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P4A Let's Talk Rare

P4A Let's Talk Rare

By Partners4Access
A monthly podcast highlighting the most important news developments and its impact on the orphan drug, cell and gene therapy world. Visit
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Monthly RoundUp - September 2021

P4A Let's Talk Rare

Part 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? 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. 
September 13, 2022
What 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 Bryant, Creative Director Produced By: Operations Awards P4A have won in 2022 (so far!) - 1. BOBI Awards 2022 - Analyst Team of the Year Link: 2. Benelux Enterprise Awards 2022- Best Orphan Drug & Gene Therapy Experts Link: 3. International Life Science Awards 2022 - Best Cell & Gene Therapy Global Market Access Consultancy Link: 4. Healthcare & Pharmaceutical Awards 2022 - Leading Experts in Orphan Drug & Gene Therapy Access Link:
July 29, 2022
Special Episode with EUCOPE-Part 2 with Alexander Natz
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? 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: LinkedIn: 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:
June 30, 2022
Special series with Eucope: Part 1 featuring Victor Maertens
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: 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: 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.
May 31, 2022
China 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: Presenter: Aparna Krishnan, Partner – Global Operations Contributor: Fisentzos Stylianou, Analyst Producer: Operations team
April 29, 2022
Should 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 team
March 31, 2022
Rare Disease Day Special featuring Janet Bloor and Nick Meade - February 2022
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
February 28, 2022
P4A Insights: Trends to look out for in 2022
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
February 04, 2022
Special 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 team
December 13, 2021
Special episode featuring James Mackay - November 2021
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.
November 30, 2021
Monthly Roundup - October 2021
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
October 29, 2021
Monthly RoundUp - September 2021
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
September 30, 2021
Monthly RoundUp: July 2021
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
July 30, 2021
Monthly RoundUp - June 2021
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
June 30, 2021
Monthly RoundUp - May 2021
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
May 31, 2021
Monthly RoundUp - April 2021
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   
April 30, 2021
Monthly RoundUp - March 2021
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
March 31, 2021
Monthly RoundUp - February 2021
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
February 27, 2021
Rare Disease Day special episode: Reclaiming the rare disease patient’s voice
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 Nicola Whitehill's blog:  Dylan Myers' story:  and
January 29, 2021
End of year 2020 review
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
December 21, 2020
Special episode: Gene Therapy access from a specialty pharmacy perspective
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.
June 22, 2020
Weekly RoundUp: February 29, 2020
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  Presenter and Producer: Aparna Krishnan Contributor: Annie Kennedy, Chief of Policy & Advocacy, EveryLife Foundation for Rare Diseases
February 29, 2020
Weekly Roundup: December 30, 2019
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
December 30, 2019
Weekly Roundup: December 23, 2019
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
December 23, 2019
Weekly Roundup: November 12, 2019
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
November 12, 2019
Weekly Roundup: November 5, 2019
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
November 05, 2019
Weekly RoundUp: August 5, 2019
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   
August 05, 2019
Weekly Roundup: July 29, 2019
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
July 29, 2019
Weekly Roundup: July 22, 2019
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
July 24, 2019
Weekly Roundup: July 1, 2019
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
July 01, 2019
Weekly Roundup: June 23, 2019
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
June 23, 2019
Weekly Roundup: June 16, 2019
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
June 16, 2019
Weekly Roundup: June 12, 2019
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
June 12, 2019
Weekly RoundUp: June 3, 2019
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
June 03, 2019
Weekly Roundup: May 25, 2019
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
May 25, 2019
Weekly Roundup: May 18, 2019
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
May 18, 2019
Weekly Roundup: May 12, 2019
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  
May 12, 2019
Weekly Roundup: May 3, 2019
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  Presenter: Aparna Krishnan Contributor: Scott Dorfman, CEO, Odylia Therapeutics Producer: Aparna Krishnan
May 03, 2019
Weekly Roundup: April 27, 2019
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
April 27, 2019
Weekly 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, Terrapinn
April 22, 2019
Weekly Roundup: April 12, 2019
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
April 17, 2019
Weekly Roundup: March 28, 2019
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
March 28, 2019
Weekly Roundup: March 22, 2019
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
March 22, 2019
Weekly Roundup: March 15, 2019
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
March 15, 2019
Weekly Roundup: March 8, 2019
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
March 08, 2019
Weekly Roundup: February 28, 2019
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
February 28, 2019
Weekly Roundup: February 22, 2019
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
February 22, 2019
Weekly 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 health technology assessment analyst and reimbursement decision maker for the national insurance scheme in Norway.  
February 15, 2019
Weekly Roundup: February 1, 2019
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
February 01, 2019
Weekly 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 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
January 25, 2019
Weekly Roundup: January 18, 2019
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
January 18, 2019
Weekly Roundup: January 11, 2019
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
January 14, 2019
Weekly Roundup: December 14, 2018
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.
December 14, 2018
Weekly Roundup: December 7, 2018
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
December 07, 2018
Weekly Roundup: November 30, 2018
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
November 30, 2018
Weekly Roundup: November 23, 2018
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
November 23, 2018
Weekly Roundup: November 15, 2018
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
November 15, 2018
Weekly Roundup: November 9, 2018
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
November 09, 2018
Weekly Roundup: November 2, 2018
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
November 02, 2018
Weekly Roundup: October 25, 2018
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
October 25, 2018
Weekly Roundup: October 18, 2018
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
October 18, 2018
Weekly Roundup: October 11, 2018
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
October 11, 2018
Weekly Roundup: Oct 5, 2018
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
October 05, 2018
Weekly Roundup: Sep 27, 2018
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
September 27, 2018
Weekly Roundup: September 20, 2018
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
September 20, 2018
Weekly Roundup: August 16, 2018
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
August 16, 2018
Weekly Roundup: August 9, 2018
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
August 09, 2018
Weekly Roundup: August 2, 2018
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
August 02, 2018
Weekly Roundup: July 26, 2018
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
July 26, 2018
Weekly Roundup: July 17, 2018
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
July 17, 2018
Weekly Roundup: July 11, 2018
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
July 11, 2018
Weekly Roundup: July 4, 2018
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
July 04, 2018
Weekly Roundup: June 26, 2018
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
June 26, 2018
Weekly Roundup: June 19, 2018
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
June 19, 2018
Weekly Roundup: June 12, 2018
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
June 12, 2018
Weekly Roundup: June 5, 2018
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
June 05, 2018
Weekly Roundup: May 31, 2018
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.
May 31, 2018