Shows
P4A Let's Talk Rare: The Life Science PodcastAI for Rare Disease: From Discovery to Trials, Smarter and FasterDiscover 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 never been greater. Ilya sheds light on the real-world applications of AI in identifying new genetic links, optimizing cli...2025-05-2937 min
P4A Let's Talk Rare: The Life Science PodcastImproving Alignment In Drug DevelopmentIn 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.2025-04-2836 minP4A Let's Talk Rare: The Life Science PodcastBreaking BarriersBrain 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.2025-03-3127 minP4A Let's Talk Rare: The Life Science PodcastP4A's 5 Top Trends for 2025Dive 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.2025-01-3127 min
P4A Let's Talk Rare: The Life Science PodcastThe JCA is live
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.
2025-01-1325 min
P4A Let's Talk Rare: The Life Science PodcastInnovative Treatments for Cutaneous T-Cell Lymphoma with Leonard Mazur of Citius PharmaceuticalsWelcome 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 i...2024-11-1926 min
P4A Let's Talk Rare: The Life Science PodcastAmplifying Patient Voices: Wes Michael on Rare Disease AdvocacyIn 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 highlighting the company’s organic growth through patient referrals and advocacy partnerships.
Wes discusses the challenges of reac...2024-10-1023 min
P4A Let's Talk Rare: The Life Science PodcastImpact of Mergers, Acquisitions, and Patent Expirations on Job Cuts in Pharma and Biotech: Insights from Aurelija Luko and Darren CallananWelcome 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 co...2024-07-0227 min
P4A Let's Talk Rare: The Life Science PodcastThe Role of Patient Experience Data in Drug Development with Pina Haberl and Sam Morrison
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 patient experience data can improve drug development and clinical trial design while differentiating products in the...2024-05-0334 min
P4A Let's Talk Rare: The Life Science PodcastWorld EPA Conference 2024 SpecialWelcome 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 d...2024-04-1028 min
P4A Let's Talk Rare: The Life Science PodcastRare Disease Day Special 2024: The Role of Genetic Diagnosis in Rare Conditions with Nick MeadeWelcome 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 hurdle. He highlights the progress being made in genetic diagnoses and screening but emphasizes the need fo...2024-02-2924 min
P4A Let's Talk Rare: The Life Science PodcastCelebrating 5 Years of P4A: Reflections on Rare Diseases, Cell and Gene Therapies, and Legislation in Europe
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...2024-01-0253 minP4A Let's Talk Rare: The Life Science PodcastRevolutionizing Patient Outcomes: The Power of Digital Health Solutions with RJ KedzioraIn 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 the co-founder of Estenda Solutions, a leading company specializing in custom software and data...2023-12-0128 minP4A Let's Talk Rare: The Life Science PodcastThe Future of Patient Engagement and the Role of Technology in Healthcare with Iola ForsterWelcome 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, wit...2023-10-0623 minP4A Let's Talk Rare: The Life Science PodcastNavigating the New EU HTA Regulation and Its Impact on ATMP Development
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 ATMPs and oncology medicines to not adopt a wait and watch str...2023-07-3116 minP4A Let's Talk Rare: The Life Science PodcastAlejandro Dorenbaum, CMO of Reneo Pharmaceuticals Discusses Clinical-Stage Therapies
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 chal...2023-06-0116 minP4A Let's Talk Rare: The Life Science PodcastInterview with Xortx Therapeutics CEO, Dr Allen Davidoff discussing their journey from early development to potential launchP4A 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 record time by engaging with patients and advocacy groups early, to really understand the patient journey and how...2023-04-2919 minP4A Let's Talk Rare: The Life Science PodcastWorld EPA Congress 2023 SpecialP4A 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/stefaanfiers/
Juliette Sinclair Spence:
2023-04-1724 min
P4A Let's Talk Rare: The Life Science PodcastRare Disease Day Special 2023In 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 experience data and its relevance, revamping data sharing with families, and how pharma can involve patients earlier.
...2023-02-2840 minP4A Let's Talk Rare: The Life Science PodcastTrends to look out for in 2023The 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:
Neil has worked in Industry for over 20 years starting at Decision Resources as a researcher in 1997 a...2023-02-0344 minP4A Let's Talk Rare: The Life Science PodcastWorld Orphan Drug Congress Europe 2022 SpecialDid 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 and the key takeaways from the conference.
