Let’s Talk Rare

Let's Talk Rare

A monthly podcast highlighting:

2022 Podcasts Archive

2021 Podcasts Archive

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

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

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

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

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

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

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

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

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

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

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.

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

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

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

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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    Podcasts Archive