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Let’s Talk Rare

Let's Talk Rare

A monthly podcast highlighting:

  • Important medical & pharma news developments
  • Impacts on the orphan drug, cell and gene therapy world
  • Research advancements, corporate events and regulatory changes

2022 Podcasts Archive

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/

 

Presenter
Georgie Rack
Contributor
Owen Bryant

Special series with EUCOPE-Part 2 featuring 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: [email protected]

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/

Presenter
Chloe Sheppard
Contributor
Alexander Natz

Special series with EUCOPE-Part one 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 lastly the future of ATMPs for manufacturers and patients.

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.

 

 

Presenter
Aparna Krishnan
Contributor
Victor Maertens

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: https://www2.deloitte.com/cn/en/pages/life-sciences-and-healthcare/solutions/life-sciences.html

Presenter: Aparna Krishnan, Partner – Global Operations

 

Contributor: Fisentzos Stylianou, Analyst

 

Producer: Operations team

 

Presenter
Aparna Krishnan
Contributor
Fisentzos Stylianou

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

Lavni Varyani is the Founder and Principal Consultant at Pharma Business Partners, a market access consultancy based in Dubai supporting the life science industry to generate market insights across the Middle East and Africa. More information on Pharma Business Partners: https://pharmabp.com/

Presenter
Akshay Kumar
Contributor
Lavni Varyani

2021 Podcasts Archive

December 2021 special episode – Bluebird Bio: What went wrong?

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

Special episode featuring James Mackay – November 2021

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

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! 

Producer: Operations Team 

Presenter
Richard Wang
Contributor
Adama Anozie

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!

Producer: Operations Team

Presenter
Georgie Rack
Contributor
Bruce Chin

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.

Producer: Operations Team

Presenter
Akshay Kumar
Contributor
Richard Wang

2020 Podcasts Archive

End of Year episode: A review of key events in 2020

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

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.

Weekly Roundup: February 29, 2020

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

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

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

Weekly Roundup: November, 12 2019

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

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

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

2018 Podcasts Archive

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.

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

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

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

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

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