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...
Feb 28, 2022•29 min•Season 4Ep. 2
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 latest podcast on the top trends in drug development, HTA and pricing from Akshay Kumar and Andrea Bernardini to help you navigate the year ahead. Presenter: Georgie Rack Speakers: Akshay Kumar & Andrea Bernardini Produced by: Ops team
Feb 04, 2022•11 min•Season 4Ep. 1
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
Dec 13, 2021•17 min•Season 3Ep. 12
This episode featuring James Mackay, President & CEO of Aristea Therapeutics. James 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 c...
Nov 30, 2021•24 min
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 H...
Oct 29, 2021•11 min
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! Presenter: Georgie Rack Contributor: Bruce Chin Producer: Operations Team
Sep 30, 2021•16 min
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. Presenter: Akshay Kumar Contributors: Richard Wang Producer: Operations Team
Jul 30, 2021•15 min
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
Jun 30, 2021•8 min
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...
May 31, 2021•10 min
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 Healthca...
Apr 30, 2021•21 min
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 ...
Mar 31, 2021•13 min
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
Feb 27, 2021•23 min
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 this pandemic include disruption in their access to treatments, struggle to continue participation in clinical trials, lack of access to vital equipment such as PPE (Personal Protective Equipment) and also to healthcare personnel. We speak to Genetic Alliance chief executive officer Jayne Spink, patient Nicola Whitehill and Danielle Myers, mother ...
Jan 29, 2021•37 min
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! Presenter and Contributors: Sophie Schmitz and Akshay Kumar Producer: Aparna Krishnan...
Dec 21, 2020•18 min
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 Hea...
Jun 22, 2020•21 min
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 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...
Feb 29, 2020•18 min
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
Dec 30, 2019•10 min
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. Presente...
Dec 23, 2019•5 min
The team discuss the NORD summit particularly FDA commissioner Scott Gottleib’s presentation, 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 ...
Nov 12, 2019•14 min
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
Nov 05, 2019•8 min
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
Aug 05, 2019•19 min
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
Jul 29, 2019•6 min
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
Jul 24, 2019•11 min
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
Jul 01, 2019•12 min
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 Contributor: Joanna Fernandes Producer: Aparna Krishnan
Jun 23, 2019•7 min
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
Jun 16, 2019•6 min
This week, the team discuss Vertex's acquisition of Exonics and expansion of its collaboration with CRISPR Therapeutics. Also, we take an indepth look at the European Medicines Agency's approval of Bluebirdbio's gene therapy Zynteglo as a treatment for beta-thalassemia . Presenter: Aparna Krishnan Contributor: Jack Rawson Producer: Aparna Krishnan
Jun 12, 2019•10 min
On our anniversary episode, the P4A team discuss the USFDA approval of Novartis' Zolgensma, a one time gene therapy for paediatric spinal muscular atrophy patients and a look back at the last 12 months of our podcasting journey. Presenter: Joanna Fernandes Contributor: Christina Poschen, Aparna Krishnan Producer: Aparna Krishnan
Jun 03, 2019•7 min
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 identify how much value a cure represents. Also, Italy's attempts at introducing a draft resolution to improve transparency in drug pricing at the World Health Assembly. Presenter: Aparna Krishnan Contributors: Joanna Fernandes, Christina Poschen Producer: Aparna Krishnan...
May 25, 2019•10 min
This week, the team discuss the deal between UK's National Health Service and Biogen prompting the successful reimbursement of Biogen’s Spinraza as a treatment for spinal muscular atrophy and Solid Biosciences’ clinical update on its Duchenne Muscular Dystrophy gene therapy product SGT-001. Presenter: Aparna Krishnan Contributor: Max Rex Producer: Aparna Krishnan
May 18, 2019•7 min