Special thanks t...2022-12-2042 minP4A Let's Talk Rare: The Life Science PodcastWinds of change for German Healthcare Market! 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 orphan drug (OD) manufacturers. Will OD manufacturers still see Germany as the first go to ma...2022-10-3138 minP4A Let's Talk Rare: The Life Science PodcastPart 2 -Patient Empowerment: Is it a no-brainer?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 completely new approaches to medicine approval and patient access. Lastly, we will look at initiatives or services our panel are...2022-10-0328 minP4A Let's Talk Rare: The Life Science PodcastPart 1 Patient Empowerment; Why involving patients from early drug development through to launch is a no-brainer!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 for patients living with a rare disease vs drug developers within the rare/ultra-rare sphere?2022-09-1331 minP4A Let's Talk Rare: The Life Science PodcastWhat makes P4A an award winning consultancy?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.
Host: Georgie Rack, Communication Executive & Owen...2022-07-2915 min
P4A Let's Talk Rare: The Life Science PodcastSpecial Episode with EUCOPE-Part 2 with Alexander NatzIn 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 a major influence in the development of final methodology? Finally, we look at how Pharma companies should be best preparing for the implementation of...2022-06-3028 minP4A Let's Talk Rare: The Life Science PodcastSpecial series with Eucope: Part 1 featuring Victor MaertensPart 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 report addresses the affordability issues surrounding ATMP’s drug pricing and if this is even possible in the current land...2022-05-3124 minP4A Let's Talk Rare: The Life Science PodcastChina market access: obstacle or opportunity for orphan drug, cell and gene therapy manufacturers?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 available to OD manufacturers in China.
Reference: https://www2.deloitte.com/cn/en/pages/life-sciences-and-healthcare/solutions/life-sciences.html
Presenter: Aparna Krishnan, Partner – Global O...2022-04-2910 minP4A Let's Talk Rare: The Life Science PodcastShould the Middle East be a priority launch market for orphan drug manufacturers?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
Producer: Operations team2022-03-3112 minP4A Let's Talk Rare: The Life Science PodcastRare Disease Day Special featuring Janet Bloor and Nick Meade - February 2022For 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 Duchenne Nexus Advocacy (DNA) and Nick Meade, Director of Policy and Joint Interim Chief Executive of Genetic Alliance UK
Producer: Operations team2022-02-2828 minP4A Let's Talk Rare: The Life Science PodcastP4A Insights: Trends to look out for in 2022Lots 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 Bernardini
Produced by: Ops team2022-02-0410 min
P4A Let's Talk Rare: The Life Science PodcastSpecial episode - Bluebird Bio: What went wrong? -December 2021: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: Sophie Schmitz, Managing Partner and Joanna Fernandes, Senior Consultant
Producer: Operations team2021-12-1317 minP4A Let's Talk Rare: The Life Science PodcastSpecial episode featuring James Mackay - November 2021This 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 Aristea Therapeutics
Producer: Operations team
About Aristea Therapeutics, a San Diego-based clinical-stage immunology-focused drug development company developing novel therapies for serious, rare inflammatory diseases.2021-11-3023 minP4A Let's Talk Rare: The Life Science PodcastMonthly Roundup - October 2021In 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 well as the implications of HE on the traditional pharma model, the need for collaboration between big pharma and hospitals/institutions to develop HE therapies and much more!
Presenter: Richard Wang
Contributor: Adama Anozie
Producer: Operations Team2021-10-2910 minP4A Let's Talk Rare: The Life Science PodcastMonthly RoundUp - September 2021In 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
Producer: Operations Team2021-09-3015 minP4A Let's Talk Rare: The Life Science PodcastMonthly RoundUp: July 2021In 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 the industry.
Presenter: Akshay Kumar
Contributors: Richard Wang
Producer: Operations Team2021-07-3015 minP4A Let's Talk Rare: The Life Science PodcastMonthly RoundUp - June 2021In 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 access. Listen in to learn more.
Presenter and Contributor: Ciaran Cassidy
Producer: Aparna Krishnan2021-06-3007 minP4A Let's Talk Rare: The Life Science PodcastMonthly RoundUp - May 2021Its 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 Biologics Evaluation and Research (CBER) director Peter Marks on the need for consistency in the manufacture of cell and gene therapies.
