P4A Let's Talk Rare: The Life Science Podcast

Rare Disease Day Special | Let’s Talk RareThis Rare Disease Day, Let’s Talk Rare releases a special episode focused on the data and real-world context behind rare diseases.The episode features Iro Malekous, consultant at Partners4Access, who discusses:Key statistics shaping the rare disease landscapeWhat those numbers mean for patients and familiesGaps in diagnosis, access, and treatmentWhy Rare Disease Day remains an important global awareness momentThis episode takes a clear, evidence-based look at rare diseases while grounding the discussion in lived experience.

In this episode of Let’s Talk Rare, we looked back at P4A’s recent trip to Glasgow, where our colleagues, Sam Morrison & Iro Malekou were on the ground for ISPOR activities leading up to ISPOR 2025. They weren’t just observing—they were actively contributing, presenting a poster abstract, connecting with experts, and taking the pulse of what’s shaping the HEOR and rare disease landscape.We reflected on their experiences: the conversations that stood out, the insights gained from engaging with stakeholders, and the early signals about where health economics is heading in 2025. From lively discussions in the exhibition hall to the themes emerging in scientific sessions, our colleagues returned with a clear sense of the momentum—and the challenges—facing rare disease access.This episode highlights their key takeaways: what we learned from presenting our work, how the community reacted, and what the atmosphere in Glasgow suggested about the priorities and pressures that will influence ISPOR 2025.Whether you attended, followed from afar, or simply want an insider’s view of what’s happening in HEOR and rare disease, this recap brings you right into the room.🎧 Tune in now to hear P4A’s colleagues share their insights from Glasgow—the people they met, the conversations that mattered, and the signals shaping ISPOR 2025.

In this episode, we tune into the unfiltered voices of patients and caregivers navigating life with rare conditions.Through the power of social listening, we uncover the hidden struggles around mental health—stories often left unsaid in clinics but shared openly online. Joined by Lauren Roberts from Rareminds, we explore what these conversations reveal, why they matter, and how they can reshape support for rare disease communities.

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 Faria Matthews.Don’t miss this powerful conversation on the frontlines of rare cancer research.Tune in now—because every rare voice matters. #LetsTalkRare #Osteosarcoma #OSTHER2 #RareDisease #OSTherapies

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.

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.

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 covers Citius's commercialisation plans and ongoing trials exploring Lymphir’s use for other conditions. Join Georgie, Leonard, and co-host Owen for an insightful discussion on how Citius is committed to improving patient quality of life through groundbreaking treatments! Leonard explains how Citius acquired Lymphir in 2021, a drug facing regulatory challenges that they’ve addressed for FDA resubmission, with potential approval expected. They discuss Lymphir’s benefits, especially for CTCL patients, including its potential to relieve severe itching and outline the company's commercialisation plans with Eversana. Leonard emphasises the team’s extensive expertise as vital to navigating the regulatory process. They also explore Lymphir’s off-label potential for other cancers, with ongoing trials at major universities. The conversation closes on the rewarding impact of pharmaceutical work on patient lives, underscoring Citius's commitment to addressing unmet medical needs through innovative treatments. Leonard Mazur Bio: Leonard Mazur is a seasoned entrepreneur and executive with over five decades of experience in the pharmaceutical industry. He is known for his skill in founding and growing multiple healthcare companies. Currently CEO of Citius Pharmaceuticals, Leonard previously co-founded Leonard-Meron Biosciences and Akrimax Pharmaceuticals. He also led Triax Pharmaceuticals as COO, specialising in dermatology, and successfully sold his dermatological company, Genesis Pharmaceuticals, to Pierre Fabre in 2003. His extensive background spans roles in sales, marketing, and business development at Medicis, ICN, Knoll Pharma, and Cooper Labs. Born in Germany, Mazur holds an MBA from Temple University, where he also earned his undergraduate degree. He is an Ellis Island Medal of Honor recipient. Episode Resources: Leonard Mazur on LinkedIn Citius Pharmaceuticals Website Owen Bryant on LinkedIn Georgina Rack on LinkedIn Partners4Access Website P4A Let’s Talk Rare podcast on Apple Podcasts

