P4A Let's Talk Rare: The Life Science Podcast

Solving Patient Matching in Rare Disease Trials

29 Apr 2026

Contributed by Lukas

One of the most persistent barriers in rare disease drug development isn’t the science — it’s identifying and reaching the small number of patie...

Rare Disease Day Special: The stats and stories you need to hear.

27 Feb 2026

Contributed by Lukas

Rare Disease Day Special | Let’s Talk RareThis Rare Disease Day, Let’s Talk Rare releases a special episode focused on the data and real-world c...

2026 Trends: A Year Of Change & Opportunity

30 Jan 2026

Contributed by Lukas

26 isn’t just another year in market access — it’s a turning point.In this episode of Let’s Talk Rare, P4A dives into the trends that are abo...

Going To Glasgow: ISPOR 2025

02 Dec 2025

Contributed by Lukas

In this episode of Let’s Talk Rare, we looked back at P4A’s recent trip to Glasgow, where our colleagues, Sam Morrison & Iro Malekou were on ...

Conversations Beyond the Clinic

03 Nov 2025

Contributed by Lukas

Recorded live from ESMO 2025, Conversations Beyond the Clinic explores the different perspectives of the attendees and stakeholders at the ESMO conf...

Voices Uncovered: Understanding Mental Health in Rare Diseases using Social Listening

29 Sep 2025

Contributed by Lukas

In this episode, we tune into the unfiltered voices of patients and caregivers navigating life with rare conditions.Through the power of social listen...

Stronger Together: Collaborating for Better Patient Information with Catherine Richards Golini

26 Aug 2025

Contributed by Lukas

How do we make sure patients receive health information that is clear, trustworthy, and truly helpful? We sit down with Catherine Richards Golini fro...

OST-HER2 and the Fight Against Osteosarcoma: A New Hope After 40 Years

01 Jul 2025

Contributed by Lukas

Join us as we sit down with Paul Romness of OS Therapies to explore an exciting breakthrough in OST-HER2 targeted therapies for osteosarcoma—...

AI for Rare Disease: From Discovery to Trials, Smarter and Faster

29 May 2025

Contributed by Lukas

Discover how artificial intelligence is transforming the future of rare disease research in the latest episode of Let’s Talk Rare, the official pod...

Improving Alignment In Drug Development

28 Apr 2025

Contributed by Lukas

In the latest episode of Let's Talk Rare, Owen Bryant & Georgie Rack delve into the world of drug development in rare diseases.They are joined...

Breaking Barriers

31 Mar 2025

Contributed by Lukas

Brain cancers are notoriously difficult to manage with current treatments offering limited effectiveness. But what if there was a way to change that? ...

P4A's 5 Top Trends for 2025

31 Jan 2025

Contributed by Lukas

Dive into P4A's annual appraisal of the year ahead. In this episode of Let's Talk Rare we examine the most important trends as identified and ...

The JCA is live

13 Jan 2025

Contributed by Lukas

After3 years in the making, the Joint Clinical Assessment for the EU is in effect. From 12th January 2025 new cancer medicines and advanced therapy ...

Innovative Treatments for Cutaneous T-Cell Lymphoma with Leonard Mazur of Citius Pharmaceuticals

19 Nov 2024

Contributed by Lukas

Welcome to this month's P4A Let’s Talk Rare podcast episode by Partners4Acess. Today, Georgie and Owen are joined by Leonard Mazur, CEO of Citius Ph...

Amplifying Patient Voices: Wes Michael on Rare Disease Advocacy

10 Oct 2024

Contributed by Lukas

In this episode of P4A Let's Talk Rare, hosts Georgie and Owen Bryant from Partners For Access are joined by Wes Michael, founder and president of Rar...

Impact of Mergers, Acquisitions, and Patent Expirations on Job Cuts in Pharma and Biotech: Insights from Aurelija Luko and Darren Callanan

02 Jul 2024

Contributed by Lukas

Welcome to this month's episode of the P4A Let’s Talk Rare podcast by Partners4Acess. Today, Georgie and Owen are joined by their colleagues Aur...

