EP 145: Navigating rare disease drug development regulations with Daniel O’Connor
August 1, 2024
In this episode, we’re joined by Daniel O’Connor, Director of Regulatory Policy and Early Access at The Association of the British Pharmaceutical Industry. Daniel previously spent almost 18 years at the Medicines and Healthcare products Regulatory Agency (MHRA) and over his career has specialized in improving and incentivizing pathways to rare disease treatment development.
Join Patrick and Daniel as they discuss orphan drug designation, the importance of international collaboration, and how unlocking new regulatory approaches to preventative medicine can help change the future of rare and ultra-rare conditions.
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