P4A Let's Talk Rare

In this episode of Let’s Talk Rare, as we commemorate World Rare Disease Day, Georgie and Owen are joined by Louise Fish and Dan Lewi to discuss all aspects surrounding rare diseases, from key challenges patients face in getting access to life-saving medicines, the clinical trial burden, EU pharmaceutical strategy and more. The topics covered include: Why genetic screening of newborns in the UK lags behind the EU, funding rare disease research, clinical trial burden, patient registries, patient experience data and its relevance, revamping data sharing with families, and how pharma can involve patients earlier. Louise Fish Bio: Louise is a senior leader with over 25 years of executive and non-executive board experience in health and social care in charitable, public and commercial sectors. She is passionate about improving the NHS and social care services by listening to and learning from the experiences of patients and their families. She has a strong understanding of how to drive change. Dan Lewi Bio: Dan is the Chief Executive and co-founder of the only Tay-Sachs and Sandhoff disease-specific charity in the UK called The Cure & Action for Tay-Sachs (CATS) Foundation. Selected and appointed as the Chairman of the European Tay-Sachs and Sandhoff Charity Consortium (ETSCC), which has brought together all the Tay-Sachs and Sandhoff charities in Europe.

The P4A team have hand-picked trends to look out for in 2023. This episode we are joined by special guest Neil Grubert who will be discussing the topics in further detail, alongside our P4A experts, Shrishti & Jodie. The topics covered are: EU Pharmaceutical Strategy, OMP Legislation, Joint HTA Assessments - cross-country HTA cooperation, Expansion of CAR-T & Hospital Exemptions across Europe and finally the Patient - Putting patients at the forefront of drug developers. Neil Grubert Bio:  Neil has worked in Industry for over 20 years starting at Decision Resources as a researcher in 1997 and leaving as Vice President of Global Market Access Insights in 2013. Currently he is an independent MA consultant and has been doing this for the last 8 years. LinkedIn: https://www.linkedin.com/in/neil-grubert/ Hosts: Georgie Rack & Owen Bryant P4A Speakers: Srishti Gupta, Senior Consultant & Jodie Lyons, Analyst Produced by: Operations

Did you miss the World Orphan Drug Congress Europe 2022, held in Sitges, Barcelona in November? If you did, don't worry P4A have you covered! This month's special  podcast was part recorded live at the conference and part recorded back in London with members of the team that attended! We will be speaking to a few presenters & sponsors to discuss their presentations, key learnings and their personal highlights from the conference. Also the P4A team will be talking about our favourite sessions and the key takeaways from the conference. Special thanks to our guests for their insights and learnings throughout the podcast. Presenters: Georgie Rack & Owen Bryant Guests: Alexander Natz, Secretary General at EUCOPE - https://www.eucope.org/ Leon van Wouwe, Clinical Innovation Director at VOLV Global https://www.volv.global/ Wing-yun Cheung, General Manager at Terrapinn & organiser of WODC EU - https://www.terrapinn.com/conference/world-orphan-drug-congress/index.stm Chloe Sheppard, Consultant at P4A Joanna Fernandes, Senior Consultant at P4A

 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 market within Europe and what does this means for rare disease patients?  Will there be delays to new treatments or will manufacturers decide not to launch in Germany at all to protect the price of their new drug?  We will be discussing this and so much more! If you are a drug manufacturer planning your launch strategy, this podcast is for you!  Presenters: Georgie Rack & Owen Bryant Guests Stefan Walzer, CEO President & Founder of MArS Market Access & Pricing Strategy GmbH Fisentzos Stylianou, senior analyst and P4A's German country expert Dr Stefan Walzer, CEO President & Founder at MArS Market Access &Pricing Strategy GmbH https://marketaccess-pricingstrategy.de/en/ Bio & Fun Facts  1) Economist, PhD in health economics, diploma in clinical trials 2) Experience in MA, reimbursement, HE and pricing since 2004 in consultancy and industry 3) Founder and CEO of MArS - THE D-A-CH market access consultancy. Linked to that also co-founder of SMS2DACH including the full spectrum for D-A-CH support (distribution, management, launch, etc.): www.sms2dach.com 4) Founder of P&N (pricing-and-negotiations.ca) with a focus on negotiations across the world including being the co-founder of www.thenegotiationlab.com 5) Member of a European network Tesseract (https://www.tesseracteurope.com/) which can serve companies moving from outside Europe into Europe including not only reimbursement services but also logistics, customs, etc. 6) Teaching at various German universities 7) Love spending time with my family, being a soccer coach of under 14 years-old and being a supporter of Borussia Dortmund Fisentzos Stylianou, Senior Analyst & German Country Expert Fisentzos role at Partners4Access includes conducting primary and secondary research to support the development of market access and reimbursement strategies for clients in the pharmaceutical and biotechnology industries. With a passion for innovative treatments, he closely follows the cell and gene therapy field as it expands to treat more patients with rare diseases.  Prior to joining Partners4Access, Fisentzos worked as a Research Associate at Imperial College London, where he also earned his Ph.D. in Structural Biology in 2020. During this time, he conducted research as part of a multidisciplinary team to advance his understanding of the structure and function of biofilm-forming proteins, paving the way for the design of novel antimicrobial therapeutics. Fisentzos also holds an M.Sc. in Biomedical and Molecular Sciences Research and a B.Sc. in Biomedical Science, both from King’s College London. 