The Role of Patient Experience Data in Drug Development with Pina Haberl and Sam Morrison

03 May 2024

Contributed by Lukas

Welcome to this month's episode of the P4A Let’s Talk Rare podcast by Partners4Acess. Today, Georgie and Owen are joined by their colleagues Pi...

World EPA Conference 2024 Special

10 Apr 2024

Contributed by Lukas

Welcome to this month's episode of Let’s Talk Rare: The Life Science Podcast brought to you by Partners4Access. Host Georgie records this episod...

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

29 Feb 2024

Contributed by Lukas

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, H...

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

02 Jan 2024

Contributed by Lukas

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...

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

01 Dec 2023

Contributed by Lukas

In this podcast, Georgie, RJ, and Owen discuss revolutionizing patient outcomes and the power of digital health solutions. RJ, a healthcare industry e...

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

06 Oct 2023

Contributed by Lukas

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 Publicati...

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

31 Jul 2023

Contributed by Lukas

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 ...

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

01 Jun 2023

Contributed by Lukas

In this episode we will be talking to Alejandro (Alex) Dorenbaum, M.D., CMO of Reneo Pharmaceuticals. Alex discusses how the company is developing ...

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

29 Apr 2023

Contributed by Lukas

P4A are joined by Xortx Therapeutics' CEO, Dr Allen Davidoff, as he discusses the journey from early development to potential launch for their nov...

World EPA Congress 2023 Special

17 Apr 2023

Contributed by Lukas

P4A host a special episode live from World Evidence, Pricing & Access Conference 2023 in Amsterdam. Join Georgie & Owen hosting live from Wor...

Rare Disease Day Special 2023

28 Feb 2023

Contributed by Lukas

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...

Trends to look out for in 2023

03 Feb 2023

Contributed by Lukas

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 topi...

World Orphan Drug Congress Europe 2022 Special

20 Dec 2022

Contributed by Lukas

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 mon...

Winds of change for German Healthcare Market!

31 Oct 2022

Contributed by Lukas

 Winds of change are howling in Germany, with the draft healthcare bill now approved to stabilise SHI fund finances. What will manufacturers, wit...

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

03 Oct 2022

Contributed by Lukas

P4A’s 2 part -Patient Empowerment podcast series has been released. Our incredible panel of experts Laurence Woollard, Neil Bertelsen & Sophie S...

Part 1 Patient Empowerment; Why involving patients from early drug development through to launch is a no-brainer!

13 Sep 2022

Contributed by Lukas

This month we have a special 2-part series focused on Patient Empowerment, with special guests Laurence Woollard, On the Pulse, Neil Bertelsen, Indepe...

What makes P4A an award winning consultancy?

29 Jul 2022

Contributed by Lukas

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...

Special Episode with EUCOPE-Part 2 with Alexander Natz

30 Jun 2022

Contributed by Lukas

In this episode Chloe & Alexander will be starting the discussion with an overview of EU HTA regulation, looking at the harmonization debate and h...

Special series with Eucope: Part 1 featuring Victor Maertens

31 May 2022

Contributed by Lukas

Part 1 featuring Victor Maertens, Government Affairs Manager at EUCOPE In this episode Victor and Aparna will be focusing on Advanced Therapy Medicina...

China market access: obstacle or opportunity for orphan drug, cell and gene therapy manufacturers?

29 Apr 2022

Contributed by Lukas

In 2020 China’s pharmaceutical market passed an important milestone, a Deloitte report cited that China was expected to grow its pharmaceutical mark...

Should the Middle East be a priority launch market for orphan drug manufacturers?

31 Mar 2022

Contributed by Lukas

Over the last few years, the Middle East has piqued the interest of orphan drug manufacturers, and some have prioritised launching in this region befo...

Rare Disease Day Special featuring Janet Bloor and Nick Meade - February 2022

28 Feb 2022

Contributed by Lukas

For this Rare Disease Day episode, Janet and Nick discuss a range of topics associated with the rare disease community and patient advocacy. They addr...

