In this issue

 

 

Our editors examine how real-world evidence impacts regulatory decisions in the United States, the current state of reimbursement for advanced therapy medicinal products (ATMPs) in Germany, and a look at two value assessment frameworks, Innovation and Value Initiative (IVI) and the Institute for Clinical and Economic Review (ICER).

Real-world evidence in United States regulatory decisions

Real-world evidence (RWE) has garnered increased attention in recent years to advance drug development. The framework for the Food and Drug Administration’s (FDA’s) RWE Program, published in 2018, serves as a roadmap for the inclusion of real-world data (RWD) and RWE in regulatory decisions, including standards on how RWD are defined, collected, and analyzed. This article reviews the use of RWE in United States (US) regulatory decisions after the publication of the FDA’s RWE framework and describes how manufacturers can leverage RWE to optimize their product launch.

Outlook for advanced therapy medicinal product price negotiations in Germany

Aligning the interests of pharmaceutical manufacturers, health technology assessment authorities, and healthcare payers is a complex venture, particularly for advanced therapy medicinal products (ATMPs). In this article, we present the current state of reimbursement for ATMPs and strategies to facilitate reimbursement under unique circumstances.  

ATMPs include gene therapy medicinal products, somatic cell therapy medicinal products, tumor vaccines, and tissue-engineered products. Most of the approved ATMPs in Germany are orphan drugs and, due to small patient population sizes (and the oftentimes accelerated authorization), only limited evidence is available to determine the long-term value of these products. These factors are not only a challenge for the benefit assessment but also for the subsequent price negotiation. 

A comparison of the Innovation and Value Initiative’s Open-Source Value Project and the Institute for Clinical and Economic Review’s value assessment framework

With the rise in high-cost treatments and growing emphasis on value-based healthcare, value assessment frameworks (VAFs) have been increasing in prominence in the United States (US). These frameworks have been recently evolving to better reflect the input of patients and various other stakeholders. Two well-known VAFs, the Institute for Clinical and Economic Review (ICER) and Innovation and Value Initiative (IVI), have both recently refined their methods in this regard; however, there remain notable differences in terms of how thoroughly their analyses reflect multistakeholder perceptions of value.

HTA by the numbers

 

501 million

According to data collected by Bloomberg, more than 501 million doses of COVID-19 vaccinations have been administered across 140 countries. The latest rate of administration is approximately 12.2 million doses a day.

Heard on the street

 

“We are proposing to establish statutory ICSs, made up of an ICS NHS Body and an ICS Health and Care Partnership (together referred to as the ICS), to strengthen the decision-making authority of the system leadership and to embed accountability for system performance into the NHS accountability structure. This dual structure recognises that there are 2 forms of integration which will be underpinned by the legislation: the integration within the NHS to remove some of the cumbersome barriers to collaboration and to make working together across the NHS an organising principle; and the integration between the NHS and others, principally local authorities, to deliver improved outcomes to health and wellbeing for local people.”

The UK Department of Health & Social Care has released a policy paper with potentially significant implications titled Integration and Innovation: Working Together to Improve Health and Social Care for All. The policy paper includes several changes to promote greater collaboration and reduce barriers to integrated care. Some of the most significant changes proposed are related to integration within the NHS and between NHS and local governments.

Featured ICER Research

In the latest edition of Value & Outcomes Spotlight, Xcenda’s Ken O’Day brings clarity to ICER’s use of the equal value of life years gained (evLYG) metric in its 2020–2023 Value Assessment Framework. Check out the article Demystifying ICER’s Equal Value of Life Years Gained Metric to understand why the evLYG metric was created, what it is, how it is calculated, and the impact of evLYG on ICER’s assessments.

 

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Featured contributors

 

Advisory board:

Josep Darba, PhD
Professor | Department of Economics | University of Barcelona | Spain

George Papadopoulos, BSc(Hons), GradDipEpi 
Director & Partner | Lucid Health Consulting | Australia

Ken Redekop, PhD  
Associate Professor I Erasmus University I The Netherlands

Eldon Spackman, BA, MA, PhD 
Co-Director | Institute for Public Health | University of Calgary | Canada

Keith Tolley, BA, MPhil 
Director | Tolley Health Economics | United Kingdom

Thomas Mittendorf, MSc, PhD 
Managing Director | Xcenda GmbH | Germany

Trent McLaughlin, BSc(Pharm), PhD
Vice President, Real-World Evidence | Xcenda | United States

Michael Eaddy, PharmD, PhD  
Vice President, Scientific Consulting | Xcenda | United States

Jay Jackson, PharmD, MPH
Senior Vice President, Consulting Services | Xcenda | United States

Contributing authors:

Charley Hallock, PharmD | Mareike Konstanski | Ulrike Kuchenbecker, PhD | Trent McLaughlin, BSc(Pharm), PhD | Aishani Patel, PharmD | Robin Tan, PhD | Kristina Thamm, PhD

Production:

Ellen Olson | Lisa Peachey | Elana Gruen