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Implemented in 2012, the most recent of these is the breakthrough therapy designation (\n                    <jats:styled-content style=\"fixed-case\">BTD<\/jats:styled-content>\n                    ). The purpose of this article was to review the evidence surrounding approval of medications with nononcology indications approved with the\n                    <jats:styled-content style=\"fixed-case\">BTD<\/jats:styled-content>\n                    designation from 2012 to 2016. Fifteen medications were identified for eight conditions, ranging from conditions that are relatively common, such as chronic hepatitis C infection, to those that are extremely rare, such as lysosomal acid lipase deficiency. The quality of evidence behind these approvals was highly heterogeneous. Much remains unknown about the safety and efficacy of many agents approved through the\n                    <jats:styled-content style=\"fixed-case\">BTD<\/jats:styled-content>\n                    . Health care professionals should be aware of these limitations to better educate patients and other providers appropriately.\n                  <\/jats:p>","DOI":"10.1002\/phar.2167","type":"journal-article","created":{"date-parts":[[2018,7,25]],"date-time":"2018-07-25T02:10:39Z","timestamp":1532484639000},"page":"967-980","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":23,"title":["<scp>FDA<\/scp>\n                    Breakthrough Therapy Designation: Evaluating the Quality of the Evidence behind the Drug Approvals"],"prefix":"10.1002","volume":"38","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6402-0260","authenticated-orcid":false,"given":"Megan C.","family":"Herink","sequence":"first","affiliation":[{"name":"Department of Pharmacy Practice Oregon State University\/Oregon Health &amp; Science University College of Pharmacy Portland Oregon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2086-4493","authenticated-orcid":false,"given":"Adriane N.","family":"Irwin","sequence":"additional","affiliation":[{"name":"Department of Pharmacy Practice Oregon State University\/Oregon Health &amp; Science University College of Pharmacy Corvallis Oregon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gregory M.","family":"Zumach","sequence":"additional","affiliation":[{"name":"Department of Pharmacy Practice Oregon State University\/Oregon Health &amp; Science University College of Pharmacy Corvallis Oregon"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2018,9,10]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1602\/neurorx.1.3.307"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.e4348"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.h4633"},{"key":"e_1_2_6_5_1","volume-title":"Fast track, breakthrough therapy, accelerated approved, priority review","author":"U.S. Food & Drug Administration"},{"key":"e_1_2_6_6_1","unstructured":"U.S. Food and Drug Administration Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Researc (CBER).Guidance for Industry: Expedited Programs for serious Conditions \u2010 Drugs and Biologics. 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