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 offering to try to bridge the gaps and barriers for sustainable patient access. P4A’s’’Let’s talk rare’’ monthly podcasts are available wherever you listen to your podcasts. If you are a drug manufacturer and you haven’t yet listened, this needs to be at the top of your to do list! If you missed out on part 1 - listen here: https://spotifyanchor-web.app.link/e/hOBI3UqkPtb Hosts: Georgie Rack (G-Rack)  & Owen Bryant (OB1) Produced by: Operations Team Links: P4A’s PCAC: https://partners4access.com/services/pc-council/ Part One: Patient Empowerment: https://spotifyanchor-web.app.link/e/hOBI3UqkPtb ABPI article in recognition of Rare Disease Day 2022 - “Moving      beyond box-ticking and lip-service - why patient involvement matters in a      new era of ATMPs for rare diseases” James Lind Alliance Priority Setting Partnership - “Setting       priorities for bleeding disorders - Final report” (2018) “More than a Top 10: How James Lind Alliance Priority Setting Partnerships transform research, people and organisations” (2019)

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? The second part will solely focus on the WHY! Why should drug manufacturers, payors, regulators and HTA bodies involve and listen to the patients? What do they bring to the table? We will also dive into the implications of not including the patient voice and use examples of companies that have successfully managed to do this and the benefits of doing so. Laurence Woollard, Owner On The Pulse Laurence Woollard is founder and director of On The Pulse – an independent, strategic consultancy providing specialist insight to global healthcare providers and multi-agencies on the development and rollout of patient activation campaigns in haemophilia and rare disease. Laurence has extensive patient advocacy experience, having advised on and implemented a broad scope of community awareness, research and educational initiatives for commercial partners and third-sector organisations.  Neil Bertelsen, Independent Consultant Neil Bertelsen is an independent consultant and patient advocate with over 25 years of experience bringing the patient voice to health care decision makers and communicating the science of health care to patients in a way that truly informs their own personal health choices. Neil is passionate about bringing the patient experience and perspectives to decision-makers including industry and health technology assessment (HTA) bodies. Neil is the past chair of HTAi (the global scientific society of HTA) Patient and Citizen Involvement Interest Group. This is an international multi-stakeholder group whose remit is to work alongside HTA organizations and patient organizations to bring patient involvement processes and patient insights and experiences into access decision making processes. Sophie Schmitz, Managing Partner P4A Sophie is managing partner at Partners4Access(P4A),an award winning global consultancy focused on access for orphan drugs & ATMPS. P4A support the biotechnology & pharma industry along their launch journey to help secure successful price, reimbursement and access for orphan drugs & ATMPs. The company has solid partnerships with clients supporting their strategy & operations to effectively launch success. 