Presenter and Contributor: Max Rex
Producer: Aparna Krishnan2021-05-3109 minP4A Let's Talk Rare: The Life Science PodcastMonthly RoundUp - April 2021In 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 potentially spark a reform.
But will that happen? P4A's Joanna Fernandes and Jayne Watson consider two distinct initiatives - MHRA's (Medicines Healthcare Regulatory Agency's) new I-LAP (Innovative Licensing and Access Pathway) scheme and England's HTA b...2021-04-3020 minP4A Let's Talk Rare: The Life Science PodcastMonthly RoundUp - March 2021The 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 patient for their treatment at the right time.
The team also explore the role of digital tools such as Artificial Intelligence (AI) and telemedicine in helping drugmakers optimise manufacturing and treatment delivery. This combined w...2021-03-3113 minP4A Let's Talk Rare: The Life Science PodcastMonthly RoundUp - February 2021In 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 Krishnan2021-02-2722 minP4A Let's Talk Rare: The Life Science PodcastRare Disease Day special episode: Reclaiming the rare disease patient’s voiceIn 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) and also to healthcare personnel. We speak to Genetic Alliance chief executive officer Jayne Spink, patient Nicola Whitehill and Danielle Myers, mother and carer of patient 10 year old Dylan Myers.
Presenter: Aparna Krishnan
Contributors: Jayne Spink, Nicola Whitehill and Danielle...2021-01-2936 minP4A Let's Talk Rare: The Life Science PodcastEnd of year 2020 reviewIn 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 up of 2020. So do listen in!
Presenter and Contributors: Sophie Schmitz and Akshay Kumar
Producer: Aparna Krishnan
2020-12-2118 minP4A Let's Talk Rare: The Life Science PodcastSpecial episode: Gene Therapy access from a specialty pharmacy perspectiveThis episode discusses patient access to AveXis' gene therapy Zolgensma from Orsini Healthcare, US based specialty pharmacy's perspective. The senior team at Orsini provide insight on their experience of Zolgensma access including key manufacturer criteria, payer landscape along with challenges and lessons learnt.
Presenter: Aparna Krishnan
Contributors: Dave Frobel, Senior Vice President, Trade & Tom Shaughnessy, Senior Vice President, Health Plans
Producer: Aparna Krishnan
About Orsini Healthcare: Founded in 2009, Orsini is a leading independent specialty pharmacy focused on rare conditions and gene therapies. Orsini was one of the first specialty pharmacies to provid...2020-06-2220 minP4A Let's Talk Rare: The Life Science PodcastWeekly RoundUp: February 29, 2020This week is a special episode celebrating Rare Disease Day 2020. Our guest speaker is Annie Kennedy, head of policy and advocacy at Every Life Foundation, a US based non profit organisation that works to advance the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. Here she speaks about the organisation's initiatives on drug access.
To know more about the foundation, visit https://everylifefoundation.org/
Presenter and Producer: Aparna Krishnan
Contributor: Annie Kennedy, Chief of Policy & Advocacy, EveryLife Foundation for Rare Diseases2020-02-2917 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: December 30, 2019As 2019 winds down, P4A's Sophie Schmitz and Akshay Kumar discuss the key trends that defined this year - marketing approval of cell and gene therapies; pricing and reimbursement challenges in US & EU5 as well as industry merger and acquisitions.
Presenter: Sophie Schmitz
Contributor: Akshay Kumar
Producer: Aparna Krishnan2019-12-3009 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: December 23, 2019The team discuss the latest update on the proposed EU health technology assessment (HTA) regulation. The key objectives of the 2018 proposal was to promote convergence in HTA tools, procedures and methodologies; reduce duplication of efforts for HTA bodies and industry and improve joint use of outputs. This episode goes indepth into the one of the controversial aspects of the proposal - The joint clinical assessment - that caused a near stand still in negotiations between member states.
Presenter: Christina Poschen
Contributor: Ciaran Cassidy
Producer: Aparna Krishnan2019-12-2304 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: November 12, 2019The team discuss the NORD summit particularly FDA commissioner Scott Gottleib’s presentation, panel discussion on cell and gene therapy pricing as well as P4A’s lunch and learn on cell and gene therapy access – learnings from the EU.
