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P4A Let's Talk Rare: The Life Science Podcast

P4A Let's Talk Rare: The Life Science Podcast

By Partners4Access

Welcome to P4A Let’s Talk Rare, a monthly podcast highlighting the most important developments in the world of rare diseases orphan drug, cell and gene therapy, hosted by Georgie Rack and Owen Bryant of Partners For Access. To find out more about Partners For Access and our commitment to sustainable orphan drug access for patients with high unmet need, visit partners4access.com
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Weekly Roundup: June 5, 2018

P4A Let's Talk Rare: The Life Science PodcastJun 05, 2018

00:00
04:38
Rare Disease Day Special 2024: The Role of Genetic Diagnosis in Rare Conditions with Nick Meade

Rare Disease Day Special 2024: The Role of Genetic Diagnosis in Rare Conditions with Nick Meade

Welcome to this month's episode of the Let’s Talk Rare: The Life Science Podcast by Partners4Acess. Georgie and Owen are joined by Nick Meade, Head of Policy at Genetic Alliance, to discuss the challenges faced by rare patients in accessing life-saving medicines.

Together they explore the EU joint HTA legislation coming into force in January 2025, and the importance of patient experience data. Nick explains that the challenges for rare patients remain the same, with diagnosis being the first hurdle. He highlights the progress being made in genetic diagnoses and screening but emphasizes the need for more harmonization in Europe. 


Finally, they touch on the concept of patient experience data, which refers to the inclusion of patient perspectives and outcomes in decision-making processes. Nick emphasizes the importance of this data in understanding rare conditions and leveraging it for better access to treatments. They also discuss the various events and initiatives planned by Genetic Alliance for  the upcoming Rare Disease Day on February 29th, to raise awareness and share stories of rare conditions.


Nick Meade Bio:

Nick Meade is Head of Policy at Genetic Alliance, the largest alliance of organizations supporting people with genetic, rare and undiagnosed conditions in the UK. He has over twenty years of experience in the field and is a member of the NIHR Advanced Therapy Medicinal Product (ATMP) Group. 


Episode Resources:

Nick Meade on LinkedIn

Genetic Alliance website

Partners4Access website  

P4A Let’s Talk Rare podcast on Apple



Feb 29, 202424:32
Celebrating 5 Years of P4A: Reflections on Rare Diseases, Cell and Gene Therapies, and Legislation in Europe

Celebrating 5 Years of P4A: Reflections on Rare Diseases, Cell and Gene Therapies, and Legislation in Europe

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


A special episode calls for special guests, and joining us today are our in-house expert and Managing Partner at P4A, 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.


Jan 02, 202453:45
Revolutionizing Patient Outcomes: The Power of Digital Health Solutions with RJ Kedziora

Revolutionizing Patient Outcomes: The Power of Digital Health Solutions with RJ Kedziora

In this podcast, Georgie, RJ, and Owen discuss revolutionizing patient outcomes and the power of digital health solutions. RJ, a healthcare industry expert, shares insights on the role of technology in improving patient care and the future of digital health. They also touch on RJ's involvement in triathlons and his passion for running. With a friendly and engaging tone, this podcast offers valuable insights into healthcare and the potential of digital solutions.


RJ Kedziora Bio:

Mr. Kedziora is the co-founder of Estenda Solutions, a leading company specializing in custom software and data 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


Dec 01, 202328:03
The Future of Patient Engagement and the Role of Technology in Healthcare with Iola Forster

The Future of Patient Engagement and the Role of Technology in Healthcare with Iola Forster

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

Together, they cover the future of HCPs (healthcare practitioners) and patient 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 Publications and Portfolio 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 Karger. 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

Oct 06, 202323:39
Navigating the New EU HTA Regulation and Its Impact on ATMP Development

Navigating the New EU HTA Regulation and Its Impact on ATMP Development


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

The topics covered include: key milestones of the EU HTA regulation; the importance of drug developers of ATMPs and oncology medicines to not adopt a wait and watch 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



Jul 31, 202316:36
Alejandro Dorenbaum, CMO of Reneo Pharmaceuticals Discusses Clinical-Stage Therapies

Alejandro Dorenbaum, CMO of Reneo Pharmaceuticals Discusses Clinical-Stage Therapies

In this episode we will be talking to Alejandro (Alex) Dorenbaum, M.D., CMO of Reneo Pharmaceuticals. Alex discusses how the company is developing drugs for patients with #rare mitochondrial diseases, a high unmet disease with no current treatments available. Their lead candidate #Mavodelpar has recently completed enrolment for their pivotal STRIDE clinical trial. STRIDE is a global, randomized double-blind, 6 months, placebo-controlled trial designed to assess the efficacy and safety of Mavodelpar.