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 Bryant, Creative Director Produced By: Operations Awards P4A have won in 2022 (so far!) - https://partners4access.com/about/awards/ 1. BOBI Awards 2022 - Analyst Team of the Year Link: https://www.bhbia.org.uk/bobi-awards/award-winners/bobi-2022 2. Benelux Enterprise Awards 2022- Best Orphan Drug & Gene Therapy Experts Link: https://www.eubusinessnews.com/awards/benelux-business-awards/#ourwinners 3. International Life Science Awards 2022 - Best Cell & Gene Therapy Global Market Access Consultancy Link: https://www.ghp-news.com/awards/international-life-sciences-awards/ 4. Healthcare & Pharmaceutical Awards 2022 - Leading Experts in Orphan Drug & Gene Therapy Access Link: https://www.ghp-news.com/awards/healthcare-pharmaceuticals-awards/

In this episode Chloe & Alexander will be starting the discussion with an overview of EU HTA regulation, looking at the harmonization debate and how this came about. We then discuss JCA evaluation methodology (e.g. comparator selection, acceptability of clinical evidence), the potential new role of individual country agencies and how the joint HTA process will link to national processes. EUnetHTA21 and how the methodology is evolving with G-BA & IQWIG increasingly involved, will this be a major influence in the development of final methodology? Finally, we look at how Pharma companies should be best preparing for the implementation of the regulation. Host: Chloe Sheppard Guest Speaker: Alexander Natz Produced by: Operations team Helpful links: #EUHTAPriorities – EUCOPE’s Five Priorities for a Successful EU HTA Procedure Sounds of Science Podcast – Episode 1: A Future-Proof HTA system in Europe? https://partners4access.com/whitepapers/pan-european-joint-hta-what-does-the-future-hold-for-innovative-therapies/ About Alex: Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) in Brussels and advises innovative pharmaceutical and biotech companies, including start-ups, in regulatory and pricing & reimbursement matters from the EU law and German law perspective. From 2008 to 2013, he was Head of the Brussels Office of Bundesverband der Pharmazeutischen Industrie e.V. (BPI). Before, he worked as a lawyer at Sträter Law Firm in Germany with a special focus on managed entry agreements and licensing of pharmaceuticals. Dr. Natz has also worked in the field of competition law with the European Commission and in the pharmaceutical industry. As a research assistant at Duke University (USA) he has dealt with international pharmaceutical law. His doctorate was supervised by the former judge at the European Court of Justice, Prof. Dr. Dr. Ulrich Everling Email: natz@eucope.org LinkedIn: https://www.linkedin.com/in/alexander-natz-63467433/ About EUCOPE: For more than a decade, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) has been the voice of small to mid-sized health technology companies in Europe. As a trade association, we advocate for sound public policy that supports innovation, while fostering a community built on a shared purpose: improving and saving the lives of European patients through innovative therapies and medical technology. Website: https://www.eucope.org/

Part 1 featuring Victor Maertens, Government Affairs Manager at EUCOPE In this episode Victor and Aparna will be focusing on Advanced Therapy Medicinal Products (ATMPs). We will start by looking at the main challenges for manufacturers in the ATMP approval process and what is required from an HTA perspective. We then discuss EUCOPE’s position paper on the different payment models, including the motivation for the report and key learnings. We look at sustainable patient access and whether the report addresses the affordability issues surrounding ATMP’s drug pricing and if this is even possible in the current landscape. The EU’s revision of Blood, Tissue & Cells legislation and lastly the future of ATMPs for manufacturers and patients. Host: Aparna Krishnan Guest speaker: Victor Maertens Produced by: Operations team About Victor Maertens: https://www.linkedin.com/in/victormaertens/ Victor Maertens is the Government Affairs Manager for the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE). Victor leads EUCOPE’s policy work on the Pharmaceutical Strategy and in the area of Cell and Gene Therapy. Prior to joining EUCOPE, Victor worked for Brussels-based consultancies, providing pharmaceutical companies, trade associations, and NGOs advocacy, strategy, and communications advice. His experience and interests include ATMPs, antimicrobial resistance, Orphan medicinal products (OMP), blood policy, and neglected tropical diseases. About Eucope: https://www.eucope.org/ For more than a decade, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) has been the voice of small to mid-sized health technology companies in Europe. As a trade association, we advocate for sound public policy that supports innovation, while fostering a community built on a shared purpose: improving and saving the lives of European patients through innovative therapies and medical technology.

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 Operations Contributor: Fisentzos Stylianou, Analyst Producer: Operations team

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 team

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 Duchenne Nexus Advocacy (DNA) and Nick Meade, Director of Policy and Joint Interim Chief Executive of Genetic Alliance UK Producer: Operations team

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 Bernardini Produced by: Ops team

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 team

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 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.

