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Welcome to P4A Let’s Talk Rare, a monthly podcast highlighting the most important developments in the world of rare diseases orphan drug, cell and gene therapy, hosted by Georgie Rack and Owen Bryant of Partners For Access. To find out more about Partners For Access and our commitment to sustainable orphan drug access for patients with high unmet need, visit partners4access.com
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Welcome to this month's episode of the P4A Let’s Talk Rare podcast by Partners4Acess. Today, Georgie and Owen are joined by their colleagues Pina Haberl, Senior Director, and Sam Morrison, Executive Director at Partners4Access. Join us as we discuss the crucial role of patient experience data in drug development. The conversation explores the need …
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Welcome to this month's episode of Let’s Talk Rare: The Life Science Podcast brought to you by Partners4Access. Host Georgie records this episode at the World EPA Conference in Amsterdam. She holds a panel discussion with Juliette Sinclair-Spence, Sandrine Ruiz, Neil Grubert, and Seema Sondhi, and together we shared our experiences at the EPA confe…
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Welcome to this month's episode of the Let’s Talk Rare: The Life Science Podcast by Partners4Acess. Georgie and Owen are joined by Nick Meade, Head of Policy at Genetic Alliance, to discuss the challenges faced by rare patients in accessing life-saving medicines. Together they explore the EU joint HTA legislation coming into force in January 2025, …
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Hello and welcome to this special episode of Let’s Talk Rare: The Life Science Podcast as we celebrate 5 years of bringing you the podcast that is now the number one life science podcast across all platforms. We at Partners 4 Access want to thank every single one of our 35,000 subscribers and all the guests who have graced the podcast from the bott…
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In this podcast, Georgie, RJ, and Owen discuss revolutionizing patient outcomes and the power of digital health solutions. RJ, a healthcare industry expert, shares insights on the role of technology in improving patient care and the future of digital health. They also touch on RJ's involvement in triathlons and his passion for running. With a frien…
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Welcome to this month's episode of the Let’s Talk Rare podcast by Partners4Acess. Georgie and Owen are joined by Iola Forster, Head of Publications and Portfolio at Karger Publishers, to discuss the future of patient engagement and the role of technology in healthcare. Together, they cover the future of HCPs (healthcare practitioners) and patient e…
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Welcome to this month's episode of the Let’s Talk Rare podcast brought to you by Partners4Acess. Georgie and Owen are joined by Chloe Sheppard and Akshay Kumar to discuss the new EU HTA regulation set to be implemented in 2025. They explore the implications for drug developers of ATMPs, the attractiveness of the EU market, and the importance of pro…
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In this episode we will be talking to Alejandro (Alex) Dorenbaum, M.D., CMO of Reneo Pharmaceuticals. Alex discusses how the company is developing drugs for patients with #rare mitochondrial diseases, a high unmet disease with no current treatments available. Their lead candidate #Mavodelpar has recently completed enrolment for their pivotal STRIDE…
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P4A are joined by Xortx Therapeutics' CEO, Dr Allen Davidoff, as he discusses the journey from early development to potential launch for their novel therapy XRX-008 to treat patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD). Allen discusses the company vision for developing novel therapies for rare progressive kidney disorders, and…
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P4A host a special episode live from World Evidence, Pricing & Access Conference 2023 in Amsterdam. Join Georgie & Owen hosting live from World EPA Conference 2023. We will be bringing you speaker interviews, giving a flavour of the atmosphere and discussing some of the trends & key takeaways that came out of the conference. Max Rex: LinkedIn: http…
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In this episode of Let’s Talk Rare, as we commemorate World Rare Disease Day, Georgie and Owen are joined by Louise Fish and Dan Lewi to discuss all aspects surrounding rare diseases, from key challenges patients face in getting access to life-saving medicines, the clinical trial burden, EU pharmaceutical strategy and more. The topics covered inclu…
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The P4A team have hand-picked trends to look out for in 2023. This episode we are joined by special guest Neil Grubert who will be discussing the topics in further detail, alongside our P4A experts, Shrishti & Jodie. The topics covered are: EU Pharmaceutical Strategy, OMP Legislation, Joint HTA Assessments - cross-country HTA cooperation, Expansion…
