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Rare Disease, Cell & Gene Therapy Monthly Roundup

A monthly podcast highlighting the most important news developments and its impact on the orphan drug, cell and gene therapy world including research advancements, corporate events and regulatory changes.

2021 Podcasts Archive

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

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

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

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

2020 Podcasts Archive

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

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.

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

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

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

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

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

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

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.

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

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

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

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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