In this podcast we discuss the evolution of science that is driving the increasing focus on rare indications with Anne Pariser, the Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS). Anne is a noted international expert or rare diseases, and has been involved in numerous collaborations within the FDA, as well as with drug developers, governmental agencies, patient groups and other stakeholders in the development of treatments for rare indications.

Dr Pariser comes to NCATS from the FDA, where she had worked since 2000 on the development of drug and biological products for orphan and rare conditions. Dr Pariser also served as an associate director in the FDA’s Office of Translational Sciences, which is part of the Center for Drug Evaluation and Research (CDER).

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Peter Young is a Venture Partner at Pappas Capital, and brings to his investment approach thirty-five years of experience in the biopharma sector. The former Vice President and Therapy Head at Glaxo Wellcome in the mid-1990s, Peter led the global introduction of combination HIV therapies that revolutionized HIV treatment, quintupled Glaxo’s HIV sales, and maintained leadership on global treatment access. Peter has since served as CEO of two private start-ups, a vaccine company that raised over $80 million in NIH, HIV, and biodefense grants, and a specialty pharmaceutical company he led to a successful exit.

In a podcast recorded at the BIO International conference, Peter discusses the ramifications of the Trump Administration’s proposals for international reference pricing in Medicare Part B, as well as how to balance the higher prices being driven by smaller, targeted indications and the need to support the natural movement of science in rare therapies. We also discuss the ‘hot-button’ issues around the orphan drug legislation and how we can constructively move forward without throwing the orphan baby out with the bathwater.

This podcast is made possible with the support of EUCOPE.

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EURORDIS, a portmanteau of European Rare Disease, is a non-governmental alliance of organisations representing 860 rare disease patient organisations in 70 countries, and the 30 million people affected by rare diseases throughout Europe. CEO Yann Le Cam in 1997, one of the founders of EURORDIS, who's eldest of three daughters is living with cystic fibrosis.

With the upcoming European Commission Conference on Rare Diseases occurring on June 17th coupled with the ongoing inquiry around the OMP regulation, this timely podcast with EURORDIS’ Director of Public Affairs, Simone Boselli, puts the discussions squarely in the context of patient access to needed medicines for rare indications by developing a pan-European value-based evaluation network.

This Better Science, Better Health Podcast is made possible with the support of EUCOPE.

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Erik Tambuyzer is one of the world’s thought leaders in orphan drugs and rare disease indications. While he was Genzyme’s EU and International Senior Vice-President for Corporate Affairs, he was also the industry’s negotiation lead with the European Commission and Parliament on the Orphan Medicinal Products Regulation, introduced in the year 2000, working together with patients’ organisations and academics. As an entrepreneur in 1985, he had co-founded and managed the biotech company Innogenetics, ultimately acquired by Fujirebio. He is the Executive Chairperson and co-founder of the BioPontis Alliance for Rare Diseases, an international philanthropic foundation turning science into medicines.

This Better Science, Better Health Podcast is made possible with the support of EUCOPE.

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Nigel Hughes is the Scientific Director of Real World Evidence at Janssen Clinical Innovation. He collaborates with the EFPIA Digital Strategic Governing Group, and he is also co-lead of Big Data for Better Outcome’s €30 million flagship consortium, the European Health Data & Evidence Network, or EHDEN. Janssen and Nigel have long been at the forefront of developing the needed tools and infrastructure to harness health data, both through their involvement with previous IMI projects such as EMIF and EHR4CR, as well as supporting open source data sharing platforms such as OMOP and OHDSI.

Nigel brings his decades of experience in nursing and directing NGOs in AIDS and Hepatitis C to IMI's EHDEN. His unique perspective on the need for global collaboration to address societal challenges like Alzheimer's disease is well-reasoned and refreshing in today's fractured and divisive political climate.

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Dr. Clemens Martin Auer is currently Special Envoy for Health at the Austrian Federal Ministry of Labour, Social Affairs, Health and Consumer Protection and formerly longstanding Director General of the same institution. In this podcast, we discuss his leadership for implementation of better tools to evaluate healthcare efficiency and outcomes as the co-chair of the European eHealth Network to establish cross-border eHealth applications. In 2017, he was elected President of the European Health Forum Gastein, which under his direction has focused on improving the delivery of healthcare in the EU member states. He has been nominated by the Austrian Government to be the next WHO Regional Director for Europe, and we discuss his vision and objectives if he were to be successfully elected to that position.

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Vaccines are one of the most effective public health measures available, saving some two to three million lives worldwide every year. However, in Europe where vaccine preventable disease almost disappeared, the public is now worried about the safety of the vaccine, limiting their use, resulting in outbreaks of preventable diseases like Measles. IMI’s Advance project, which concluded in March of 2019, brought together a large cross-section of European stakeholders to create a system and network to rapidly provide real world evidence on vaccine coverage, safety and effectiveness using existing health data in EU countries.

ADVANCE has built VAC4EU: a non-for profit legal entity that will implement the ADVANCE system and vision, and in this podcast we are joined by two members of the ADVANCE co-ordinating team responsible for its launch and rebranding.

Miriam Sturkenboom, ADVANCE’s Project Coordinator
Patrick Mahy, Scientific Institute of Public Health in Belgium

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Peter Pitts is President and co-founder of the Center for Medicine in the Public Interest, Prior to founding CMPI, Pitts was a Senior Fellow for healthcare studies at the Pacific Research Institute. From 2002-2004 Peter was FDA’s Associate Commissioner for External Relations, serving as senior communications and policy adviser to the Commissioner. He is the editor of the new book, Coincidence or Crisis, a discussion of global prescription medicine counterfeiting.

In this podcast, we discuss the potential impact of the International Pricing Index (IPI) on global innovation, as well as several alternatives which could be put in place that would create less havoc for innovators and patients. As well, we discuss the increasing divergence of the innovative capacity between the US and Europe, and how the current systems of reimbursement are impacting the ability to support and R&D ecosystem.

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Joe Damond is the Executive Vice President for International Affairs at the Biotechnology Innovation Organization (BIO), the largest service organisation for biotech companies in the world. In this podcast we discuss the continuing attacks on Intellectual Property in Europe and the US, as well as how the International Pricing Index (IPI) of the Trump administration will impact biotech investment and innovation.

Joe spent 12 years as a trade negotiator at the Office of the United States Trade Representative for Asia and Pacific/APEC Affairs, and is a well-respected thought-leader helping governments develop sustainable biotech ecosystems. Joe Damond received his master’s degree from Princeton University’s Woodrow Wilson School of Public and International Affairs.

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Vital Transformation launches its new series of Better Science, Better Health podcasts focused on the challenges and opportunities of new medicines. With the increasing focus on drug pricing in the US Congress and Senate, and with the Trump Administration's proposals for international reference pricing on therapies covered under Medicare Part B, Vital Transformation shares the insights of Dr. Mark McClellan, one of the world's most important thought leaders in value-based healthcare.

Mark McClellan is the founding Director of the Duke-Margolis Center for Health Policy at Duke University. He is a world-renowned physician and economist who has researched outcomes-based reimbursement and real-world evidence evaluation models for new therapies. Dr McClellan was both the former administrator of the Centers for Medicare & Medicaid Services and Commissioner of the FDA. He was a Senior Fellow in Economic Studies at the Brookings Institution, where he was the Director of the Health Care Innovation and Value Initiatives.

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