A weekly podcast highlighting the most important news developments and its impact on the orphan drug, cell and gene therapy world including research advancements, corporate events and regulatory changes.

Weekly Roundup: May 31, 2018

In its very first podcast of Rare Diseases 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.

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 result 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

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 Axovant Sciences.
Presenter: Max Rex
Contributors: Aparna Krishnan and Joanna Fernandes

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 as well as UK cost watchdog NICE’s rejection of Crysvita.
Presenter: Max Rex
Contributors: Aparna Krishnan, Joanna Fernandes

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.
Presenter: Joanna Fernandes
Contributor: Aparna Krishnan

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 & Alison Kneen

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

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

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

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

Weekly Roundup: August 9, 2018

In this episode, the team 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

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

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

Weekly Roundup: September 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

Weekly Roundup: October 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

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

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

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

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

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.

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

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

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

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

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

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.

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