Join us as we sit down with Paul Romness of OS Therapies to explore an exciting breakthrough in OST-HER2 targeted therapies for osteosarcoma —a rare and aggressive bone cancer. Learn how this innovative approach is offering new hope to patients, advancing precision medicine, and pushing the boundaries of rare disease treatment, not just for humans but for dogs too. Joining the discussion as always is host Owen Bryant, along with George Rack and guest presenter and P4A's MA Oncology expert, Rosa ...
Jul 01, 2025•28 min
Discover how artificial intelligence is transforming the future of rare disease research in the latest episode of Let’s Talk Rare , the official podcast from Partners4Access (P4A). Join us as we sit down with Ilya Burkov, AI expert at Nebius to explore how cutting-edge machine learning tools are accelerating the discovery, diagnosis, and development of treatments for rare conditions. With over 300 million people affected by rare diseases worldwide, the need for faster, smarter solutions has neve...
May 29, 2025•38 min
In the latest episode of Let's Talk Rare, Owen Bryant & Georgie Rack delve into the world of drug development in rare diseases. They are joined by the brilliant Rob Freishtat, president of Uncommon Cures. Rob brings unique insights on who the key stakeholder are in developing drugs and the many ways that we can bring them together in order to improve and enhance the delivery of much-needed therapies.
Apr 28, 2025•37 min
Brain cancers are notoriously difficult to manage with current treatments offering limited effectiveness. But what if there was a way to change that? Michael Roberts from Adaptin Bio joins Owen Bryant to explore how a pioneering new treatment called BRiTE is driving hope for improved outcomes for patients.
Mar 31, 2025•28 min
Dive into P4A's annual appraisal of the year ahead. In this episode of Let's Talk Rare we examine the most important trends as identified and voted for by the P4A team. Ranging from Donald Trump's tariff impact on EU's ability to price orphan drugs to the change of mindset in HTA bodies to include patient experience data in their decision making calculations. Join Owen & Georgie, along with Sophie Schmitz and Pina Haberl for a wide ranging and dynamic discussion on the year ahead....
Jan 31, 2025•27 min
After3 years in the making, the Joint Clinical Assessment for the EU is in effect. From 12th January 2025 new cancer medicines and advanced therapy medicinal products (ATMPs) will require a JCA across all 27 member states. Join Owen and Georgie, along with guests Darren Callanan and Sam Morrison to discuss the main talking points of the JCA, and what to look out for when dealing with the new regulations.
Jan 13, 2025•25 min
Welcome to this month's P4A Let’s Talk Rare podcast episode by Partners4Acess . Today, Georgie and Owen are joined by Leonard Mazur , CEO of Citius Pharmaceuticals , to discuss their innovative therapy, Lymphir, for cutaneous T-cell lymphoma (CTCL). Leonard shares his journey with Citius, from its founding in 2013 to the recent resubmission of Lymphirto the FDA, and discusses the drug's potential to alleviate severe itching for the 21,000 annual CTCL patients in the U.S. The conversation also co...
Nov 19, 2024•26 min
In this episode of P4A Let's Talk Rare , hosts Georgie and Owen Bryant from Partners For Access are joined by Wes Michael , founder and president of Rare Patient Voice LLC . Wes shares the organization’s journey from its beginnings as a community project for hemophilia patients to a global platform that connects rare disease patients and caregivers with healthcare professionals. Rare Patient Voice has awarded over $30 million to participants and completed more than 210,000 projects, with Wes hig...
Oct 10, 2024•24 min
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 Aurelija Luko , the CFO, and Darren Callanan , the Global Digital Lead at Partners4Access . Join them as they discuss the recent job cuts in the pharma and biotech industry and the reasons behind them. Aurelia explains that redundancies are not limited to the pharma industry and are influenced by factors like mergers, expiring patents, and macroeconomic ins...
