Rare Disease, Cell & Gene Therapy Weekly RoundUp

Rare Disease, Cell & Gene Therapy Weekly RoundUp

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A weekly podcast highlighting the most important news developments and its impact on the orphan drug, cell and gene therapy world. Visit www.partners4access.com/podcasts/


July 1, 2019
Jul 1 • 12 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…
June 23, 2019
Jun 23 • 6 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
June 16, 2019
Jun 16 • 6 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
June 12, 2019
Jun 12 • 10 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…
June 3, 2019
Jun 3 • 7 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…
May 25, 2019
May 25 • 10 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…
May 18, 2019
May 18 • 6 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…
May 12, 2019
May 12 • 6 min
This week, the P4A team discuss a new experimental therapy - Phage therapy or genetically engineered phages in order to treat bacterial infections and the acquisition of Theracon by Big Pharma firm Pfizer for upto $810 million. Presenter: Aparna Krishnan…
May 3, 2019
May 3 • 18 min
This week, we feature a special guest podcast speaker - Scott Dorfman, chief executive officer of non-profit gene therapy developer Odylia Therapeutics on his personal journey and the future in gene therapy development. If you would like to know more…
April 27, 2019
Apr 27 • 19 min
This week, we look at the European Federation of Pharmaceutical Industries and Associations or EFPIA’s patient wait survey and its key findings as well as news developments in the spinal muscular atrophy world relating to gene therapy Novartis’ Zolgensma…
April 22, 2019
Apr 22 • 14 min
As part of the continuing World Orphan Drug Congress (WODC) special, this episode discusses the cGMP facilities and biosafety solutions from Germfree’s Carol Houts, P4A’s Sophie Schmitz looks back at the 2019 conference and Terrapinn’s Andre Singer talks…
April 12, 2019
Apr 17 • 17 min
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 Cellectis on the challenges of commercializing a CAR-T cell product and ethical concerns surrounding it, and Anna…
March 28, 2019
Mar 28 • 6 min
The team discuss the creation of a new health economics advisory committee in Spain expected to influence pricing and reimbursement decisions for drugs; and Japan’s approval of its first gene therapy and CAR-T therapy - AnGes’ HGF Plasmid and Novartis’…
March 22, 2019
Mar 22 • 8 min
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, and the establishment of a national agency to evaluate drug effectiveness and negotiate prices in Canada.…
March 15, 2019
Mar 15 • 9 min
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 Germany. Presenter: Joanna Fernandes Contributors: Nader Murad, Ciaran Cassidy Producer: Aparna Krishnan
March 8, 2019
Mar 8 • 8 min
This week, the P4A team discuss the spate of mergers and acquisitions involving Big Pharma companies in the gene therapy space. Particularly, we looked at the deals behind Roche’s acquisition of Spark Therapeutics and Biogen’s buyout of Nightstar. Also,…
February 28, 2019
Feb 28 • 15 min
On Rare Disease Day, P4A discusses the role of policy-makers in the healthcare system and specifically, the issues surrounding cross-border healthcare as part of its ‘6P’ campaign. This directive is a key cornerstone legislation by EU officials that…
February 22, 2019
Feb 22 • 9 min
In another special edition episode dedicated to the ‘6P’ campaign to mark Rare Disease Day, the Weekly RoundUp team discusses the pharmaceutical perspective on partnerships in the rare disease space. Presenter: Max Rex Contributor: Jan-Willem Schmitz,…
February 15, 2019
Feb 15 • 14 min
Throughout February, Partners4Access is running a campaign to mark the annual Rare Disease Day on February 28. The ‘6P’ campaign is aimed at creating awareness on the impact of rare diseases on key stakeholders and the need for partnership to achieve…
February 1, 2019
Feb 1 • 9 min
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 new 6P campaign aimed at promoting awareness about rare disease challenges and its impact on people and…
January 25, 2019
Jan 25 • 11 min
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 significant development in a new controversial procedure called gene drive. Also, P4A is starting a new campaign that…
January 18, 2019
Jan 18 • 9 min
This week, the P4A team discusses the Louisiana Medicaid program implementing the ‘Netflix’ subscription model to pay for hepatitis C drugs and the potential Brexit options facing the UK government. Presenter: Joanna Fernandes Contributor: Max Rex…
January 11, 2019
Jan 14 • 6 min
