How can we, humans, look at our relationship to nature differently? In season three of Going Wild, on top of stories about animals, we invite you to journey through the entire ecological web — from the tiniest of life forms to apex predators — alongside the scientists, activists and adventurers who study it. Wildlife biologist and host Dr. Rae Wynn-Grant has been studying wild animals in their natural habitats all over the world for years. Our award-winning podcast takes you inside the hidde ...
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เนื้อหาจัดทำโดย Sano Genetics เนื้อหาพอดแคสต์ทั้งหมด รวมถึงตอน กราฟิก และคำอธิบายพอดแคสต์ได้รับการอัปโหลดและจัดหาให้โดยตรงจาก Sano Genetics หรือพันธมิตรแพลตฟอร์มพอดแคสต์ของพวกเขา หากคุณเชื่อว่ามีบุคคลอื่นใช้งานที่มีลิขสิทธิ์ของคุณโดยไม่ได้รับอนุญาต คุณสามารถปฏิบัติตามขั้นตอนที่แสดงไว้ที่นี่ https://th.player.fm/legal
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EP 145: Navigating rare disease drug development regulations with Daniel O’Connor
MP3•หน้าโฮมของตอน
Manage episode 431713956 series 2631947
เนื้อหาจัดทำโดย Sano Genetics เนื้อหาพอดแคสต์ทั้งหมด รวมถึงตอน กราฟิก และคำอธิบายพอดแคสต์ได้รับการอัปโหลดและจัดหาให้โดยตรงจาก Sano Genetics หรือพันธมิตรแพลตฟอร์มพอดแคสต์ของพวกเขา หากคุณเชื่อว่ามีบุคคลอื่นใช้งานที่มีลิขสิทธิ์ของคุณโดยไม่ได้รับอนุญาต คุณสามารถปฏิบัติตามขั้นตอนที่แสดงไว้ที่นี่ https://th.player.fm/legal
0:00 Intro to The Genetics Podcast
01:00 Welcome to Daniel
02:04 Defining rare disease in the age of personalized medicine
04:57 Key touchpoints with the Medicines and Healthcare products Regulatory Agency (MHRA) when developing a new medicine
09:27 Improvements over the course of Daniel’s career when it comes to incentivizing and making the path to developing therapeutics for rare diseases easier
12:03 The importance of orphan drug designation and what it means for treatment development
14:28 The unique challenges within clinical trial design for rare diseases, including sample size and ethical considerations such as control arms
17:22 How to quantify the scientific rigor of ultra-rare studies and small population research
18:56 The technologies which have been most impactful during the past 20 years and those which Daniel predicts will be the most impactful going forward
22:14 Rare Therapies Launchpad: Building the infrastructure and policies to ensure patients with ultra-rare mutations are connected with potentially relevant therapies
23:19 Working with the International Rare Disease Consortium and the importance of coordinating on an international level
26:30 How to enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of seeking medical attention
29:28 How a career move from working with the MHRA to The Association of the British Pharmaceutical Industry (ABPI) has impacted Daniel’s perspective
32:17 The changes the UK could make to further improve its position as one of the best places in the world to do research
35:33 Creating greater flexibility within regulatory systems to better enable use of preventive treatments
37:26 The challenges of mitigating the uncertainties of preventive treatments and benefit-risk balance over time
38:39 Considering scientific validity versus cost of therapy when developing new preventative treatments
41:17 Closing remarks
Please consider rating and reviewing us on your chosen podcast listening platform!
Find out more:
Find Daniel on LinkedIn
186 ตอน
MP3•หน้าโฮมของตอน
Manage episode 431713956 series 2631947
เนื้อหาจัดทำโดย Sano Genetics เนื้อหาพอดแคสต์ทั้งหมด รวมถึงตอน กราฟิก และคำอธิบายพอดแคสต์ได้รับการอัปโหลดและจัดหาให้โดยตรงจาก Sano Genetics หรือพันธมิตรแพลตฟอร์มพอดแคสต์ของพวกเขา หากคุณเชื่อว่ามีบุคคลอื่นใช้งานที่มีลิขสิทธิ์ของคุณโดยไม่ได้รับอนุญาต คุณสามารถปฏิบัติตามขั้นตอนที่แสดงไว้ที่นี่ https://th.player.fm/legal
0:00 Intro to The Genetics Podcast
01:00 Welcome to Daniel
02:04 Defining rare disease in the age of personalized medicine
04:57 Key touchpoints with the Medicines and Healthcare products Regulatory Agency (MHRA) when developing a new medicine
09:27 Improvements over the course of Daniel’s career when it comes to incentivizing and making the path to developing therapeutics for rare diseases easier
12:03 The importance of orphan drug designation and what it means for treatment development
14:28 The unique challenges within clinical trial design for rare diseases, including sample size and ethical considerations such as control arms
17:22 How to quantify the scientific rigor of ultra-rare studies and small population research
18:56 The technologies which have been most impactful during the past 20 years and those which Daniel predicts will be the most impactful going forward
22:14 Rare Therapies Launchpad: Building the infrastructure and policies to ensure patients with ultra-rare mutations are connected with potentially relevant therapies
23:19 Working with the International Rare Disease Consortium and the importance of coordinating on an international level
26:30 How to enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of seeking medical attention
29:28 How a career move from working with the MHRA to The Association of the British Pharmaceutical Industry (ABPI) has impacted Daniel’s perspective
32:17 The changes the UK could make to further improve its position as one of the best places in the world to do research
35:33 Creating greater flexibility within regulatory systems to better enable use of preventive treatments
37:26 The challenges of mitigating the uncertainties of preventive treatments and benefit-risk balance over time
38:39 Considering scientific validity versus cost of therapy when developing new preventative treatments
41:17 Closing remarks
Please consider rating and reviewing us on your chosen podcast listening platform!
Find out more:
Find Daniel on LinkedIn
186 ตอน
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