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
Presenter: Max Rex
Contributors: Sophie Schmitz & Alison Kneen
Weekly Roundup: July 11, 2018
Presenter: Joanna Fernandes
Contributor: Aparna Krishnan
Weekly Roundup: July 17, 2018
Presenter: Joanna Fernandes
Contributors: Christina Poschen & Aparna Krishnan
Weekly Roundup: July 26, 2018
Presenter: Max Rex
Contributor: Stuart Tutt
Weekly Roundup: August 2, 2018
Presenter: Max Rex
Contributor: Aparna Krishnan
Weekly Roundup: August 9, 2018
Presenter: Joanna Fernandes
Contributor: Aparna Krishnan
Weekly Roundup: August 16, 2018
Presenter: Max Rex
Contributor: Aparna Krishnan
Weekly Roundup: September 20, 2018
Presenter: Max Rex
Contributor: Aparna Krishnan
Weekly Roundup: September 27, 2018
Presenter: Max Rex
Contributor: Aparna Krishnan
Weekly Roundup: October 5, 2018
Presenter: Max Rex
Contributor: Matthew Harold, International Developed Markets Public Affairs Lead, Pfizer
Weekly Roundup: October 11, 2018
Presenter: Max Rex
Contributor: Nader Murad, Senior Analyst
Weekly Roundup: October 18, 2018
Presenter: Max Rex
Contributor: Christina Poschen, Consultant, P4A
Weekly Roundup: October 25, 2018
Presenter: Aparna Krishnan
Contributor: Joanna Fernandes
Weekly Roundup: November 2, 2018
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
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.