Conference Program/Sessions

Overview
This Issue Panel will focus on current market access challenges with innovative cell and gene therapies in oncology and rare diseases and potential solutions for enabling patient access. It will address key access considerations in defining a compelling value proposition of a new therapy in diseases with low awareness as well as value-based pricing of one-time, high-cost “curative” therapies.  In addition, it will highlight a prominent European HTA perspective on the valuation of cell and gene therapies.

Learning Objectives

• To discuss the challenges of the standard value framework when applied to one-time “curative” therapies and potential solutions to address them
• To outline current market access challenges with innovative therapies in oncology rare diseases and cell & gene therapies
• To understand pragmatic challenges as well as opportunities in the health technology appraisal of oncology rare diseases and cell & gene therapies through the lens of German HTA

This workshop will focus on market access challenges and evidence-generation opportunities associated with innovative treatments for neurological diseases. Although we will focus on neurological conditions, the issues and potential approaches discussed broadly apply to many therapeutic areas, particularly where outcomes are (i) not easily monetizable, (ii) reliant on patient and/or proxy reporting, or (iii) may lack objectivity and are not easily measured. In addition, the issues discussed in the workshop will also apply to conditions where it is challenging to translate the impact on daily functioning or caregiver outcomes into payer savings.

Learning Objectives

• To outline challenges and potential strategies and tools that can support the generation of value-based evidence in neurological conditions, particularly patient populations with unmet needs.
• To illustrate an approach to leveraging patient registry data to develop new patient-reported outcome measures that improve the ability to capture change in patients' health and quality of life.
• To discuss strategic approaches to value assessment frameworks and other approaches to quantify patient value, such as value-based agreements with payers predicated on outcomes.

Topics will include:

• ‘Market access thinking’ across medical device product life cycle
• Stakeholders and value frameworks 
• Role of (real-world) evidence in medical device market access 
• Maximizing the value of healthcare robotics and digital solutions

• Antipasto Bar
• Miso Glazed Chilean Seabass Station
• Churrascaria Grilled Skirt Steak Station
• Dim Sum Bar
• Pasta Station (Includes Vegetarian)
• Wynwood Pastry Art (Dessert Station)
• Sparkle Premium Alcohol Beverage Package

This session will present current application and barriers of using RWE in decision making.  A brief review of the purpose of RWE, the barriers in both US payer decisions, use in EU HTA decisions and unique value-based contracting for high-cost precisions medicines will stimulate a panel discussion of how these barriers can be overcome.

Overview
Engage with a small panel of industry experts to discuss how global pricing trends such as evolving International Reference Pricing Rules and government pricing policy reforms shapes global market access strategies, both in terms of the evolution to date and expected future trends.

Learning Goals:

• Deeper understanding of payer, government & Biopharma perspectives on pricing pressures
• Learn about how evolving pricing rules (such as IRP), pricing policies & reforms (such as IRA) will impact the BioPharma industry
• Gain insight into how these pricing dynamics & trends will continue to share BioPharma pricing and access strategies across the globe

Overview
This plenary session will focus on the recently adopted EU-HTA regulation, the aim of which is to harmonize HTA assessments across the EU member stats, increase predictability of reimbursement decisions and thus facilitate early access to market of innovative and life saving products. In 2025 joint EU HTA will become mandatory for cancer drugs and advanced therapy medicinal products.

The expert panel will discuss challenges related to its implementation, process, hurdles in data requirements (such as multiple PICOs) and concerns of different stakeholders: health-technology developers, patients, regulators and HTA bodies. Initiatives for providing common HTA involving several non-EU countries also exist.
 

Experts will discuss whether EU-HTA model might be used outside Europe – common comparative HT assessment to support evidence-based decision making - and possible implications for manufacturers, their investments in market access and how to navigate in the evolving ecosystem.

Learning Objectives

• Understand common comparative health technology assessment to support evidence-based decision making
• Understand EU-HTA process and content
• Explore if EU-HTA model may be adapted to other multi-country collaborations.

