Let’s Talk Rare

Monthly podcast highlighting:

Georgie Rack

Presenter

Owen Bryant

Presenter

2023 Podcasts Archive

Hello and welcome to this special episode of Let’s Talk Rare: The Life Science Podcast as we celebrate 5 years of bringing you the podcast that is now the number one life science podcast across all platforms. We at Partners 4 Access want to thank every single one of our 35,000 subscribers and all the guests who have graced the podcast from the bottom of our hearts, we would not have gotten here without you. 

A special episode calls for special guests, and joining us today are our in-house expert and Managing Partner at P4A, Sophie Schmitz, our host from 2018-2021, Aparna Krishnan, and Prasan Subedi, Access Strategy Team Lead at Pfizer. Together, we walk down memory lane and relive the high points and challenges of the past five years, and share insights on rare diseases and cell and gene therapies.

Aparna Krishnan Bio:

Aparna has over 10 years of experience in the pharmaceutical and healthcare industry. As part of the senior leadership team, her primary focus is on strategic corporate engagement, legal, financial operations, and human resources. She has previously worked as Manager of Life Sciences at IHS Markit and as Consultant at Parexel’s Commercialization unit, among others specializing in market access, pricing, and reimbursement.

Sophie Schmitz Bio:

Sophie is the managing partner at Partners 4 Access, a company that believes in a sustainable rare disease world, which means helping our clients unlock the true opportunity of orphan drugs and ATMPs. Her 20+ year career spans various disease areas and medical devices, successfully supporting the strategy development and operationalization for orphan drugs. She brings a wealth of commercial and access experience across orphan and cell and gene therapies to the benefit of clients. She plays an active role in advising clients on price and market access strategies, with the ultimate goal of optimizing fair access for all stakeholders in rare diseases.

Presenter
Georgie Rack & Owen Bryant
Contributor
Aparna Krishnan, Sophie Schmitz & Prasun Subedi

Welcome to this month's episode of the Let’s Talk Rare podcast by Partners4Acess. Today, Georgie and Owen are joined by RJ Kedziora, Co-Founder of Estenda Solutions, a leading company specialising in custom software and data analytics for healthcare and medical companies.

Join us as we discuss the role of digital health in improving patient outcomes and the challenges in the healthcare system, particularly access and equity. RJ explains that digital health involves using technology to provide care and treatment to patients, and it has evolved to include digital therapeutics, which are clinically tested and approved solutions. He also emphasises the importance of patient engagement in achieving better outcomes and mentions using technologies like telehealth, continuous glucose monitors, and sensors for remote patient monitoring. RJ highlights the shift towards passive technology that seamlessly collects and processes data, enabling better patient care.

RJ encourages healthcare professionals to stay updated on the latest advancements in healthcare technology by attending conferences, engaging with start-ups, and keeping up with medical and technology journals. Regarding advice for healthcare professionals looking to embrace innovation, RJ emphasises the importance of understanding the patient's perspective and focusing on communication and empathy.

RJ Kedziora Bio:

Mr. Kedziora is the co-founder of Estenda Solutions, a leading company specializing in custom software and data analysis for healthcare and medical companies. With a remarkable journey spanning over 30 years, he possesses a deep understanding of designing, developing, and deploying successful software projects. His extensive experience enables him to provide valuable guidance and innovative insights, resulting in cost-effective solutions that improve patient outcomes.

Mr. Kedziora received his M.B.A. from West Chester University and a Bachelor of Science in Computer Science from Duquesne University where he received the Excellence in Computer Science Award. He has spoken at numerous technical and healthcare conferences on a variety of topics and written or co-authored multiple articles focused on healthcare information technology, several published in peer-reviewed scientific journals.

Social Media Links:

FACEBOOK (BUSINESS): https://www.facebook.com/EstendaSolutions

LINKEDIN: https://www.linkedin.com/in/rjkedziora/

INSTAGRAM: https://www.instagram.com/estendasolutions/

TWITTER: https://twitter.com/Estenda

YOUTUBE: https://www.youtube.com/channel/UCuAfCbIFW0DmeI5PDS1PXcg

Presenter
Georgie Rack & Owen Bryant
Contributor
RJ Kedziora

Welcome to this month's episode of the Let’s Talk Rare podcast by Partners4Acess. Georgie and Owen are joined by Iola Forster, Head of Industry and Society Markets at Karger 23Publishers, to discuss the future of patient engagement and the role of technology in healthcare.

Together, they cover the future of HCPs (healthcare practitioners) and patient engagement, highlighting the importance of technology and patient empowerment in driving this evolution. They also emphasize the need for equitable access to healthcare information.

The conversation touches on the changing landscape of patient communication over the past fifteen years, with the advent of digital platforms and social media. They discuss how digitization has democratized access to health information and created opportunities for patients to connect with others facing similar conditions.