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 instability. Darren adds that high drug development costs and pricing pressures also contribute to layoffs. However, he suggests that companies can use digital tools to enhance efficiency and innovation to address industry challenges. They also discuss a digital launch platform and a social listening tool created by Partners4Access. Aurelia highlights that companies are looking to increase shareholder value and reduce costs. Darren adds that thanks to the high cost of drug development and downward pressure on prices, companies are forced to narrow their focus and compete in the same space, leading to layoffs. The conversation then shifts to discuss the digital launch platform created by Partners for Access. Darren explains that the platform streamlines the access and utilization of launch materials, ensuring that affiliates have access to the most up-to-date and compliant versions. This helps overcome the challenge of inconsistent distribution and saves time for the teams. Darren also mentions another tool called RarePulse, a social listening platform. This tool collects and analyzes data from social media and other sources to provide insights on key opinion leaders, unmet patient needs, and other relevant topics. He emphasizes that social media listening allows for a broader understanding of patient experiences and can help shape primary research and launch strategies. Aurelija Luko Bio: Aurelija’s work at P4A includes overseeing all accounting activities, from managing financial risk to setting budgets. Also, to ensure smooth day-to-day finance operations within the business.​ Aurelija is a Chartered Accountant with 10+ years of experience; she holds an MSc in Financial Economics from the University of Exeter and a BSc in the same field. ​ ​Before P4A, she worked for small start-ups and big listed companies in healthcare, consulting, and property industries. She is a problem solver who can adapt well to new situations and review and improve the internal controls within the organization. Darren Callanan Bio: Darren Callanan is the Global Digital Lead at Partners4Access. He works in a specialist consultancy team to develop digital solutions addressing healthcare delivery barriers. Darren holds a master’s degree in Biotechnology and Business Management from the University of Warwick and an honors degree in Physiology from the National University of Ireland, Galway. His expertise includes strong communication and interpersonal skills, supported by a robust scientific background across various therapeutic areas. Previously, he worked at S3 Connected Health. Episode Resources: Aurelija Luko on LinkedIn Darren Callanan on LinkedIn Owen Bryant on LinkedIn Georgina Rack on LinkedIn Partners4Access Website P4A Let’s Talk Rare podcast on Apple Podcasts

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 approaches in healthcare. Gain insights on inclusivity and involving patients in drug development. Get ready for a meaningful, engaging conversation that will leave you inspired. Take a deep dive into the benefits of tiered pricing, the challenges and concerns of companies and HTA agencies in joint HTA assessments, the importance of inclusivity, and the importance of involving and educating others. Juliette Sinclair-Spence: Bio and Quote LinkedIn Juliette is the Funder and Chairman of the AK Eye Foundation and is a committed 𝐀𝐜𝐚𝐧𝐭𝐡𝐚𝐦𝐨𝐞𝐛𝐚 𝐊𝐞𝐫𝐚𝐭𝐢𝐭𝐢𝐬 (𝐀𝐊) 𝐏𝐚𝐭𝐢𝐞𝐧𝐭 𝐀𝐝𝐯𝐨𝐜𝐚𝐭𝐞. She has experienced this rare eye disease, and her key learning was that there is much that needs to be done on many fronts, including prevention, education, diagnosis, treatment, research, and support. Her passion for fighting against the disease led her to set up the first worldwide foundation focused on Acanthamoeba Keratitis. “I think involving patients in the drug development process is so important. We need to hear their voices and understand their journeys.” Sandrine Ruiz: Bio and Quote LinkedIn Sandrine, or Sandy as she likes to be called, is the Senior Director of MarketAccess, Pricing, and HEOR at Immunocore. She is an experienced Pricing & Market Access professional with 25+ years in the biopharmaceutical industry. She has hands-on experience in giving strategic and science-based input across the pharmaceutical development workflow, from early stage to late stage, into Market/Patient Access, Pricing & Reimbursement, Payer Strategy, Real World Evidence, Clinical Development, Clinical Operations, and Business Development. “I was really intrigued by the panel discussion on tiered pricing. It's such a hot topic right now, and it was interesting to hear the different perspectives on patient access. I'm also fascinated by digital information and the new trends in our field.” Neil Grubert: Bio and Quote LinkedIn Neil is an Independent Global Market Access Consultant and a multilingual pharmaceutical market access specialist with 30 years of experience tracking the global prescription drug and self-medication markets. He has spearheaded the establishment and growth of Decision Resources’ international market access business. As the author of more than 150 reports covering 20 mature and emerging markets, multiple therapeutic areas, and numerous industry issues, he has earned a reputation for extensive knowledge of market access environments around the world. “Yes, tiered pricing is definitely a challenging issue. Speaking of challenges, the EU HTA implementation has been causing many uncertainties for companies and HTA agencies. We need to navigate it carefully.” Seema Sondhi: Bio and Quote LinkedIn Seema is the Head of Payer Value and Access for the Vaccine Business Unit at Takeda. She is passionate about patient access to innovative medicines and has worked diligently in her country, regional, and global roles to ensure access to pharmaceuticals, vaccines, medical devices, and orphan drugs. This has been achieved through key activities such as influencing global clinical trial development to ensure the collection of appropriate evidence, all the way through to launching products at prices that allow good levels of patient access and ensuring continued access post-launch via mechanisms such as innovative access schemes. “Inclusivity is such an important aspect of our work. We can't just think about it; we have to be involved and educate others actively. Patients need to understand the questions asked and the impact of their input.”

