Rare Disease, Cell & Gene Therapy Weekly RoundUp

48 EpisodesProduced by Partners4Access

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/

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Weekly Roundup: July 1, 2019

July 1st, 2019


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 …

Weekly Roundup: June 23, 2019

June 23rd, 2019


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: …

Weekly Roundup: June 16, 2019

June 16th, 2019


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

Presenter: Aparna …

Weekly Roundup: June 12, 2019

June 12th, 2019


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

Weekly RoundUp: June 3, 2019

June 3rd, 2019


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

Weekly Roundup: May 25, 2019

May 25th, 2019


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 …

Weekly Roundup: May 18, 2019

May 18th, 2019


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 …

Weekly Roundup: May 12, 2019

May 12th, 2019


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 …

Weekly Roundup: May 3, 2019

May 3rd, 2019


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 …

Weekly Roundup: April 27, 2019

April 27th, 2019


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

Weekly Roundup: April 22, 2019

April 22nd, 2019


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

Weekly Roundup: April 12, 2019

April 17th, 2019


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

Weekly Roundup: March 28, 2019

March 28th, 2019


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 …

Weekly Roundup: March 22, 2019

March 22nd, 2019


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 …

Weekly Roundup: March 15, 2019

March 15th, 2019


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, …

Weekly Roundup: March 8, 2019

March 8th, 2019


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

Weekly Roundup: February 28, 2019

February 28th, 2019


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

Weekly Roundup: February 22, 2019

February 22nd, 2019


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

Weekly Roundup: February 15, 2019

February 15th, 2019


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

Weekly Roundup: February 1, 2019

February 1st, 2019


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 …

Weekly Roundup: January 25, 2019

January 25th, 2019


 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, …

Weekly Roundup: January 18, 2019

January 18th, 2019


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 …

Weekly Roundup: January 11, 2019

January 14th, 2019


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 …

Weekly Roundup: December 14, 2018

December 14th, 2018


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 …

Weekly Roundup: December 7, 2018

December 7th, 2018


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

November 30th, 2018


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 …

Weekly Roundup: November 23, 2018

November 23rd, 2018


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

November 15th, 2018


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: …

Weekly Roundup: November 9, 2018

November 9th, 2018


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 …

Weekly Roundup: November 2, 2018

November 2nd, 2018


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

Weekly Roundup: October 25, 2018

October 25th, 2018


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 …

Weekly Roundup: October 18, 2018

October 18th, 2018


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

Weekly Roundup: October 11, 2018

October 11th, 2018


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

Weekly Roundup: Oct 5, 2018

October 5th, 2018


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

September 27th, 2018


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

Weekly Roundup: September 20, 2018

September 20th, 2018


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 …

Weekly Roundup: August 16, 2018

August 16th, 2018


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 …

Weekly Roundup: August 9, 2018

August 9th, 2018


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 …

Weekly Roundup: August 2, 2018

August 2nd, 2018


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 …

Weekly Roundup: July 26, 2018

July 26th, 2018


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

Weekly Roundup: July 17, 2018

July 17th, 2018


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 …

Weekly Roundup: July 11, 2018

July 11th, 2018


This week, the team discuss the U.S. Centres for Medicare & Medicaid Services withdrawing support for a novel payment model for Novartis' …

Weekly Roundup: July 4, 2018

July 4th, 2018


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 …

Weekly Roundup: June 26, 2018

June 26th, 2018


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 …

Weekly Roundup: June 19, 2018

June 19th, 2018


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: …

Weekly Roundup: June 12, 2018

June 12th, 2018


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

Weekly Roundup: June 5, 2018

June 5th, 2018


This week the P4A team discuss key advancements in oncology treatments - a genetic test indicating whether chemotherapy is beneficial in a particular …

Weekly Roundup: May 31, 2018

May 31st, 2018


In its very first podcast of Rare Disease, Cell & Gene Therapy Weekly RoundUp, P4A discusses the US FDA's accelerated regulatory process for gene …

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