{"id":37953,"date":"2022-05-12T15:15:07","date_gmt":"2022-05-12T19:15:07","guid":{"rendered":"https:\/\/wfh.org\/general-plenary-vp-medical-address-with-glenn-pierce\/"},"modified":"2026-03-12T10:08:22","modified_gmt":"2026-03-12T14:08:22","slug":"general-plenary-vp-medical-address-with-glenn-pierce","status":"publish","type":"post","link":"https:\/\/wfh.org\/fr\/article\/general-plenary-vp-medical-address-with-glenn-pierce\/","title":{"rendered":"General Plenary: VP Medical address with Glenn Pierce"},"content":{"rendered":"\t\t
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Pierce began his presentation by showing a slide which indicates that the global disparity in treatment has shown little improvement over time. The Americas and Europe account for 31% of the world population but use 78% of the available factor FVIII treatment product. There is evidence that about eight times more factor is needed to bring global use to North American and European levels. This shows, he said, that much would have to change for us to get to Treatment for All using existing technologies.<\/p>\n

Furthermore, Treatment for All as per WFH guidelines may not be possible, said Pierce, with existing manufacturing technologies and capacities. Gene therapy promises an optimal \u201conce and done\u201d cure but there are still many issues at play. While hemophilia B may have a first-generation cure, there is less good news for hemophilia A. Molecular therapies still represent the most likely solution to a cure. Importantly, however, solutions for ensuring that most of world can access state of the art treatment are elusive.<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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\u201cThere are cost-effective solutions that can help address the disparity [in care around the world] short of gene therapy. But gene therapy can fully address this disparity because it can get us to curative therapies.\u201d<\/h2>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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\u2014 Glenn Pierce, MD, PhD, Vice President Medical, WFH<\/p>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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Phase 3 trails show that efficacy has been established for both hemophilia A and B treatments. Also, durability for hemophilia A treatments is likely less than 10 years, while durability for hemophilia is likely established. When compared to hemophilia A, hemophilia B gene therapy appears to offer long term durability of response, and appears to have less toxicity; variability in response is high, but not as much as with hemophilia A. In the context of high- or lower-income countries, the story is more complicated, since high income countries have choice of state-of-the-art therapies, and gene therapy will be one option. Meanwhile, lower income countries will have limited to no choice, and gene therapies offer a different benefit\/risk scenario. He also said that when developing a drug, it\u2019s important to attempt to predict the economic level of different countries in even to ten years\u2014when the drug will actually go to market. This is because the economic level of countries is changing fast, and researchers need to ensure that their drug will \u201cfit\u201d in a future world.<\/p>\n

Glenn Pierce continued by saying that we need to think about the Phase 3 gene therapy results in context of other treatment options. For example, although extended half-life (EHL) and bispecific antibody products are available through the WFH Humanitarian Aid Program, there remains a huge gap to achieve Treatment for All. An analysis of the risk\/benefit of gene therapy depends on the resources available for treatment. He went on to caution that although gene therapy has great benefits, research has been going on for 25 years and all AAV clinical trials to date have failed to achieve FDA approval\u2014with only two exceptions. Despite all of this, however, Pierce said the potential is too great and we can\u2019t give up.<\/p>\n

When efficacy is unreliable, and safety issues arise, understanding how a product works is critical. Pierce says this is what is required for current AAVs in clinical trials, but it\u2019s not happening because of the rush to the clinic with clinical trials that are a variation on a theme. Achieving consistency, reliability, durability through understanding AAV biology, he says, will open the door toward Treatment for All.<\/p>\n

Pierce said he remains cautiously optimistic about gene therapy. He believes that it will revolutionize medical care by offering cures\u2014not treatments\u2014for all with bleeding disorders. Besides the current research challenges of gene therapy, there are still other hurdles. For example, how do we pay for treatment? Also, we need low burden therapies. There are several possible solutions. These include next generation AAV gene therapy; subcutaneously delivered, low frequency protein therapies; and new technologies, such as gene editing, non-viral delivery, etc.<\/p>\n

Glenn Pierce concluded his plenary by saying that curative therapies for hemophilia A and B treatments are in different places. Also, people with von Willebrand disease (VWD), rare bleeding disorders (RBD), children, and women and girls with bleeding disorders (WGBD) are still only at the very early stages of care and research progress. Our collective futures, he said, depend on further success of next generation medicines, most likely genetic (DNA, RNA)-based and curative.<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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WFH 2022 World Congress<\/h3>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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\n\t\t\t\t\tMay 8 to 11, 2022<\/span>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Register now!<\/strong><\/a><\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<\/div>\n\t\t","protected":false},"excerpt":{"rendered":"

At the WFH 2022 World Congress in Montreal, Canada, Glenn Pierce, MD, PhD, Vice President Medical at the World Federation of Hemophilia (WFH), gave his plenary address, \u201cLooking Forward with the View of Learnings of the Past\u201d. In his presentation, Pierce addressed where we are, and where we need to go to accomplish our shared vision of Treatment for All.<\/p>\n","protected":false},"author":3,"featured_media":37855,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"inline_featured_image":false,"footnotes":""},"categories":[169,33],"tags":[],"search-category":[85],"class_list":["post-37953","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-events-2","category-wfh-world-congress","search-category-article"],"yoast_head":"\nGeneral Plenary: VP Medical address with Glenn Pierce | FMH - F\u00e9d\u00e9ration mondiale de l\u2019h\u00e9mophilie<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/wfh.org\/fr\/article\/general-plenary-vp-medical-address-with-glenn-pierce\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"General Plenary: VP Medical address with Glenn Pierce\" \/>\n<meta property=\"og:description\" content=\"At the WFH 2022 World Congress in Montreal, Canada, Glenn Pierce, MD, PhD, Vice President Medical at the World Federation of Hemophilia (WFH), gave his plenary address, \u201cLooking Forward with the View of Learnings of the Past\u201d. 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