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 and Managing Partner at P4A, Sophie Schmitz, our host from 2018-2021, Aparna Krishnan, and Prasan Subedi, Access Strategy Team Lead at Pfizer. Together, we walk down memory lane and relive the high points and challenges of the past five years, and share insights on rare diseases and cell and gene therapies. Aparna Krishnan Bio: Aparna has over 10 years of experience in the pharmaceutical and healthcare industry. As part of the senior leadership team, her primary focus is on strategic corporate engagement, legal, financial operations, and human resources. She has previously worked as Manager of Life Sciences at IHS Markit and as Consultant at Parexel’s Commercialization unit, among others specializing in market access, pricing, and reimbursement. Sophie Schmitz Bio: Sophie is the managing partner at Partners 4 Access, a company that believes in a sustainable rare disease world, which means helping our clients unlock the true opportunity of orphan drugs and ATMPs. Her 20+ year career spans various disease areas and medical devices, successfully supporting the strategy development and operationalization for orphan drugs. She brings a wealth of commercial and access experience across orphan and cell and gene therapies to the benefit of clients. She plays an active role in advising clients on price and market access strategies, with the ultimate goal of optimizing fair access for all stakeholders in rare diseases.

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 access to healthcare information. The conversation touches on the changing landscape of patient communication over the past fifteen years, with the advent of digital platforms and social media. They discuss how digitization has democratized access to health information and created opportunities for patients to connect with others facing similar conditions. They further explore the impact of digitization on the orphan drug and cell and gene therapy world, emphasizing the role of patient involvement in content creation and the use of multimedia resources like infographics to cater to diverse learning styles. Iola Forster Bio: Iola Forster is the Head of Publications and Portfolio at Karger Publishers. She has over fifteen years of experience in the publishing industry and is skilled at fostering partnerships between the life sciences, healthcare clients, and Karger. Iola brings a wealth of knowledge and expertise to the table. Episode Resources: Iola Forster on LinkedIn Karger Publishers website Partners4Access website P4A Let’s Talk Rare podcast on Apple

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 Mavodelpar. Alex also discusses the importance of engaging early with #patients and patient organisations for #PMM across the world to truly understand the patient journey, their challenges & daily routines which help to shape the study design. This ensures successful enrolment and patient retention throughout the clinical trial, something Reneo have successfully completed throughout the development of Mavodelpar. By engaging early with patients, clinicians & regulators, they have completed enrolment in record time, this is especially difficult for #rarediseases as it is often difficult to find the patients and something Alex is extremely proud of. Reneo Pharma: https://reneopharma.com/ Reneo is a clinical-stage pharmaceutical company focused on the development of therapies for patients with rare genetic diseases including mitochondrial diseases with significant unmet medical needs. LinkedIn: Alejandro Dorenbaum https://www.linkedin.com/in/alejandro-alex-dorenbaum-m-d-b01a6ab/ JustGiving: https://www.justgiving.com/fundraising/partners4access-b-v?utm_source=copyLink&utm_med[…]ampaign=pfp-share&utm_term=0c6d8180e0f3440b8383845adedb6513