Also in this episode, Novartis’ Kymriah innovative payment agreement in Italy, the changing evaluation trends for gene therapies among HTA bodies and Alliance for Regenerative Medicines’ Q3 report.
Presenter: Jack Rawson
Contributor: Sophie Schmitz
Producer: Aparna Krishnan2019-11-1214 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: November 5, 2019Following an autumn hiatus, the team come back to discuss Vertex's journey to commercialize Orkambi picking up the story from the latest deal struck with NHS England in October 2019.
Presenter: Nicola Allen
Contributor: Joanna Fernandes
Producer: Aparna Krishnan2019-11-0508 minP4A Let's Talk Rare: The Life Science PodcastWeekly RoundUp: August 5, 2019In this episode, P4A speaks to a special guest - Alliance of Regenerative Medicine's chief executive officer Janet Lambert on the ARM's key initiatives such as the Foundation of Cell and Gene Therapy medicines as well as the 2019 report on ATMPs (Advanced Therapy Medicinal Products).
Presenter: Aparna Krishnan
Contributor: Janet Lambert, CEO of Alliance of Regenerative Medicine
Producer: Aparna Krishnan 2019-08-0518 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: July 29, 2019The team takes a look at the most recent legislation in Germany known as GSAV or the law for more safety in the supply of pharmaceuticals and its implications on the access to orphan drugs. GSAV was approved by the German cabinet on January 30, 2019 and is due to come into effect in August this year.
Presenter: Akshay Kumar
Contributor: Joanna Fernandes
Producer: Aparna Krishnan
2019-07-2905 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: July 22, 2019This week, the P4A team discuss the coverage of Novartis' Zolgensma in the US in particular how US health insurers have received the gene therapy. Also on the agenda was the impact of Zolgensma's launch on rival Biogen's Spinraza and the potential for dominance of the gene therapy in the spinal muscular atrophy landscape.
Presenter: Aparna Krishnan
Contributor: Max Rex
Producer: Aparna Krishnan2019-07-2411 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: July 1, 2019The debate over US drug pricing reform has reached fever pitch as the US Department of Health and Human Sciences announced its latest Medicare Part B proposal to include international reference pricing. The Partners4Access team looks at the background to this proposal; the political and patient organisation criticisms; the potential industry response and its ramifications.
Presenter: Aparna Krishnan
Contributor: Max Rex
Producer: Aparna Krishnan2019-07-0112 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: June 23, 2019This week, we analyze Bluebirdbio's commercialization plans for its gene therapy Zynteglo including the new payment model and a delay in launch due to manufacturing issues.
Presenter: Aparna Krishnan
Contributor: Joanna Fernandes
Producer: Aparna Krishnan2019-06-2306 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: June 16, 2019This week, the team discuss the emergence of 'drug buyer's clubs' in the UK involving Vertex's cystic fibrosis drug Orkambi.
Presenter: Aparna Krishnan
Contributors: Sophie Schmitz, Ciaran Cassidy
Producer: Aparna Krishnan2019-06-1606 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: June 12, 2019This week, the team discuss Vertex's acquisition of Exonics and expansion of its collaboration with CRISPR Therapeutics. Also, we take an indepth look at the European Medicines Agency's approval of Bluebirdbio's gene therapy Zynteglo as a treatment for beta-thalassemia .
Presenter: Aparna Krishnan
Contributor: Jack Rawson
Producer: Aparna Krishnan2019-06-1210 minP4A Let's Talk Rare: The Life Science PodcastWeekly RoundUp: June 3, 2019On our anniversary episode, the P4A team discuss the USFDA approval of Novartis' Zolgensma, a one time gene therapy for paediatric spinal muscular atrophy patients and a look back at the last 12 months of our podcasting journey.
Presenter: Joanna Fernandes
Contributor: Christina Poschen, Aparna Krishnan
Producer: Aparna Krishnan2019-06-0307 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: May 25, 2019In this episode, we look at Novartis CEO Vas Narasimhan calling for a change in the US drug payment systems, arguing for new economic models to identify how much value a cure represents. Also, Italy's attempts at introducing a draft resolution to improve transparency in drug pricing at the World Health Assembly.