At the end of this session, participants will be able to:

• Describe the key elements of the health care provisions of the Inflation Reduction Act
• Understand the Medicare Prescription Drug Negotiation Program and its Implementation
• Discuss the implications of the MPDNP for industry and payors.

View and discuss the latest research and best practices with industry leaders, researchers, and stakeholders. Presenters will be available next to their poster to discuss their poster content. 

Overview:

This session will focus on the utility of applying a hub and spoke model to enhance market access to cell and gene therapy (CGT) in LMICs. The overall approach will be based on geographical and service coverage of CGT in such countries through a modeled designation of health care facilities as hubs, spokes and partner spokes. The session will present the assumptions and perimeters of the hub and spoke model. In addition, a simulation exercise for applying hub and spoke model for CGT for Brazil and the Middle East and North Africa (MENA) region.

In addition, practical and systematic challenges and applications will be discussed and presented to highlight the adoption and potential uptake from such a model.

Learning Objectives

1. To discuss potential applications and challenges for the adoption of hub and spoke models to enhance access to cell and gene therapy in LMICs.
2. To present a practical approach to hub and spoke facility-based model implementation for CGT in LMICs based on geographic and service coverage.
3. To share a simulation of the implementation of the hub and spoke model for CGT in Brazil and the Middle East and North Africa (MENA) region

Innovative contracting is rapidly changing the ways in which we negotiate. This more sophisticated approach requires that we elevate both our thought process and our executional capabilities if we want to successfully realize win-win agreements. Join this workshop-style session where we will explore as a group three important themes:

1. The Landscape: Using secondary data and market research insights, we will discuss global-to-local trends in innovative contracts
2. The Possible in 2022: Post-pandemic, we see a 54% year-over-year increase in published contracts. What’s emerging that is exciting or innovative, and what do these contracts tell us about what is possible?
3. The Art of the Build: What does it take to create these types of contracts? Key considerations and best practices

Plan for a dynamic 75-minutes involving a mix of presentation and group discussion. Whether you’re already involved in building innovative contracts or new to this space, this is a session you won’t want to miss!

Overview:

This workshop will discuss the emerging and more comprehensive consideration of the Value of Vaccination (VoV) in the context of vaccines market access. An overview of vaccines market access pathways will be provided including country examples of US and EU and highlighting steps in the pathway where VoV considerations by different stakeholders in the vaccines eco-system apply. Following, the evolution from traditional to more comprehensive VoV frameworks and their application in vaccines HTA also considering the learnings from COVID-19 will be discussed. Recent examples of VoV consideration in vaccines HTA will be used to illustrate the evolution, opportunities and challenges for more comprehensive consideration of the VoV. This will be an interactive workshop for participants and panelists including for example polls and Q&A.

Learning Objectives

1. To obtain a solid understanding of vaccines market access pathways' specifities and value considerations of different stakeholders
2. To obtain a solid understanding of the evolution and the concepts underpinning more comprehensive considerations of the Value of Vaccination including Covid-19 learnings
3. To discuss opportunities and challenges associated with a more comprehensive consideration of the VoV for future vaccines market access

• Conventional measures of value (surveys and health economics) can be biased, costly, and time consuming.
• Often “actions speak louder than words” and past behavior can be a reliable indicator of future behavior.
• Two approaches to finding patterns in past behavior are AI and econometrics. We discuss how they work and the benefits of combining them.
• The importance of the right approach: curation, validation, and avoiding over-fitting.

Overview

This workshop will discuss key aspects of how Covid-19 vaccines and vaccination may shape in the near to mid-term vaccines market access. Following an introductory overview of the current state for Covid-19 vaccination programmes, the panellists will debate how Covid-19 has led to broader value considerations in vaccines HTA and potential future evolvements. As Covid-19 vaccination programmes were key to control the pandemic, arising market access schemes for vaccination based on pandemic and insurance value will be discussed. Lastly, Covid-19 implications for vaccines market access on a mid-term horizon will be debated considering new technologies, growth of vaccines R&D pipeline, shift to lifecourse immunisation and corresponding opportunities and challenges. This workshop will be interactive with debate among participants and panellists including for example polls and Q&A.