P4A Insights: Trends to look out for in 2022

04 Feb 2022

Contributed by Lukas

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 lates...

Special episode - Bluebird Bio: What went wrong? -December 2021:

13 Dec 2021

Contributed by Lukas

Sophie Schmitz and Joanna Fernandes discuss bluebird bio’s innovative gene therapy Zynteglo and its withdrawal from the European market. Sophie and ...

Special episode featuring James Mackay - November 2021

30 Nov 2021

Contributed by Lukas

This episode featuring James Mackay, President & CEO of Aristea Therapeutics. James provides insights on his experience with setting up a biotech ...

Monthly Roundup - October 2021

29 Oct 2021

Contributed by Lukas

In this episode P4A’s Richard Wang and Adama Anozie discuss Hospital Exemption (HE) which is an emerging access pathway for Advanced Therapy Medicin...

Monthly RoundUp - September 2021

30 Sep 2021

Contributed by Lukas

In this episode P4A’s Senior Client Relationship Director, Bruce Chin discusses his experiences from the in person World Orphan Drug Congress USA 20...

Monthly RoundUp: July 2021

30 Jul 2021

Contributed by Lukas

In this episode Akshay Kumar and Richard Wang discuss how through innovative technology, decentralised clinical trials came to the limelight, facilita...

Monthly RoundUp - June 2021

30 Jun 2021

Contributed by Lukas

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...

Monthly RoundUp - May 2021

31 May 2021

Contributed by Lukas

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 Admi...

Monthly RoundUp - April 2021

30 Apr 2021

Contributed by Lukas

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 Eu...

Monthly RoundUp - March 2021

31 Mar 2021

Contributed by Lukas

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 va...

Monthly RoundUp - February 2021

27 Feb 2021

Contributed by Lukas

In this episode, the P4A team discussed the impact of cost-containment measures in Germany, the European Commission's recent move to revise legislatio...

Rare Disease Day special episode: Reclaiming the rare disease patient’s voice

29 Jan 2021

Contributed by Lukas

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...

End of year 2020 review

21 Dec 2020

Contributed by Lukas

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...

Special episode: Gene Therapy access from a specialty pharmacy perspective

22 Jun 2020

Contributed by Lukas

This episode discusses patient access to AveXis' gene therapy Zolgensma from Orsini Healthcare, US based specialty pharmacy's perspective.  The s...

Weekly RoundUp: February 29, 2020

29 Feb 2020

Contributed by Lukas

This week is a special episode celebrating Rare Disease Day 2020.  Our guest speaker is Annie Kennedy, head of policy and advocacy at Every Life ...

Weekly Roundup: December 30, 2019

30 Dec 2019

Contributed by Lukas

As 2019 winds down, P4A's Sophie Schmitz and Akshay Kumar discuss the key trends that defined this year  -  marketing approval of cell and g...

Weekly Roundup: December 23, 2019

23 Dec 2019

Contributed by Lukas

The team discuss the latest update on the proposed EU health technology assessment (HTA) regulation. The key objectives of the 2018 proposal was to pr...

Weekly Roundup: November 12, 2019

12 Nov 2019

Contributed by Lukas

The team discuss the NORD summit particularly FDA commissioner Scott Gottleib’s presentation, panel discussion on cell and gene therapy pricing as w...

Weekly Roundup: November 5, 2019

05 Nov 2019

Contributed by Lukas

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 w...

Weekly RoundUp: August 5, 2019

05 Aug 2019

Contributed by Lukas

In this episode, P4A speaks to a special guest - Alliance of Regenerative Medicine's chief executive officer Janet Lambert on the ARM's key initiative...

Weekly Roundup: July 29, 2019

29 Jul 2019

Contributed by Lukas

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 impl...

Weekly Roundup: July 22, 2019

24 Jul 2019

Contributed by Lukas

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. Als...

Weekly Roundup: July 1, 2019

01 Jul 2019

Contributed by Lukas

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 ...

Weekly Roundup: June 23, 2019

23 Jun 2019

Contributed by Lukas

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...