Presenter: Aparna Krishnan
Contributors: Joanna Fernandes, Christina Poschen
Producer: Aparna Krishnan2019-05-2510 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: May 18, 2019This week, the team discuss the deal between UK's National Health Service and Biogen prompting the successful reimbursement of Biogen’s Spinraza as a treatment for spinal muscular atrophy and Solid Biosciences’ clinical update on its Duchenne Muscular Dystrophy gene therapy product SGT-001.
Presenter: Aparna Krishnan
Contributor: Max Rex
Producer: Aparna Krishnan2019-05-1806 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: May 12, 2019This week, the P4A team discuss a new experimental therapy - Phage therapy or genetically engineered phages in order to treat bacterial infections and the acquisition of Theracon by Big Pharma firm Pfizer for upto $810 million.
Presenter: Aparna Krishnan
Contributor: Ciaran Cassidy
Producer: Aparna Krishnan
2019-05-1206 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: May 3, 2019This week, we feature a special guest podcast speaker - Scott Dorfman, chief executive officer of non-profit gene therapy developer Odylia Therapeutics on his personal journey and the future in gene therapy development.
If you would like to know more about Odylia Therapeutics, please visit https://odylia.org/
Presenter: Aparna Krishnan
Contributor: Scott Dorfman, CEO, Odylia Therapeutics
Producer: Aparna Krishnan2019-05-0318 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: April 27, 2019This week, we look at the European Federation of Pharmaceutical Industries and Associations or EFPIA’s patient wait survey and its key findings as well as news developments in the spinal muscular atrophy world relating to gene therapy Novartis’ Zolgensma and Biogen’s Spinraza.
Presenter: Aparna Krishnan
Contributors: Sophie Schmitz, Jack Rawson
Producer: Aparna Krishnan
2019-04-2719 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: April 22, 2019 As part of the continuing World Orphan Drug Congress (WODC) special, this episode discusses the cGMP facilities and biosafety solutions from Germfree’s Carol Houts, P4A’s Sophie Schmitz looks back at the 2019 conference and Terrapinn’s Andre Singer talks about what to expect from WODC 2020.
Presenter and Producer: Aparna Krishnan
Contributors: Carol Houts, Director of Regulatory and Quality, Germfree; Sophie Schmitz, Managing Partner, Partners4Access and Andre Singer, General Manager for World Orphan Drug Congress USA, Terrapinn2019-04-2214 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: April 12, 2019This week, the P4A team are podcasting from the World Orphan Drug Congress in Washington D.C. We speak to André Choulika, Chairman and CEO of Cellectis on the challenges of commercializing a CAR-T cell product and ethical concerns surrounding it, and Anna Bucsics from the Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA) on their work supporting new biotech entrepreneurs.
Presenter: Joanna Fernandes
Contributors: André Choulika, Chairman and CEO of Cellectis; Anna Bucsics, Project Advisor to MoCA and Sophie Schmitz, Managing Partner, P4A
Producer: Aparna Krishnan2019-04-1717 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: March 28, 2019The team discuss the creation of a new health economics advisory committee in Spain expected to influence pricing and reimbursement decisions for drugs; and Japan’s approval of its first gene therapy and CAR-T therapy - AnGes' HGF Plasmid and Novartis' Kymriah respectively.
Presenter: Joanna Fernandes
Contributor: Max Rex
Producer: Aparna Krishnan2019-03-2806 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: March 22, 2019This week, we are looking at The Institute for Clinical and Economic Review or ICER’s white paper on alternative options for the US rebate system, and the establishment of a national agency to evaluate drug effectiveness and negotiate prices in Canada.
Presenter: Max Rex
Contributor and Producer: Aparna Krishnan2019-03-2208 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: March 15, 2019This week, we look at Ireland's access to orphan drugs as compared to rest of western Europe and the first outcomes deal for CAR-T cell therapies in Germany.
Presenter: Joanna Fernandes
Contributors: Nader Murad, Ciaran Cassidy
Producer: Aparna Krishnan2019-03-1509 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: March 8, 2019This week, the P4A team discuss the spate of mergers and acquisitions involving Big Pharma companies in the gene therapy space. Particularly, we looked at the deals behind Roche's acquisition of Spark Therapeutics and Biogen's buyout of Nightstar. Also, Vertex's continued struggle to get its cystic fibrosis drug Orkambi reimbursed in the UK.
