Rare Disease, Cell & Gene Therapy Weekly RoundUp

Weekly Roundup: February 22, 2019

An episode of Rare Disease, Cell & Gene Therapy Weekly RoundUp

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/
Available on 11 platforms
Weekly Roundup: March 22, 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 effectiveness and negotiate prices in Canada.  Presenter: Max Rex Contributor and Producer: Aparna Krishnan
March 22, 2019
Weekly Roundup: March 15, 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, Ciaran Cassidy Producer: Aparna Krishnan
March 15, 2019
Weekly Roundup: March 8, 2019
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, Vertex's continued struggle to get its cystic fibrosis drug Orkambi reimbursed in the UK.  Presenter: Joanna Fernandes Contributor and Producer: Aparna Krishnan
March 8, 2019
Weekly Roundup: February 28, 2019
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 enables patients to find treatment in healthcare facilities outside their home country. However, there are several challenges associated with it.  Presenter: Christina Poschen Contributor: Dr Andrzej Rys, Director - Health Systems and Products Producer: Aparna Krishnan
February 28, 2019
Weekly Roundup: February 22, 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 Contributor: Jan-Willem Schmitz, General Manager for Nordics and Baltics, Sanofi Genzyme Producer: Aparna Krishnan
February 22, 2019
Weekly Roundup: February 15, 2019
 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 successful treatment access for patients. The 6Ps are Partnership, Patient, Policy-maker, Physician, Pharma and Payer. This week we focus on the payer who are decision-makers assessing the value of a product in the healthcare system.   Presenter and Producer: Aparna Krishnan Contributor: Einar Andreassen, senior advisor at the Norwegian Medicines Agency. Einar is a health technology assessment analyst and reimbursement decision maker for the national insurance scheme in Norway.  
February 15, 2019
Weekly Roundup: February 1, 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 challenges and its impact on people and society. The 6Ps are Partnership, Policy-maker, Payer, Pharma, Physician and Patient. Our message: 6Ps are essential to successfully serve the rare disease community and achieve access to medicines. This week, we discuss the first P – Partnership. The team reflects on the meaning of partnership in the rare disease context and what it can achieve through examples of different types of collaborations. From patient and clinical experts influencing HTA decisions; key stakeholders coming together for the Hercules Project to a collaboration between US based ICER, Canada's CADTH and UK's NICE. Presenter: Aparna Krishnan Contributors: Sophie Schmitz, Christina Poschen, Max Rex, Joanna Fernandes, Nader Murad and Jack Rawson
February 1, 2019
Weekly Roundup: January 25, 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,  P4A is starting a new campaign that will run throughout February to mark Rare Disease Day.  The '6P'  campaign is aimed at creating awareness on the impact of rare diseases on key stakeholders and the need for partnership to achieve successful treatment access for patients. The 6Ps are Partnership, Patient, Policy-maker, Physician, Pharma and Payer. P4A will roll out a series of podcast episodes featuring thought leaders representing these stakeholders. So make sure you listen in! Presenter: Joanna Fernandes Contributors: Nader Murad and Sophie Schmitz Producer: Aparna Krishnan
January 25, 2019
Weekly Roundup: January 18, 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 Fernandes Contributor: Max Rex Producer: Aparna Krishnan
January 18, 2019
Weekly Roundup: January 11, 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 couple of weeks in and already news developments are buzzing on the regulatory and corporate front. So for this week's episode, we start by discussing the recent wave of  mergers and acquisition deals announced by Big Pharma namely, Eli Lilly and Loxo; BMS and Celgene as well as GSK and Tesaro. Also, the P4A team look at the implications of the current US government shutdown on the FDA and the agency's initiative on assessments for innovative drugs.  Presenter : Max Rex Contributor : Joanna Fernandes Producer: Aparna Krishnan
January 14, 2019
Weekly Roundup: December 14, 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 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.
December 14, 2018
Weekly Roundup: December 7, 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 access scheme and clinical trial updates from bluebirdbio’s gene therapy LentiGlobin. Presenter: Joanna Fernandes Contributor: Aparna Krishnan
December 7, 2018
Weekly Roundup: November 30, 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 e-prescribing and the OECD's new report on access to medicines. Presenter: Aparna Krishnan Contributor: Jack Rawson