This workshop aims to familiarize market access managers in the pharmaceutical industry with artificial intelligence (AI) and its present and potential applications in market access. There will be an introduction to key AI concepts and some of the primary applications now used in the pharmaceutical sector. Several illustrations of the utilisation of AI in the field of market access will be presented: utilisation of topic modelling to identify important concepts from a large corpus of documents (HAS decision committee meeting transcripts); prediction of outcomes of HTA in European countries; generation of real-world evidence; generation of synthetic medical datasets. The workshop will conclude with a discussion around opportunities and challenges associated with applications of AI.

Learning objectives:

By the end of the workshop, participants will be able to:

• understand key concepts and take part in discussions related to digitalisation and artificial intelligence in the pharmaceutical industry.
• understand how AI can transform market access activities and improve strategic decision-making
• integrate AI into plans for generating evidence on the value of pharmaceutical products, and in particular real-world evidence (RWE)
• identify opportunities and challenges that may arise from future AI applications in market access.

This pre-conference short course will be offered in-person at the ACCESS 2023 Annual Meeting and Expo. Separate registration is required, although there is no fee to attend this session. Registration is limited to the first 50 participants. Please add this event to your main conference registration. 

Ashley Jaksa will discuss work she has done reviewing RWE guidance from HTA groups and payers to identify what are the critical features of RWE that make it most credible and impactful. She will also discuss work that she is doing with CADTH regarding SMA, to identify and prioritize evidence gaps in SMA that are most suitable for RWE studies.

Nathan Sigworth will discuss the technology platform he is working on that can support novel contracting mechanisms, and how this can be deployed to support the evidence generation needed by HTA and payers.

Learning Objectives:

1. To outline challenges and potential strategies and tools that can support the generation of evidence to address HTA and payer decision making for orphan disease therapies.
2. To better understand the perspectives of HTA groups and payers on the use of RWE to inform their decision making.
3. To become familiar with the potential role of technology in facilitating novel payments mechanisms that promote evidence generation.

This pre-conference short course will be offered in-person at the ACCESS 2023 Annual Meeting and Expo. Separate registration is required, although there is no fee to attend this session. Registration is limited to the first 50 participants. Please add this event to your main conference registration. 

DESCRIPTION

Unlike marketing authorization for pharmaceuticals, mainly regulated at the European level by EMA, pricing and reimbursement decisions in Europe are managed by individual member states. Health care services are generally covered by a single public health insurer operating under the Ministry of Health supervision. As a monopoly buyer, this situation provides a leading position for the public health insurer to set reimbursement conditions. Therefore, based on each country’s set of regulations, processes, and values, wide variations exist in pricing and reimbursement decisions of pharmaceuticals. Using up-to-date governmental regulation sources, this course will discuss health technology decision-making processes for reimbursement decisions for pharmaceuticals in various countries, including France, Germany, Hungary, Italy, Poland, Spain, Sweden, and the UK. The course will describe these reimbursement systems, as well as compare, and bring into contrast their key characteristics. This course is designed for individuals with intermediate experience within a single healthcare system wishing to broaden their appreciation of other reimbursement systems.

In this interactive workshop, participants will discuss the importance of leveraging patient voice across the development and commercialization continuum to inform key stakeholders’ decisions, and ultimately better serve the entire healthcare ecosystem—from the patients themselves, to payers, providers and manufacturers.

We will discuss the benefits of “patient centricity”, WHAT it is, and WHERE it can take place. We will provide an overview of current methods and provide a framework for increasing the usefulness and impact of patient preference studies in decision making in health.

Finally, we will break into small groups and have table-top discussions focusing on real world application of these principles and what the participants are doing—or have seen—that have been effective. These will be debriefed in the broader group, focusing on KEY LEARNINGS and TAKE-AWAYS.