Presenter: Joanna Fernandes
Contributor and Producer: Aparna Krishnan2019-03-0808 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: February 28, 2019On Rare Disease Day, P4A discusses the role of policy-makers in the healthcare system and specifically, the issues surrounding cross-border healthcare as part of its '6P' campaign.
This directive is a key cornerstone legislation by EU officials that enables patients to find treatment in healthcare facilities outside their home country. However, there are several challenges associated with it.
Presenter: Christina Poschen
Contributor: Dr Andrzej Rys, Director - Health Systems and Products
Producer: Aparna Krishnan2019-02-2815 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: February 22, 2019In another special edition episode dedicated to the '6P' campaign to mark Rare Disease Day, the Weekly RoundUp team discusses the pharmaceutical perspective on partnerships in the rare disease space.
Presenter: Max Rex
Contributor: Jan-Willem Schmitz, General Manager for Nordics and Baltics, Sanofi Genzyme
Producer: Aparna Krishnan2019-02-2209 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: February 15, 2019 Throughout February, Partners4Access is running a campaign to mark the annual Rare Disease Day on February 28. The '6P' campaign is aimed at creating awareness on the impact of rare diseases on key stakeholders and the need for partnership to achieve successful treatment access for patients. The 6Ps are Partnership, Patient, Policy-maker, Physician, Pharma and Payer. This week we focus on the payer who are decision-makers assessing the value of a product in the healthcare system.
Presenter and Producer: Aparna Krishnan
Contributor: Einar Andreassen, senior advisor at the Norwegian Medicines Agency. Einar is a he...2019-02-1514 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: February 1, 2019Throughout February 2019, the P4A team are hosting a series of special edition podcast episodes to mark Rare Disease Day. The initiative is part of a new 6P campaign aimed at promoting awareness about rare disease challenges and its impact on people and society. The 6Ps are Partnership, Policy-maker, Payer, Pharma, Physician and Patient. Our message: 6Ps are essential to successfully serve the rare disease community and achieve access to medicines.
This week, we discuss the first P – Partnership. The team reflects on the meaning of partnership in the rare disease context and what it can achieve th...2019-02-0109 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: January 25, 2019 This week we are looking at the USFDA’s proposed plans to address the expected rise in cell and gene therapy product applications and a significant development in a new controversial procedure called gene drive.
Also, P4A is starting a new campaign that will run throughout February to mark Rare Disease Day. The '6P' campaign is aimed at creating awareness on the impact of rare diseases on key stakeholders and the need for partnership to achieve successful treatment access for patients. The 6Ps are Partnership, Patient, Policy-maker, Physician, Pharma and Payer. P4A will roll out a...2019-01-2511 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: January 18, 2019This week, the P4A team discusses the Louisiana Medicaid program implementing the 'Netflix' subscription model to pay for hepatitis C drugs and the potential Brexit options facing the UK government.
Presenter: Joanna Fernandes
Contributor: Max Rex
Producer: Aparna Krishnan2019-01-1809 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: January 11, 2019Happy New Year to Weekly Roundup listeners! 2019 is shaping up to be an exciting year for the healthcare and biotechnology industry. We are only a couple of weeks in and already news developments are buzzing on the regulatory and corporate front.
So for this week's episode, we start by discussing the recent wave of mergers and acquisition deals announced by Big Pharma namely, Eli Lilly and Loxo; BMS and Celgene as well as GSK and Tesaro. Also, the P4A team look at the implications of the current US government shutdown on the FDA and the agency's i...2019-01-1406 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: December 14, 2018In the last episode of 2018, the P4A team look back at the events of the year and particularly on how their predictions on key trends in the rare disease space have fared.
Presenter: Aparna Krishnan
Contributors:
Mergers and acquisitions - Sophie Schmitz
Rare oncology drug approvals - Joanna Fernandes
Future of new technologies like gene therapies - Christina Poschen
Increasing prominence of societal burden data - Nader Murad
Emphasis on planning Real World Evidence - Aparna Krishnan
U.S drug prices - Max Rex
The Weekly RoundUp team will be back in the new year.2018-12-1415 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: December 7, 2018This week, the team discuss Novartis' AVXS-101 FDA application; the UK regulators' promise to review its HTA system under a new voluntary pricing and access scheme and clinical trial updates from bluebirdbio’s gene therapy LentiGlobin.