November 30, 2018
Weekly Roundup: November 23, 2018
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
November 23, 2018
Weekly Roundup: November 15, 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: Aparna Krishnan Contributors: Sophie Schmitz, Managing Partner, P4A and Diane Kleinermans, Adviser to the Ministry of Health and Social Affairs, Belgium
November 15, 2018
Weekly Roundup: November 9, 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 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
November 9, 2018
Weekly Roundup: November 2, 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 Contributors: Max Rex and Jack Rawson
November 2, 2018
Weekly Roundup: October 25, 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 providing recommendations on areas to prepare for as deadline for UK's exit looms. Presenter: Aparna Krishnan Contributor: Joanna Fernandes
October 25, 2018
Weekly Roundup: October 18, 2018
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
October 18, 2018
Weekly Roundup: October 11, 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 Contributor: Nader Murad, Senior Analyst
October 11, 2018
Weekly Roundup: Oct 5, 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 the context of key patient needs. Presenter: Max Rex Contributor: Matthew Harold, International Developed Markets Public Affairs Lead, Pfizer
October 5, 2018
Weekly Roundup: Sep 27, 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
September 27, 2018
Weekly Roundup: September 20, 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 ICER's influence in drug reimbursement. Presenter: Max Rex Contributor: Aparna Krishnan
September 20, 2018
Weekly Roundup: August 16, 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 use of ICER's cost effectiveness analysis to decide formulary inclusions and Express Scripts is in talks with several pharma companies for exclusive distribution rights to sell their upcoming gene therapies. Presenter: Max Rex Contributor: Aparna Krishnan
August 16, 2018
Weekly Roundup: August 9, 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 gene therapy treating heamophila A patients and finally, the Irish government plans to reform the country’s healthcare system. Presenter: Joanna Fernandes Contributor: Aparna Krishnan
August 9, 2018
Weekly Roundup: August 2, 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 that Sanofi is stockpiling drugs. Presenter: Max Rex Contributor: Aparna Krishnan
August 2, 2018
Weekly Roundup: July 26, 2018
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 discussing the impact of parallel trade and comparing it to U.S. pricing. Presenter: Max Rex Contributor: Stuart Tutt
July 26, 2018
Weekly Roundup: July 17, 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 measures by the French government to improve patient access to innovative drugs. Presenter: Joanna Fernandes Contributors: Christina Poschen & Aparna Krishnan
July 17, 2018
Weekly Roundup: July 11, 2018
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 Fernandes Contributor: Aparna Krishnan
July 11, 2018
Weekly Roundup: July 4, 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 announcement of National Coverage Analysis for CAR-T drugs in the U.S. Presenter: Max Rex Contributors: Sophie Schmitz, Managing Partner & Alison Kneen, Vice President, International Operations
July 4, 2018
Weekly Roundup: June 26, 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 assessments for innovative drug technologies; the European Medicines Agency's new portal for orphan drug designation applications and Ireland's entry into the Beneluxa Initiative. Host: Joanna Fernandes Contributor: Aparna Krishnan
June 26, 2018
Weekly Roundup: June 19, 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: Aparna Krishnan, Joanna Fernandes
June 19, 2018
Weekly Roundup: June 12, 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 Contributors: Aparna Krishnan, Corporate Affairs Lead Joanna Fernandes, Consultant
June 12, 2018
Weekly Roundup: June 5, 2018
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 breast cancer patient. In addition, news involving a potential new sickle cell therapy under development. Presenter: Max Rex Contributors: Alison Kneen and Aparna Krishnan
June 5, 2018
Weekly Roundup: May 31, 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 therapy; President's Trump's proposed drug pricing plan; the European Commission's proposal to amend SPC; the NHS England budget ringfencing; Irish government's attempt to trim the HSE and the new GDPR laws. Max Rex hosts with contributions from Aparna Krishnan, Corporate Affairs Lead and Alison Kneen, Vice President, International Operations.
May 31, 2018
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