Alex also discusses the importance of engaging early with #patients and patient organisations for #PMM across the world to truly understand the patient journey, their challenges & daily routines which help to shape the study design. This ensures successful enrolment and patient retention throughout the clinical trial, 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 #rarediseases as it is often difficult to find the patients and something Alex is extremely proud of.

Reneo Pharma: 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/


JustGiving: https://www.justgiving.com/fundraising/partners4access-b-v?utm_source=copyLink&utm_med[…]ampaign=pfp-share&utm_term=0c6d8180e0f3440b8383845adedb6513

 


Jun 01, 202316:14
Interview with Xortx Therapeutics CEO, Dr Allen Davidoff discussing their journey from early development to potential launch

Interview with Xortx Therapeutics CEO, Dr Allen Davidoff discussing their journey from early development to potential launch

P4A are joined by Xortx Therapeutics' CEO, Dr Allen Davidoff, as he discusses the journey from early development to potential launch for their novel therapy XRX-008 to treat patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD).


Allen discusses the company vision for developing novel therapies for rare progressive kidney disorders, and tells us about the launch journey for their main therapeutic candidate for AKPKD. How they managed to complete enrolment for late phase study in record time by engaging with patients and advocacy groups early, to really understand the patient journey and how the disease affects their quality of life. This helped to shape the clinical trial design by truly putting the patients at the forefront of all decisions and to ensure they were involved at all stages of the process.


Bio: Dr. Allen W. Davidoff, Ph.D. (15 years drug development experience ), Currently President and CEO of XORTX Therapeutics Inc (2013- ), Formerly, Chief Scientific Officer, VP Product Development and co-founder of Stem Cell Therapeutics Corp (Trillium Therapeutics- TRIL: NASDAQ) (7 yrs). Prior to Stem Cell Therapeutics Corp., Senior Scientist and Head of Pharmacology at Cardiome Pharma Corp. Dr. Davidoff has 12 + years of therapeutic drug development experience with a focus on clinical and regulatory developmental affairs, in the US, Canada, Germany, Denmark, and India. Senior management and leadership experience includes pharmaceutical non-clinical and clinical R&D including 2 investigational new drug (“IND”) applications or supplemental IND’s, 2 phase I studies (4 multi-country), 7 phase II studies, and 1 NDA.

Dr Allen Davidoff: https://www.linkedin.com/in/allen-davidoff-9a80968/


XORTX Therapeutics

XORTX Therapeutics, Inc. is a drug based biotechnology company primarily focused on orphan disease indications which have aberrant purine metabolism and frequently high serum uric acid imbalance. Our focus on developing kidney therapies also includes therapeutics programs for large markets such as diabetic nephropathy and acute kidney injury associated with respiratory virus infections such as SARS-CoV-2.

We possess patents and patent applications that may include U.S. and international rights regarding the development of uric acid-lowering agents for the treatment of hypertension, insulin resistance, diabetes, metabolic syndrome, and kidney injury.

XORTX Therapeutics: https://www.xortx.com/

Apr 29, 202319:22
World EPA Congress 2023 Special
Apr 17, 202324:10
Rare Disease Day Special 2023

Rare Disease Day Special 2023

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.

Feb 28, 202340:03
Trends to look out for in 2023

Trends to look out for in 2023

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

Feb 03, 202344:18
World Orphan Drug Congress Europe 2022 Special

World Orphan Drug Congress Europe 2022 Special

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

Dec 20, 202242:40
Winds of change for German Healthcare Market!

Winds of change for German Healthcare Market!