Jul 02, 2024•27 min
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 for drug developers to understand patient perspectives and gather data on their experiences, preferences, and needs. Pina and Sam highlight how pat...
May 03, 2024•35 min•Season 6Ep. 4
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 conference. We also shared our excitement for AI and patient experience topics, as well as the importance of sustainability and finding new pricing app...
Apr 10, 2024•29 min
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, and the importance of patient experience data. Nick explains that the challenges for rare patients remain the same, with diagnosis being the first hur...
Feb 29, 2024•25 min
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 bottom of our hearts, we would not have gotten here without you. A special episode calls for special guests, and joining us today are our in-house expert ...
Jan 02, 2024•54 min•Season 5Ep. 12
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 friendly and engaging tone, this podcast offers valuable insights into healthcare and the potential of digital solutions. RJ Kedziora Bio: Mr. Kedziora is ...
Dec 01, 2023•28 min•Season 5Ep. 11
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 engagement, highlighting the importance of technology and patient empowerment in driving this evolution. They also emphasize the need for equitable a...
Oct 06, 2023•24 min
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 proactive preparation for the upcoming changes. The topics covered include: key milestones of the EU HTA regulation; the importance of drug developers of...
Jul 31, 2023•17 min•Season 5Ep. 7
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 clinical trial. STRIDE is a global, randomized double-blind, 6 months, placebo-controlled trial designed to assess the efficacy and safety of Mavod...
Jun 01, 2023•16 min
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 tells us about the launch journey for their main therapeutic candidate for AKPKD. How they managed to complete enrolment for late phase study in reco...
Apr 29, 2023•19 min•Season 5Ep. 4
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: https://www.linkedin.com/in/max-rex-7a364789/ Stefaan Friers: Takeda: https://www.takeda.com/ LinkedIn: https://www.linkedin.com/in/stefaanfie...
Apr 17, 2023•24 min
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 include: Why genetic screening of newborns in the UK lags behind the EU, funding rare disease research, clinical trial burden, patient registries, patient ...
Feb 28, 2023•40 min
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 of CAR-T & Hospital Exemptions across Europe and finally the Patient - Putting patients at the forefront of drug developers. Neil Grubert Bio...
Feb 03, 2023•44 min
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 their presentations, key learnings and their personal highlights from the conference. Also the P4A team will be talking about our favourite sessions ...
Dec 20, 2022•43 min•Season 4Ep. 11
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 trend we are likely to see continue? Join Stefan Walzer & Fisentzos Stylianou discuss the new bill, in regards to the biggest changes that impact...
Oct 31, 2022•39 min•Season 4Ep. 10
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 the implications, if any, for drug manufacturers who do not include the patient voice. How can we bring all stakeholders together to work com...
Oct 03, 2022•28 min•Season 4Ep. 9
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 part will focus on how do we ensure there is genuine patient involvement at all stages of drug development? What are the challenges and barriers ...
Sep 13, 2022•31 min•Season 4Ep. 8
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, and we take a closer look into each one. Later in the show we have a very special announcement from our Managing Partner, Sophie Schmitz. Hos...
Jul 29, 2022•15 min•Season 4Ep. 7
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 process will link to national processes. EUnetHTA21 and how the methodology is evolving with G-BA & IQWIG increasingly involved, will this be ...
Jun 30, 2022•29 min•Season 4Ep. 6
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 on the different payment models, including the motivation for the report and key learnings. We look at sustainable patient access and whether the rep...
May 31, 2022•25 min
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 Stylianou discusses China’s healthcare system, key challenges for OD manufacturers, Spinraza’s commercialisation journey in China, and incentives availa...
Apr 29, 2022•10 min•Season 4Ep. 4
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 manufacturers aiming to launch in the Middle East. Presenter: Akshay Kumar, Partner, P4A Contributor: Lavni Varyani, Founder, Pharma Business Partners P...
Mar 31, 2022•13 min•Season 4Ep. 3