Presenter: Joanna Fernandes
Contributor: Aparna Krishnan2018-12-0709 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: November 30, 2018This week's episode looks at the proposed rules by the Centres for Medicare and Medicaid Services (CMS) in a bid to reduce drug prices and improve e-prescribing and the OECD's new report on access to medicines.
Presenter: Aparna Krishnan
Contributor: Jack Rawson2018-11-3006 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: November 23, 2018The P4A team discuss the latest update on Brexit and its implication on life science companies; Novartis' CAR-T therapy commercialization efforts and the Hercules Project, a unique initiative supporting new drug reimbursements in Duchenne Muscular Dystrophy.
Presenter: Aparna Krishnan
Contributors: Joanna Fernandes and Sophie Schmitz2018-11-2311 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: November 15, 2018In the second part of our World Orphan Drug Congress special, P4A's Sophie Schmitz speaks to Diane Kleinermans, adviser to the Belgian Federal Government on the origins and future of the Beneluxa initiative.
Presenter: Aparna Krishnan
Contributors: Sophie Schmitz, Managing Partner, P4A and
Diane Kleinermans, Adviser to the Ministry of Health and Social Affairs, Belgium2018-11-1510 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: November 9, 2018In the first of a two-part special, the P4A team are at the World Orphan Drug Congress in Barcelona discussing access to new treatments with a Porphyria patient; hear from a company offering a unique service to clinical trial patients; a special interview with Ségolène Aymé on rare disease challenges and a chat with organisers of the congress.
Presenter: Aparna Krishnan
Contributors: Sophie Schmitz, Managing Partner, P4A; Dr Jasmin Burman-Aksözen, Vice President of International Porphyria Patient Network; Helen Springford, Vice President, Illingworth Research Group; Prof Ségolène Aymé, Founder of Orphanet; Andrew Mears, Business Develop...2018-11-0926 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: November 2, 2018The team analyze the new Medicare drug pricing plan unveiled by the Trump administration and the prospects of Vertex's cystic fibrosis drug Symkevi which gained EU approval recently.
Presenter: Aparna Krishnan
Contributors: Max Rex and Jack Rawson2018-11-0211 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: October 25, 2018This week, we look at Biogen's spinal muscular atrophy drug Spinraza's prospects in the face of emerging competition and the latest Brexit update providing recommendations on areas to prepare for as deadline for UK's exit looms.
Presenter: Aparna Krishnan
Contributor: Joanna Fernandes2018-10-2507 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: October 18, 2018This week, the P4A team analyse the new regulations surrounding health technology assessments in Europe and US-based CAR-T start-up Allogene Therapeutic's record-breaking initial public offering.
Presenter: Max Rex
Contributor: Christina Poschen, Consultant, P4A2018-10-1806 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: October 11, 2018The P4A team analyze Novartis' deal with Celluar Biomedicine Group to supply the CAR-T therapy Kymriah and the successes and failures of the reimbursement of Vertex's cystic fibrosis drug, Orkambi.
Presenter: Max Rex
Contributor: Nader Murad, Senior Analyst2018-10-1107 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: Oct 5, 2018This week, Pfizer's Matthew Harold joins us to discuss the findings of his recent research study that reviewed national policies for rare diseases in the context of key patient needs.
Presenter: Max Rex
Contributor: Matthew Harold, International Developed Markets Public Affairs Lead, Pfizer2018-10-0515 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: Sep 27, 2018This week, P4A's podcast will cover Luxturna's CHMP recommendation, Novartis' restructuring plans and Alexion's acquisition of Syntimmune.
Presenter: Max Rex
Contributor: Aparna Krishnan2018-09-2706 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: September 20, 2018Back from a summer break! The P4A team discuss the reasons behind NICE's rejection of Novartis' CAR-T therapy Kymriah, Denmark and Norway's joint drug purchasing agreement and US patient groups pushing back against ICER's influence in drug reimbursement.
Presenter: Max Rex
Contributor: Aparna Krishnan2018-09-2006 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: August 16, 2018In this episode, the team discuss UK health technology body NICE not recommending Biogen's Spinraza for routine use in the NHS; CVS announcing the use of ICER's cost effectiveness analysis to decide formulary inclusions and Express Scripts is in talks with several pharma companies for exclusive distribution rights to sell their upcoming gene therapies.