Weekly Roundup: June 16, 2019

16 Jun 2019

Contributed by Lukas

This week, the team discuss the emergence of 'drug buyer's clubs' in the UK involving Vertex's cystic fibrosis drug Orkambi.  Presenter: Aparna K...

Weekly Roundup: June 12, 2019

12 Jun 2019

Contributed by Lukas

This week, the team discuss Vertex's acquisition of Exonics and expansion of its collaboration with CRISPR Therapeutics. Also, we take an indepth look...

Weekly RoundUp: June 3, 2019

03 Jun 2019

Contributed by Lukas

On our anniversary episode, the P4A team discuss the USFDA approval of Novartis' Zolgensma, a one time gene therapy for paediatric spinal muscular atr...

Weekly Roundup: May 25, 2019

25 May 2019

Contributed by Lukas

In this episode, we look at Novartis CEO Vas Narasimhan calling for a change in the US drug payment systems, arguing for new economic models to identi...

Weekly Roundup: May 18, 2019

18 May 2019

Contributed by Lukas

This week, the team discuss the deal between UK's National Health Service and Biogen prompting the successful reimbursement of Biogen’s Spinraza as ...

Weekly Roundup: May 12, 2019

12 May 2019

Contributed by Lukas

This week, the P4A team discuss a new experimental therapy - Phage therapy or genetically engineered phages in order to treat bacterial infections and...

Weekly Roundup: May 3, 2019

03 May 2019

Contributed by Lukas

This week, we feature a special guest podcast speaker - Scott  Dorfman, chief executive officer of non-profit gene therapy developer Odylia Thera...

Weekly Roundup: April 27, 2019

27 Apr 2019

Contributed by Lukas

This week, we look at the European Federation of Pharmaceutical Industries and Associations or EFPIA’s patient wait survey and its key findings as w...

Weekly Roundup: April 22, 2019

22 Apr 2019

Contributed by Lukas

 As part of the continuing World Orphan Drug Congress (WODC) special, this episode discusses the cGMP facilities and biosafety solutions from Ger...

Weekly Roundup: April 12, 2019

17 Apr 2019

Contributed by Lukas

This week, the P4A team are podcasting from the World Orphan Drug Congress in Washington D.C. We speak to André Choulika, Chairman and CEO of Cellect...

Weekly Roundup: March 28, 2019

28 Mar 2019

Contributed by Lukas

The team discuss the creation of a new health economics advisory committee in Spain expected to influence pricing and reimbursement decisions for drug...

Weekly Roundup: March 22, 2019

22 Mar 2019

Contributed by Lukas

This week, we are looking at The Institute for Clinical and Economic Review or ICER’s white paper on alternative options for the US rebate system, a...

Weekly Roundup: March 15, 2019

15 Mar 2019

Contributed by Lukas

This week, we look at Ireland's access to orphan drugs as compared to rest of western Europe and the first outcomes deal for CAR-T cell therapies in G...

Weekly Roundup: March 8, 2019

08 Mar 2019

Contributed by Lukas

This week, the P4A team discuss the spate of mergers and acquisitions involving Big Pharma companies in the gene therapy space. Particularly, we looke...

Weekly Roundup: February 28, 2019

28 Feb 2019

Contributed by Lukas

On Rare Disease Day, P4A discusses the role of policy-makers in the healthcare system and specifically, the issues surrounding cross-border healthcare...

Weekly Roundup: February 22, 2019

22 Feb 2019

Contributed by Lukas

In another special edition episode dedicated to the '6P' campaign to mark Rare Disease Day, the Weekly RoundUp team discusses the pharmaceutical persp...

Weekly Roundup: February 15, 2019

15 Feb 2019

Contributed by Lukas

 Throughout February, Partners4Access is running a campaign to mark the annual Rare Disease Day on February 28. The '6P' campaign is aimed at cre...

Weekly Roundup: February 1, 2019

01 Feb 2019

Contributed by Lukas

Throughout February 2019, the P4A team are hosting a series of special edition podcast episodes to mark Rare Disease Day. The initiative is part of a ...