 Winds of change are howling in Germany, with the draft healthcare bill now approved to stabilise SHI fund finances. What will manufacturers, with innovative orphan drugs and cell and gene therapies, launch strategies be?

With the latest decision from Janssen to avoid the German market altogether for x2 Rare Oncology innovative drugs, will this be a trend we are likely to see continue? 

Join Stefan Walzer & Fisentzos Stylianou discuss the new bill, in regards to the biggest changes that impact orphan drug (OD) manufacturers. Will OD manufacturers still see Germany as the first go to 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. 


Oct 31, 202238:42
Part 2 -Patient Empowerment: Is it a no-brainer?

Part 2 -Patient Empowerment: Is it a no-brainer?

P4A’s 2 part -Patient Empowerment podcast series has been released. Our incredible panel of experts Laurence Woollard, Neil Bertelsen & Sophie Schmitz are back with us to carry on the discussion in part 2 on true patient empowerment and really looking at the WHY’s! Why should drug manufacturers involve & listen to and involve the patients. What are the implications, if any, for drug manufacturers who do not include the patient voice. How can we bring all stakeholders together to work completely new approaches to medicine approval and patient access. Lastly, we will look at initiatives or services our panel are 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)


Oct 03, 202228:25
Part 1 Patient Empowerment; Why involving patients from early drug development through to launch is a no-brainer!

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. 

Sep 13, 202231:08
What makes P4A an award winning consultancy?

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!) - 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/

Jul 29, 202215:03
Special Episode with EUCOPE-Part 2 with Alexander Natz

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?

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/

Jun 30, 202228:57
Special series with Eucope: Part 1 featuring Victor Maertens

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:

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.

May 31, 202224:52
China market access: obstacle or opportunity for orphan drug, cell and gene therapy manufacturers?
Apr 29, 202210:28
Should the Middle East be a priority launch market for orphan drug manufacturers?

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

Mar 31, 202212:34
Rare Disease Day Special featuring Janet Bloor and Nick Meade - February 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

Feb 28, 202228:47
P4A Insights: Trends to look out for in 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

Feb 04, 202210:32
Special episode - Bluebird Bio: What went wrong? -December 2021:

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

Dec 13, 202117:16
Special episode featuring James Mackay - November 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.

Nov 30, 202123:60
Monthly Roundup - October 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

Oct 29, 202110:59
Monthly RoundUp - September 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

Sep 30, 202115:42
Monthly RoundUp: July 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

Jul 30, 202115:02
Monthly RoundUp - June 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

Jun 30, 202107:44
Monthly RoundUp - May 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, 202109:32
Monthly RoundUp - April 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

  

Apr 30, 202120:58
Monthly RoundUp - March 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

Mar 31, 202113:22
Monthly RoundUp - February 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

Feb 27, 202122:50
Rare Disease Day special episode: Reclaiming the rare disease patient’s voice

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 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/


Jan 29, 202136:56
End of year 2020 review

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


Dec 21, 202018:04
Special episode: Gene Therapy access from a specialty pharmacy perspective

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.

Jun 22, 202020:50
Weekly RoundUp: February 29, 2020
Feb 29, 202017:57
Weekly Roundup: December 30, 2019

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

Dec 30, 201909:57
Weekly Roundup: December 23, 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

Dec 23, 201904:55
Weekly Roundup: November 12, 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

Nov 12, 201914:27
Weekly Roundup: November 5, 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

Nov 05, 201908:16
Weekly RoundUp: August 5, 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   

Aug 05, 201918:36
Weekly Roundup: July 29, 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


Jul 29, 201905:55
Weekly Roundup: July 22, 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

Jul 24, 201911:10
Weekly Roundup: July 1, 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

Jul 01, 201912:13
Weekly Roundup: June 23, 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

Jun 23, 201906:36
Weekly Roundup: June 16, 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

Jun 16, 201906:30
Weekly Roundup: June 12, 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

Jun 12, 201910:11
Weekly RoundUp: June 3, 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

Jun 03, 201907:14
Weekly Roundup: May 25, 2019
May 25, 201910:22
Weekly Roundup: May 18, 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, 201906:50
Weekly Roundup: May 12, 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, 201906:31