Nawab","family":"Qizilbash","sequence":"additional","affiliation":[{"name":"London School of Hygiene and Tropical Medicine, Epidemiology Oxon Epidemiology SL  London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2019,10,31]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ncl.2014.04.010"},{"key":"e_1_2_9_3_1","unstructured":"European Medicines Agency.Guideline on good pharmacovigilance practices (GVP)\u2014Module XVI\u2014Risk minimisation measures: selection of tools and effectiveness indicators (Rev 2). 2017."},{"key":"e_1_2_9_4_1","unstructured":"European Medicines Agency and Heads of Medicines Agencies.Guideline on good pharmacovigilance practices (GVP) Module VIII\u2014Post\u2010authorisation safety studies. 2013."},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.5688\/aj720111"},{"key":"e_1_2_9_6_1","doi-asserted-by":"crossref","unstructured":"American Association for Public Opinion Research.Standard definitions final dispositions of case codes and outcome rates for surveys. 2016.https:\/\/doi.org\/10.1038\/nsmb.2010.","DOI":"10.1038\/nsmb.2010"},{"key":"e_1_2_9_7_1","unstructured":"Tysabri TOUCH Prescriber Program Risk Management Program (RiskMAP) Survey. Available at:https:\/\/www.rtihs.org\/sites\/default\/files\/B.Calingaert_ICPEPoster.pdf."},{"key":"e_1_2_9_8_1","unstructured":"European Medicines Agency and Heads of Medicines Agencies.Guideline on good pharmacovigilance practices (GVP) Module XVI\u2014Risk minimisation measures: selection of tools and effectiveness indicators (Rev 1). 2014."},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/pds.3596"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.13165"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40264-017-0619-x"},{"key":"e_1_2_9_12_1","unstructured":"ISPE Whitepaper\u2014Evaluating the Effectiveness of Additional Risk Minimisation Measures via Surveys in Europe: Challenges and Recommendations."},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40264-013-0126-7"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40290-017-0184-8"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/pds.3494"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.13469"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/pds.3305"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1080\/14740338.2019.1612875"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40264-017-0604-4"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/pds.4434"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1002\/pds.3596"}],"container-title":["Pharmacoepidemiology and Drug Safety"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pds.4905","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/pds.4905","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pds.4905","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,8]],"date-time":"2023-09-08T07:46:23Z","timestamp":1694159183000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pds.4905"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,10,31]]},"references-count":20,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2020,1]]}},"alternative-id":["10.1002\/pds.4905"],"URL":"https:\/\/doi.org\/10.1002\/pds.4905","archive":["Portico"],"relation":{},"ISSN":["1053-8569","1099-1557"],"issn-type":[{"type":"print","value":"1053-8569"},{"type":"electronic","value":"1099-1557"}],"subject":[],"published":{"date-parts":[[2019,10,31]]},"assertion":[{"value":"2019-03-22","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-09-09","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-10-31","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}