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 couple of weeks in and already news developments are buzzing on the regulatory and corporate front. So for…
December 14, 2018
Dec 14, 2018 • 15 min
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…
December 7, 2018
Dec 7, 2018 • 9 min
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…
November 30, 2018
Nov 30, 2018 • 6 min
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…
November 23, 2018
Nov 23, 2018 • 11 min
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…
November 15, 2018
Nov 15, 2018 • 10 min
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…
November 9, 2018
Nov 9, 2018 • 26 min
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…
November 2, 2018
Nov 2, 2018 • 11 min
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
October 25, 2018
Oct 25, 2018 • 7 min
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…
October 18, 2018
Oct 18, 2018 • 6 min
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,…
October 11, 2018
Oct 11, 2018 • 7 min
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…
Oct 5, 2018
Oct 5, 2018 • 15 min
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 the context of key patient needs. Presenter: Max Rex Contributor: Matthew Harold, International Developed…
Sep 27, 2018
Sep 27, 2018 • 6 min
This week, P4A’s podcast will cover Luxturna’s CHMP recommendation, Novartis’ restructuring plans and Alexion’s acquisition of Syntimmune. Presenter: Max Rex Contributor: Aparna Krishnan
September 20, 2018
Sep 20, 2018 • 6 min
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 purchasing agreement and US patient groups pushing back against ICER’s influence in drug reimbursement.…
August 16, 2018
Aug 16, 2018 • 6 min
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 of ICER’s cost effectiveness analysis to decide formulary inclusions and Express Scripts is in talks…
August 9, 2018
Aug 9, 2018 • 4 min
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 Brexit transition period; Spark Therapeutics’ clinical data for its gene therapy treating heamophila A…
August 2, 2018
Aug 2, 2018 • 4 min
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 that Sanofi is stockpiling drugs. Presenter: Max Rex Contributor: Aparna Krishnan
July 26, 2018
Jul 26, 2018 • 18 min
The EU’s Commissioner for Competition Margrethe Vestager, recently said that price differences in the pharma market among different member states could be “justified”. This week, P4A delves deeper into the mechanics of drug pricing in Europe including…
July 17, 2018
Jul 17, 2018 • 8 min
The U.S. FDA’s new guidance on gene therapy was recently announced by FDA Commissioner Dr. Scott Gottlieb, the P4A team discuss key implications of these guidelines along with an update on UK’s Brexit Whitepaper and new measures by the French government…
July 11, 2018
Jul 11, 2018 • 4 min
This week, the team discuss the U.S. Centres for Medicare & Medicaid Services withdrawing support for a novel payment model for Novartis’ Kymriah; Pfizer rolling back drug price rise in the U.S. and Axovant’s deal with Benitec Biopharma. Presenter: Joanna…
July 4, 2018
Jul 4, 2018 • 9 min
This week, the P4A team discuss the CHMP approval of two CAR-T Therapies; Scotland’s new rules for ultra-orphan drugs and the reaction to the announcement of National Coverage Analysis for CAR-T drugs in the U.S. Presenter: Max Rex Contributors: Sophie…
June 26, 2018
Jun 26, 2018 • 4 min
This week, the P4A team delve into the pharma industry’s proposal to the EU on the role of national HTA bodies post harmonization of clinical assessments for innovative drug technologies; the European Medicines Agency’s new portal for orphan drug…
June 19, 2018
Jun 19, 2018 • 7 min
The P4A team give a summary of the gene therapy FDA news this week and also discuss President Trump’s drug pricing plans for Medicare and UK cost watchdog NICE’s rejection of Crysvita. Presenter: Max Rex Contributors: Aparna Krishnan, Joanna Fernandes
June 12, 2018
Jun 12, 2018 • 5 min
This week’s episode discusses the EU’s research and innovation budget; Italy’s AIFA responding to physician criticism of new guidelines and Oxford Biomedica’s $842 million deal with Axovan Sciences. Presenter: Max Rex Contributors: Aparna Krishnan,…
June 5, 2018
Jun 5, 2018 • 4 min
This week the P4A team discuss key advancements in oncology treatments - a genetic test indicating whether chemotherapy is beneficial in a particular breast cancer patient group and successful results of an immunotherapy using T cells in an advanced…
May 31, 2018
May 31, 2018 • 10 min
In its very first podcast of Rare Disease, Cell & Gene Therapy Weekly RoundUp, P4A discusses the US FDA’s accelerated regulatory process for gene therapy; President’s Trump’s proposed drug pricing plan; the European Commission’s proposal to amend SPC; the…