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Did you miss the World Orphan Drug Congress Europe 2022, held in Sitges, Barcelona in November? If you did, don't worry P4A have you covered! This month's special podcast was part recorded live at the conference and part recorded back in London with members of the team that attended! We will be speaking to a few presenters & sponsors to discuss the…
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Winds of change are howling in Germany, with the draft healthcare bill now approved to stabilise SHI fund finances. What will manufacturers, with innovative orphan drugs and cell and gene therapies, launch strategies be? With the latest decision from Janssen to avoid the German market altogether for x2 Rare Oncology innovative drugs, will this be a…
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P4A’s 2 part -Patient Empowerment podcast series has been released. Our incredible panel of experts Laurence Woollard, Neil Bertelsen & Sophie Schmitz are back with us to carry on the discussion in part 2 on true patient empowerment and really looking at the WHY’s! Why should drug manufacturers involve & listen to and involve the patients. What are…
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This month we have a special 2-part series focused on Patient Empowerment, with special guests Laurence Woollard, On the Pulse, Neil Bertelsen, Independent Consultant & Sophie Schmitz, Managing Partner at P4A. This podcast is a MUST to listen too if you are a company looking to successfully develop and commercialize an orphan drug or ATMP The first…
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This month we have put together a slightly different episode with a twist. Georgie & Owen will take you behind the scenes at P4A and discuss 'What makes P4A an award-winning global market access consultancy?' Why we do what we do every day, and what it means to each and every member of staff. Pride, Passion & Partnership are the core values at P4A,…
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In this episode Chloe & Alexander will be starting the discussion with an overview of EU HTA regulation, looking at the harmonization debate and how this came about. We then discuss JCA evaluation methodology (e.g. comparator selection, acceptability of clinical evidence), the potential new role of individual country agencies and how the joint HTA …
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Part 1 featuring Victor Maertens, Government Affairs Manager at EUCOPE In this episode Victor and Aparna will be focusing on Advanced Therapy Medicinal Products (ATMPs). We will start by looking at the main challenges for manufacturers in the ATMP approval process and what is required from an HTA perspective. We then discuss EUCOPE’s position paper…
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In 2020 China’s pharmaceutical market passed an important milestone, a Deloitte report cited that China was expected to grow its pharmaceutical market volume by 2020 to $220 billion (USD) becoming the second largest market behind the US. The commercial potential is huge, so what do we know about the orphan drug (OD) potential in China? Fisentzos St…
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Over the last few years, the Middle East has piqued the interest of orphan drug manufacturers, and some have prioritised launching in this region before European markets. Akshay Kumar and Lavni Varyani discuss what is driving this behaviour, the orphan drug infrastructure within the region, and opportunities, barriers, and key considerations for ma…
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For this Rare Disease Day episode, Janet and Nick discuss a range of topics associated with the rare disease community and patient advocacy. They address new-born screening, patient registries, the UK rare disease framework, the NICE methods review and much more! Presenter: Georgie Rack, Communication Executive Contributors: Janet Bloor, CEO of Duc…
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Lots of anticipated changes are expected in the orphan drug, cell and gene therapy space and 2022 is going to be an exciting year. Listen to our latest podcast on the top trends in drug development, HTA and pricing from Akshay Kumar and Andrea Bernardini to help you navigate the year ahead. Presenter: Georgie Rack Speakers: Akshay Kumar & Andrea Be…
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Sophie Schmitz and Joanna Fernandes discuss bluebird bio’s innovative gene therapy Zynteglo and its withdrawal from the European market. Sophie and Joanna will be looking at the fall of Zynteglo from two sides: from a company perspective and from the perspective of the EU environment. Presenter: Georgie Rack, Communication Executive Contributors: S…
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This episode featuring James Mackay, President & CEO of Aristea Therapeutics. James provides insights on his experience with setting up a biotech and challenges associated, industry climate on spin off opportunities, Aristea’s pipeline, collaborations and US PRMA reforms. Presenter: Aparna Krishnan Contributors: James Mackay, President & CEO of Ari…