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 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 Team

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 Producer: Operations Team

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 the industry. Presenter: Akshay Kumar Contributors: Richard Wang Producer: Operations Team

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 access. Listen in to learn more.  Presenter and Contributor: Ciaran Cassidy Producer: Aparna Krishnan

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 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 Krishnan

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 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 body NICE's (National Institute for Health and Care Excellence's) method review.  Presenter and Contributor: Joanna Fernandes Other contributors: Jayne Watson Producer: Aparna Krishnan   

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 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 with a multi-stakeholder approach will provide the recipe for success for next generation`niche-busters'. Presenter and Contributor : Akshay Kumar Other contributors: Richard Wang, Andrea Bernardini and Erfan Akbraian Producer: Aparna Krishnan

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

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) 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 Myers Producer: Aparna Krishnan More information on : Genetic Alliance can be found at https://geneticalliance.org.uk/ Nicola Whitehill's blog: https://blog.raynaudsscleroderma.co.uk/2017/04/scleroderma-raynauds-rare-disease.html?m=1  Dylan Myers' story: https://m.facebook.com/dylansstory/  and https://www.treeofhope.org.uk/dylansstory/

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 up of 2020. So do listen in! Presenter and Contributors: Sophie Schmitz  and Akshay Kumar Producer: Aparna Krishnan

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 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 provide Zolgensma, the first gene therapy for Spinal Muscular Atrophy. Today, Orsini has access to 40 limited distribution drugs, has more than 525 direct payor contracts and is recognized as a reimbursement expert in managing both medical and pharmacy benefits.

This 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 Diseases

As 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 Krishnan

The 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 Krishnan

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 gene therapies among HTA bodies and Alliance for Regenerative Medicines’ Q3 report. Presenter: Jack Rawson Contributor: Sophie Schmitz Producer: Aparna Krishnan

Following 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 Krishnan

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 Alliance of Regenerative Medicine Producer: Aparna Krishnan   

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 Fernandes Producer: Aparna Krishnan

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 Contributor: Max Rex Producer: Aparna Krishnan

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 response and its ramifications.  Presenter: Aparna Krishnan Contributor: Max Rex Producer: Aparna Krishnan

This 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 Krishnan

This 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 Krishnan

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

On 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 Krishnan

In 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 Krishnan

This 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 Krishnan

This 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  

This 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 Krishnan

This 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

 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, Terrapinn

This 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 Krishnan

The 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 Krishnan

This 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 Krishnan

This 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 Krishnan

This 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 Krishnan

On 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 Krishnan

In 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 Krishnan

 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 health technology assessment analyst and reimbursement decision maker for the national insurance scheme in Norway.  

Throughout 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 through examples of different types of collaborations. From patient and clinical experts influencing HTA decisions; key stakeholders coming together for the Hercules Project to a collaboration between US based ICER, Canada's CADTH and UK's NICE. Presenter: Aparna Krishnan Contributors: Sophie Schmitz, Christina Poschen, Max Rex, Joanna Fernandes, Nader Murad and Jack Rawson

 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 series of podcast episodes featuring thought leaders representing these stakeholders. So make sure you listen in! Presenter: Joanna Fernandes Contributors: Nader Murad and Sophie Schmitz Producer: Aparna Krishnan

This 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 Krishnan

Happy 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 initiative on assessments for innovative drugs.  Presenter : Max Rex Contributor : Joanna Fernandes Producer: Aparna Krishnan

In 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.

This 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 Krishnan

This 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 Rawson

The 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 Schmitz

In 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, Belgium

In 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 Development Manager at Terrapinn

The 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 Rawson

This 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 Fernandes

This 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, P4A

The 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 Analyst

This 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, Pfizer

This week, P4A's podcast will cover Luxturna's CHMP recommendation, Novartis' restructuring plans and Alexion's acquisition of Syntimmune. Presenter: Max Rex Contributor: Aparna Krishnan

Back 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 Krishnan

In 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 Krishnan

In 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 Krishnan

This 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 Krishnan

The 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 Tutt

The 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 Krishnan

This 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 Krishnan

This 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 Operations

This 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 Krishnan

The 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 Fernandes

This 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, Consultant

This 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 Krishnan

In 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.