Rare Diseases Weekly Roundup

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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