They further explore the impact of digitization on the orphan drug and cell and gene therapy world, emphasizing the role of patient involvement in content creation and the use of multimedia resources like infographics to cater to diverse learning styles.

Iola Forster Bio:

Iola Forster is the Head of Industry and Society Markets at Karger Publishers. She has over fifteen years of experience in the publishing industry and is skilled at fostering partnerships between the life sciences, healthcare clients, and CARGA. Iola brings a wealth of knowledge and expertise to the table.

Episode Resources:

Iola Forster on LinkedIn

Karger Publishers website

Partners4Access website

P4A Let’s Talk Rare podcast on Apple

Presenter
Georgie Rack & Owen Bryant
Contributor
Iola Forster

Welcome to this month's episode of the Let’s Talk Rare podcast brought to you by Partners4Acess. Georgie and Owen are joined by Chloe Sheppard and Akshay Kumar to discuss the new EU HTA regulation set to be implemented in 2025. They explore the implications for drug developers of ATMPs, the attractiveness of the EU market, and the importance of proactive preparation for the upcoming changes.

The topics covered include: key milestones of the EU HTA regulation; the importance of drug developers of ATMPs and oncology medicines to not adopt a wait and watch strategy; the risks of ignoring this change and the potential impact on clinical development plans, commercialization strategies, and patient access; the opportunities the EU market presents; and the need to start planning and adapting internal processes to successfully navigate the new regulatory landscape.

Chloe Sheppard Bio:

Chloe serves as a Senior Consultant at Partners4Access, working closely with clients to craft custom solutions for their pricing and market access obstacles. Her motivation lies in transforming the immense potential of advanced medicines into tangible therapeutic benefits for patients.

Akshay Kumar Bio:

Akshay, a Partner at Partners4Access, brings over 13 years of experience assisting pharmaceutical companies in devising market access and pricing strategies that align with their broader commercial and organizational contexts. His expertise spans not only product-level solutions but also process improvements and capability enhancement. In addition to market access, Akshay is well-versed in various other aspects of drug commercialization, including opportunity assessments, product positioning, forecasting, customer insights, and organizational design.

Episode Resources:

Chloe’s LinkedIn

Akshay’s LinkedIn

Partners 4 Access Website

Presenter
Georgie Rack & Owen Bryant
Contributor
Akshay Kumar & Chloe Sheppard

In this episode we will be talking to Alejandro Dorembaum, CMO of Reneo Pharmaceuticals. Alex discusses how the company is developing drugs for patients with rare mitochondrial diseases a high unmet disease with no current treatments available. Their lead candidate Mavodelpar has recently completed enrolment for their pivotal STRIDE clinical trial study. STRIDE is a global, randomized double-blind, 6 months, placebo-controlled trial designed to assess the efficacy and safety of Mavodelpar. Alex discusses the importance of engaging early with patients and patient organisations for PMM across the world to truly understand the patient journey, their challenges & daily routines to help shape the study design, to ensure successful enrolment and patient retention throughout the clinical trials. This is something Reneo have successfully completed throughout the development of Mavodelpar, by engaging early with patients, clinicians & regulators, they have completed enrolment in record time, this is especially difficult for rare diseases as it is often difficult to find the patients and something Alex is extremely proud of. We look forward to following Reneo's journey and hope to have Alex back early next year to discuss their global commercial plans for PMM patients.

Reneo Pharmaceuticals: https://reneopharma.com/

Reneo is a clinical-stage pharmaceutical company focused on the development of therapies for patients with rare genetic diseases including mitochondrial diseases with significant unmet medical needs.

LinkedIn: Alejandro Dorenbaum

https://www.linkedin.com/in/alejandro-alex-dorenbaum-m-d-b01a6ab/

 

Presenter
Georgie Rack
Contributor
Alejandro Dorenbaum

2022 Podcasts Archive

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

Presenter
Georgie Rack & Owen Bryant
Contributor
Alexander Natz, Leon van Wouwe, Wing-yun Cheung, Chloe Sheppard & Joanna Fernandes

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!

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.

Presenter
Georgie Rack & Owen Bryant
Contributor
Stefan Walzer & Fisentzos Stylianou

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)

Presenter
Georgie Rack & Owen Bryant
Contributor
Sophie Schmitz, Neil Bertelsen and Laurence Woollard

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 toto do this and the benefits of doing so.