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In this episode P4A’s Richard Wang and Adama Anozie discuss Hospital Exemption (HE) which is an emerging access pathway for Advanced Therapy Medicinal Products (ATMPs). Main topics of discussion include the benefits and potential downsides associated with this pathway, the EU regulations involving HE and recent events in Italy, Spain and France, as…
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In this episode P4A’s Senior Client Relationship Director, Bruce Chin discusses his experiences from the in person World Orphan Drug Congress USA 2021. Topics of discussion included travel and safety precautions due to COVID, Bruce’s favourite sessions, key learnings from the sessions, and much more! Presenter: Georgie Rack Contributor: Bruce Chin …
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In this episode Akshay Kumar and Richard Wang discuss how through innovative technology, decentralised clinical trials came to the limelight, facilitating remote access and ensuring continued operation of clinical trials. In addition, we discuss the future of decentralised clinical trials in a post-COVID world, its implications and demands within t…
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In this episode, the P4A team take a deep dive into the Brazilian pharmaceutical market and discuss its potential for access to orphan drugs, cell and gene therapies. The overall Latin American pharma market is forecast to grow by 9% per year through to year 2028. However, major markets in this region such as Brazil have some major barriers to acce…
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Its almost six months since President Joe Biden came to office but the role of the head of the country's key drug regulator, the US Food and Drug Administration (USFDA) is yet to be filled. In this episode, the P4A discusses the absence of a USFDA commissioner and its impact. Also, we understand the significance of recent remarks of the Center for …
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In this month's episode, the P4A team take a deep dive into the impact of Brexit on the UK's market access landscape. Four months after leaving the European Union, the UK's attractiveness as a key destination for commercialisation of new drugs is being tested. In order to improve its credentials, the UK is exploring a slew of measures that could po…
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First, how can we see Mars and the constellations in the night sky this month? Then, an interview with Andrew Elliot (Eastern Kentucky University) about a chelator that could be useful in cancer treatment, followed by an opinion piece about the 'canary in the coal mine' of climate change. Finally, how do pets and poetry improve our lives? Thanks to…
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The world of orphan drugs is at the cusp of a transformation in the post COVID era. In this episode, the team discuss the impact of the pandemic on value demonstration of orphan drugs. In order to be successful, manufacturers of precision medicine will need to focus on carving out a niche for their drugs and support health systems to find the right…
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Scott Miller shows us how to find Mars and constellations in the night sky this month. Then, there is another panel discussion about the important environmental bills being discussed in the Kentucky House and Senate this year. And how can scientists have an impact? This is a discussion with lobbyists/advocates Tom FitzGerald of the Kentucky Resourc…
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What are the important environmental bills being discussed in the Kentucky House and Senate this year? This is an interview with lobbyists/advocates Lane Boldman and Randy Strobo of the Kentucky Conservation Committee about pending Kentucky environmental legislation. This interview was conducted by Trent Garrison, President of the Kentucky Academy …
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In this episode, the P4A team discussed the impact of cost-containment measures in Germany, the European Commission's recent move to revise legislation on orphan medicinal products and potential legislative reforms in the US under the new Biden administration. Presenter: Jens Leutloff Contributors: Chloe Sheppard, Max Rex Producer: Aparna Krishnan…
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Listen to the landing of the Perseverance rover on the planet Mars, while J Scott Miller provides background about this ambitious NASA project. Then Dr. Gretchen Goldman of the Union of Concerned Scientists speaks on 'A Roadmap to Restoring Federal Science'. Thanks to the Union of Concerned Scientists (www.ucsusa.org/about) for permission to broadc…
