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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 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 ex…
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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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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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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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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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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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This 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 Contrib…
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The 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 ge…
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In 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 All…
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The 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 Fern…
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This 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…
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The 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 respon…
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This 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 Krishnan…
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