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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

Georgie Rack

Presenter

Owen Bryant

Presenter

2023 Podcasts Archive

2022 Podcasts Archive

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

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

More information on:

Genetic Alliance UK: https://geneticalliance.org.uk/

Duchenne Nexus Advocacy Facebook page: https://www.facebook.com/pages/category/Nonprofit-organization/Duchenne-Nexus-Advocacy-DNA-103126328289199/

Presenter
Georgie Rack
Contributor
Janet Bloor and Nick Meade

2021 Podcasts Archive

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

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

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.

Other contributors: Jayne Watson

Producer: Aparna Krishnan

Presenter
Joanna Fernandes

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’.

Other contributors: Richard Wang, Andrea Bernardini and Erfan Akbraian

Producer: Aparna Krishnan

Presenter
Akshay Kumar

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

2020 Podcasts Archive

2019 Podcasts Archive

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

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

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

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

Contributors: Joanna Fernandes

Producer: Aparna Krishnan

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

2018 Podcasts Archive

Weekly Roundup: November 9, 2018

In the first of a two-part special, the P4A team are at the World Orphan Drug Congress in Barcelona discussing access to new treatments with a Porphyria patient; hear from a company offering a unique service to clinical trial patients; a special interview with Ségolène Aymé on rare disease challenges and a chat with organisers of the congress.

Presenter: Aparna Krishnan
Contributors: Sophie Schmitz, Managing Partner, P4A; Dr Jasmin Burman-Aksözen, Vice President of International Porphyria Patient Network; Helen Springford, Vice President, Illingworth Research Group; Prof Ségolène Aymé, Founder of Orphanet; Andrew Mears, Business Development Manager at Terrapinn.

Weekly Roundup: November 2, 2018

The team analyze the new Medicare drug pricing plan unveiled by the Trump administration and the prospects of Vertex’s cystic fibrosis drug Symkevi which gained EU approval recently.
Presenter: Aparna Krishnan
Contributors: Max Rex and Jack Rawson

Weekly Roundup: October 25, 2018

This week, we look at Biogen’s spinal muscular atrophy drug Spinraza’s prospects in the face of emerging competition and the latest Brexit update providing recommendations on areas to prepare for as deadline for UK’s exit looms.
Presenter: Aparna Krishnan
Contributor: Joanna Fernandes

Weekly Roundup: October 18, 2018

This week, the P4A team analyse the new regulations surrounding health technology assessments in Europe and US-based CAR-T start-up Allogene Therapeutic’s record-breaking initial public offering.
Presenter: Max Rex
Contributor: Christina Poschen, Consultant, P4A

Weekly Roundup: October 11, 2018

The P4A team analyze Novartis’ deal with Celluar Biomedicine Group to supply the CAR-T therapy Kymriah and the successes and failures of the reimbursement of Vertex’s cystic fibrosis drug, Orkambi.
Presenter: Max Rex
Contributor: Nader Murad, Senior Analyst

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