Weekly Roundup: January 25, 2019

25 Jan 2019

Contributed by Lukas

 This week we are looking at the USFDA’s proposed plans to address the expected rise in cell and gene therapy product applications and a signif...

Weekly Roundup: January 18, 2019

18 Jan 2019

Contributed by Lukas

This week, the P4A team discusses the Louisiana Medicaid program implementing the 'Netflix' subscription model to pay for hepatitis C drugs and the po...

Weekly Roundup: January 11, 2019

14 Jan 2019

Contributed by Lukas

Happy New Year to Weekly Roundup listeners! 2019 is shaping up to be an exciting year for the healthcare and biotechnology industry. We are only a cou...

Weekly Roundup: December 14, 2018

14 Dec 2018

Contributed by Lukas

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 dise...

Weekly Roundup: December 7, 2018

07 Dec 2018

Contributed by Lukas

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 ...

Weekly Roundup: November 30, 2018

30 Nov 2018

Contributed by Lukas

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-p...

Weekly Roundup: November 23, 2018

23 Nov 2018

Contributed by Lukas

The P4A team discuss the latest update on Brexit and its implication on life science companies; Novartis' CAR-T therapy commercialization efforts and ...

Weekly Roundup: November 15, 2018

15 Nov 2018

Contributed by Lukas

In the second part of our World Orphan Drug Congress special, P4A's Sophie Schmitz speaks to Diane Kleinermans, adviser to the Belgian Federal Governm...

Weekly Roundup: November 9, 2018

09 Nov 2018

Contributed by Lukas

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 Porphyr...

Weekly Roundup: November 2, 2018

02 Nov 2018

Contributed by Lukas

The team analyze the new Medicare drug pricing plan unveiled by the Trump administration and the prospects of Vertex's cystic fibrosis drug Symkevi wh...

Weekly Roundup: October 25, 2018

25 Oct 2018

Contributed by Lukas

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 prov...

Weekly Roundup: October 18, 2018

18 Oct 2018

Contributed by Lukas

This week, the P4A team analyse the new regulations surrounding health technology assessments in Europe and US-based CAR-T start-up Allogene Therapeut...

Weekly Roundup: October 11, 2018

11 Oct 2018

Contributed by Lukas

The P4A team analyze Novartis' deal with Celluar Biomedicine Group to supply the CAR-T therapy Kymriah and the successes and failures of the reimburse...

Weekly Roundup: Oct 5, 2018

05 Oct 2018

Contributed by Lukas

This week, Pfizer's Matthew Harold joins us to discuss the findings of his recent research study that reviewed national policies for rare diseases in ...

Weekly Roundup: Sep 27, 2018

27 Sep 2018

Contributed by Lukas

This week, P4A's podcast will cover Luxturna's CHMP recommendation, Novartis' restructuring plans and Alexion's acquisition of Syntimmune. Presenter: ...

Weekly Roundup: September 20, 2018

20 Sep 2018

Contributed by Lukas

Back from a summer break! The P4A team discuss the reasons behind NICE's rejection of Novartis' CAR-T therapy Kymriah, Denmark and Norway's joint drug...

Weekly Roundup: August 16, 2018

16 Aug 2018

Contributed by Lukas

In this episode, the team discuss UK health technology body NICE not recommending Biogen's Spinraza for routine use in the NHS; CVS announcing the use...

Weekly Roundup: August 9, 2018

09 Aug 2018

Contributed by Lukas

In this episode, P4A discusses the UK government’s publication of four key documents guiding the life science industry on how to operate during the ...

Weekly Roundup: August 2, 2018

02 Aug 2018

Contributed by Lukas

This week, the team discuss the consolidation among small and medium sized cell and gene therapy companies and the latest on Brexit impact with news t...

Weekly Roundup: July 26, 2018

26 Jul 2018

Contributed by Lukas

The EU's Commissioner for Competition Margrethe Vestager, recently said that price differences in the pharma market among different member states coul...