Host: Georgie Rack & Owen Bryant

Guests: Laurence Woollard, On the Pulse, Neil Bertelsen, Independent Consultant, Sophie Schmitz, Managing Partner at P4A

Links:

“Moving beyond box-ticking and lip-service - why patient involvement matters in a new era of ATMPs for rare diseases”

“Setting priorities for bleeding disorders - Final report”

“More than a Top 10: How James Lind Alliance Priority Setting Partnerships transform research, people and organisations”

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. He has published numerous thought-leadership articles on the theory and practice of patient empowerment and engagement across leading industry and advocacy platforms as well as peer-reviewed journal manuscripts related to patient education priorities and informed consent for haemophilia gene therapy. Laurence is a member of the Patient Engagement Committee for Beacon (formally Findacure), a UK based charity supporting capacity building of rare and ultra-rare patient groups to drive research and develop treatments. He is also a Patient Voice Partner on the Advanced Therapy Medicinal Product (ATMP) Patient and Public Involvement and Engagement (PPIE) Group by the Gene and Cell Therapy Catapult (supported by Genetic Alliance UK). Laurence is highly driven by his own journey and challenges of living with severe haemophilia and the impact on the family dynamic, to campaign for and effect real change in improvements to quality of life and care for his peers.

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. Neil is currently on the steering committee of this group. Neil is also a board member of PFMD (Patient Focused Medicines Development), an international consortium of stakeholders working towards more integrated inclusion of the patient voice across the medicine lifecycle. Neil also recently authored the PARADIGM toolkit for patient involvement in Early Dialogues with HTA bodies. During this work, Neil partnered with eleven HTA bodies to develop a range of tools to assist in the involvement of patients during early discussions between HTA bodies and medicine developers. Neil works directly with the patient advocacy community, the industry, and authorities such as HTA agencies to facilitate collaborations and co-creation of approaches to improve access to healthcare and better provision of care. As a facilitator of meetings and advisory boards, Neil has global experience working with multiple stakeholders on demanding issues that require a coordinated response. Neil has worked within the industry, as a member of the Global Market Access team at Bayer Pharmaceuticals, where he worked on bringing the patient voice into access strategies for pipeline products as well as working with regulatory and R&D colleagues on ensuring that evidence generation plans would capture meaningful benefits. Further background Neil was the managing director of several specialist health care consultancies where he developed integrated strategy, education, communication, and engagement programs that considered the patients as an equal partner in the health care landscape. Neil served for six years on the board of trustees of Terrence Higgins Trust, Europe’s largest HIV and sexual health advocacy group. In the 1990s Neil was the editor of Positive Times, the UK’s first national magazine for people with HIV. This pioneering magazine tackled the relevant issues of the day in a time that spanned the era before the advent of combination therapy, through to its successful introduction. Neil has also been editor and deputy editor of several peer review journals in the field of physics and mathematics. He was also a successful television director and producer making science documentaries that brought new science to life for the public and patients. This included producing and directing ‘Horizon’ for the BBC, where he was part of the team awarded a BAFTA (British Academy of Film and Television Awards) for best factual series

Sophie Schmitz, Managing Partner P4A

Sophie is Managing Partner at Partners4Access (P4A), a global consultancy 100% focused on orphan drug access. P4A support the biotechnology and pharmaceutical industry along their launch journey to help secure successful price, reimbursement, and access for orphan drugs. The company has solid partnerships with clients supporting their strategy and operations to effectively ensure launch success. Sophie has worked in a range of disease therapy areas and has industry experience in both medical device and pharmaceutical industries, as such she has developed a solid appreciation of strategic challenges and opportunities facing companies. Through her time in industry, she worked on several key product launches in both strategic planning and implementation. She has worked in global, regional, and local teams which gives her a strong appreciation of real-life challenges in modern day healthcare organizations. Sophie’s has enjoyed a successful 20-year commercial career within consultancy and industry. Prior to setting up Partners4Access, she worked at Alliance, PriceSpective, BMS, ConvaTec, SSL International and Smith & Nephew. In this time, Sophie has been involved in several product launches, organizational changes, and acquisitions.

Presenter
Georgie Rack & Owen Bryant
Contributor
Sophie Schmitz, Neil Bertelsen and Laurence Woollard

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/

 

Presenter
Georgie Rack
Contributor
Owen Bryant

2021 Podcasts Archive

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

Presenter
Georgie Rack
Contributor
Sophie Schmitz and Joanna Fernandes

This episode featuring James Mackay, President & CEO of Aristea Therapeutics discusses and 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.

Presenter
Aparna Krishnan
Contributor
James Mackay

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! 

Producer: Operations Team 

Presenter
Richard Wang
Contributor
Adama Anozie

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!

Producer: Operations Team

Presenter
Georgie Rack
Contributor
Bruce Chin

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.

Producer: Operations Team

Presenter
Akshay Kumar
Contributor
Richard Wang

2020 Podcasts Archive

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!

Producer: Aparna Krishnan

Presenter
Sophie Schmitz
Contributor
Akshay Kumar

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, its 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

Sponsorships: off

2019 Podcasts Archive

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; a 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

2018 Podcasts Archive

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

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