Presenter: Max Rex
Contributor: Aparna Krishnan2018-08-1606 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: August 9, 2018In this episode, P4A discusses the UK government’s publication of four key documents guiding the life science industry on how to operate during the Brexit transition period; Spark Therapeutics' clinical data for its gene therapy treating heamophila A patients and finally, the Irish government plans to reform the country’s healthcare system.
Presenter: Joanna Fernandes
Contributor: Aparna Krishnan2018-08-0904 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: August 2, 2018This week, the team discuss the consolidation among small and medium sized cell and gene therapy companies and the latest on Brexit impact with news that Sanofi is stockpiling drugs.
Presenter: Max Rex
Contributor: Aparna Krishnan2018-08-0204 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: July 26, 2018The EU's Commissioner for Competition Margrethe Vestager, recently said that price differences in the pharma market among different member states could be “justified”. This week, P4A delves deeper into the mechanics of drug pricing in Europe including discussing the impact of parallel trade and comparing it to U.S. pricing.
Presenter: Max Rex
Contributor: Stuart Tutt2018-07-2618 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: July 17, 2018The U.S. FDA's new guidance on gene therapy was recently announced by FDA Commissioner Dr. Scott Gottlieb, the P4A team discuss key implications of these guidelines along with an update on UK's Brexit Whitepaper and new measures by the French government to improve patient access to innovative drugs.
Presenter: Joanna Fernandes
Contributors: Christina Poschen & Aparna Krishnan2018-07-1708 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: July 11, 2018This week, the team discuss the U.S. Centres for Medicare & Medicaid Services withdrawing support for a novel payment model for Novartis' Kymriah; Pfizer rolling back drug price rise in the U.S. and Axovant's deal with Benitec Biopharma.
Presenter: Joanna Fernandes
Contributor: Aparna Krishnan2018-07-1104 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: July 4, 2018This week, the P4A team discuss the CHMP approval of two CAR-T Therapies; Scotland's new rules for ultra-orphan drugs and the reaction to the announcement of National Coverage Analysis for CAR-T drugs in the U.S.
Presenter: Max Rex
Contributors: Sophie Schmitz, Managing Partner & Alison Kneen, Vice President, International Operations2018-07-0409 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: June 26, 2018This week, the P4A team delve into the pharma industry's proposal to the EU on the role of national HTA bodies post harmonization of clinical assessments for innovative drug technologies; the European Medicines Agency's new portal for orphan drug designation applications and Ireland's entry into the Beneluxa Initiative.
Host: Joanna Fernandes
Contributor: Aparna Krishnan2018-06-2604 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: June 19, 2018The P4A team give a summary of the gene therapy FDA news this week and also discuss President Trump's drug pricing plans for Medicare and UK cost watchdog NICE's rejection of Crysvita.
Presenter: Max Rex
Contributors: Aparna Krishnan, Joanna Fernandes2018-06-1907 min
P4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: June 12, 2018This week's episode discusses the EU's research and innovation budget; Italy's AIFA responding to physician criticism of new guidelines and Oxford Biomedica's $842 million deal with Axovan Sciences.
Presenter: Max Rex
Contributors:
Aparna Krishnan, Corporate Affairs Lead
Joanna Fernandes, Consultant2018-06-1205 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: June 5, 2018This week the P4A team discuss key advancements in oncology treatments - a genetic test indicating whether chemotherapy is beneficial in a particular breast cancer patient group and successful results of an immunotherapy using T cells in an advanced breast cancer patient. In addition, news involving a potential new sickle cell therapy under development.
Presenter: Max Rex
Contributors: Alison Kneen and Aparna Krishnan2018-06-0504 minP4A Let's Talk Rare: The Life Science PodcastWeekly Roundup: May 31, 2018In its very first podcast of Rare Disease, Cell & Gene Therapy Weekly RoundUp, P4A discusses the US FDA's accelerated regulatory process for gene therapy; President's Trump's proposed drug pricing plan; the European Commission's proposal to amend SPC; the NHS England budget ringfencing; Irish government's attempt to trim the HSE and the new GDPR laws. Max Rex hosts with contributions from Aparna Krishnan, Corporate Affairs Lead and Alison Kneen, Vice President, International Operations.2018-05-3110 min