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What parts of the face are more important in recognizing emotions like happiness, sadness, fear or anger? How does age and gender affect our ability to perceive facial reflections of emotions? How do masks effect how people interpret our emotions, and what can we do about it? Dr. Andrew Mienaltowski, a psychologist at Western Kentucky University, d…
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What stars & planets can be seen in the night sky this month? And how did ancient Australian star-mappers determine the location of modern highways? Can prosthetic arms be controlled with our minds? What are the prospects for scientists in the new Biden administration? Does the pandemic affect our reading habits? What can we do about it? This week …
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In this Rare Disease Day special episode, we focus on the impact of COVID -19 on rare disease patients. Some of the key challenges for patients during this pandemic include disruption in their access to treatments, struggle to continue participation in clinical trials, lack of access to vital equipment such as PPE (Personal Protective Equipment) an…
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Two undergraduate students who recently won conference awards from the Kentucky Academy of Science talk about their research on tree buttressing (Erin Carleton, NKU) and novel ways to monitor stream contamination (Samuel Kessler, UL). The show ends with Professor J Scott Miller commenting about sea level rise. Thanks to Amanda Fuller (KAS) for the …
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On January 16, 2021, President Joe Biden introduced his picks for science advisors to the White House. Meet Drs. Eric Lander, Alondra Nelson, Frances Arnold and Maria Zuber. For the first time, there will be a scientist on the President's Cabinet! Vice President-Elect Kamala Harris finishes the show. The full press conference can be viewed here: ht…
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What interesting objects are in the night sky this month? J Scott Miller will fill you in. Then Dave Robinson describes a unique fast radio burst just detected in the cosmos. This one produces a pattern. The airborne SOFIA telescope is introduced, and Scott describes how it was recently used to confirm the existence of water on our moon. The show e…
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Hear about potential White House interference with the work of COVID-19 scientists at the CDC. Then, our interview continues with two Duke University researchers who have recently developed a simple, low-cost technique for assessing the ability of different face masks to reduce the spread of the coronavirus. We talk to Drs. Eric Westman and Martin …
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Duke University researchers have recently developed a simple, low-cost technique for assessing the ability of different face masks to reduce the spread of the coronavirus. We interview Drs. Eric Westman and Martin Fischer about how they compared 14 different masks for their efficacy in reducing respiratory droplets. Find out their results here! The…
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In this episode, the P4A team review all the key events of the year 2020 in the orphan drug, cell and gene therapy world. The emergence of COVID-19 pandemic has caused disruption but also opportunities for the biotech industry. From change in regulations, new stakeholder collaborations to impact of Brexit and US drug pricing reforms, a full round u…
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Join us as we celebrate our 100th episode! Hear more from Dr. Steven Stack (Kentucky Department of Public Health) about COVID-19. He also has general advice for scientists. This is a continuation of his plenary talk to the 2020 conference of the Kentucky Academy of Science (listen to our Nov 23 episode for the start of this talk). Then Professor J …
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Hear the first half of Dr. Steven Stack's keynote address to the 2020 annual conference of the Kentucky Academy of Science. Dr. Stack is the Commissioner of the KY Department of Public Health. The title of his address is 'COVID-19: Applied Science in Action'. The rest of his talk will be broadcast in a later episode. Thanks to Dr. Stack and the Ken…
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J Scott Miller describes what's to be seen in the night sky in November, 2020. In another story, Scott discusses the various ramifications of sea level rise due to climate change. Then, hear House of Representative Thomas Massey (KY) grill former Secretary of State John Kerry about climate change, in what has been called 'the dumbest line of questi…
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Hear Al Franken, Laurie Garrett, and Andy Slavitt discuss the approaching rollout of a vaccine for the SARS CoV-2 coronavirus. How is the White House interfering with public health policy-making and science? Then hear updates on the FDA meeting slated for Oct. 22nd. Part of this episode is from the Al Franken Podcast of Sept. 13 2020: https://alfra…
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