Agenda

Innovation based on known molecules is becoming an increasingly important driver of pharmaceutical development. Whether referred to as Value-Added Medicines, Hybrid Medicines, 505⁠(b)⁠(2) products, or other patient-centric approaches, these developments offer new opportunities to address unmet needs, improve patient outcomes, and create value for healthcare systems. This introductory session will provide an overview of the key concepts, market trends, and regulatory frameworks shaping the field in Europe and the United States, setting the scene for the opportunities and challenges discussed throughout the conference.

Value-added medicines (VAMs) represent an increasingly important category of healthcare innovation, generating additional benefits from established medicines, mainly through reformulations, fixed-dose combinations, repurposing, improved delivery systems.

As healthcare systems seek solutions that improve patient outcomes, treatment adherence, healthcare experience, and resource utilization, VAMs offer the potential to deliver meaningful benefits beyond traditional measures of efficacy and safety.

The session will exemplify the multidimensional impact of VAMs across key stakeholders, including patients and caregivers, healthcare professionals, and healthcare systems. It will also discuss the challenges associated with defining, measuring, and recognizing their contribution, including lack of consensus in the definition of “Added Value”, evidence-generation requirements, and the absence of dedicated regulatory and reimbursement pathways.

The 505⁠(b)⁠(2) regulatory pathway offers a streamlined route to U.S. FDA approval by enabling sponsors to leverage existing nonclinical and clinical data from previously approved products. It is commonly used for modified or repurposed drugs, such as new formulations or alternative routes of administration. While this approach can reduce development timelines and costs compared to a traditional New Drug Application (NDA), it does not eliminate the requirement to demonstrate safety and efficacy for the new product.

From a nonclinical perspective, the central challenge in 505⁠(b)⁠(2) development is the establishment of a robust and scientifically justified "bridge" to the reference listed drug. Failure to establish this bridge—or to adequately address product-specific differences—can lead to regulatory delays or rejection, negating the anticipated efficiencies of the pathway.

This presentation will focus on the nonclinical strategy supporting 505⁠(b)⁠(2) development, including key considerations for identifying and addressing data gaps, designing targeted nonclinical programs, and aligning with regulatory expectations. Particular emphasis will be placed on the role of pharmacokinetic and pharmacodynamic bridging, the evaluation of formulation and route-dependent risks, and the integration of prior knowledge with new data.

We will also discuss critical pre-IND planning steps and practical approaches to de-risking development through early regulatory engagement and strategic study design.

Overall, this session will provide a framework for developing fit-for-purpose nonclinical strategies that support a successful 505⁠(b)⁠(2) submission.

As the pharmaceutical industry increasingly focuses on added-value medicines, intellectual property plays a key role in supporting differentiation and commercial sustainability. This session will provide practical insights from an in-house pharmaceutical perspective and will discuss different options to build IP strategies around reformulations, repurposing, new indications, and new fixed-dose combinations in Europe and the United States. The presentation will address patent drafting strategies, freedom-to-operate considerations and litigation risks, as well as the interaction between patents and regulatory exclusivities related to VAM products.

Within the 505⁠(b)⁠(2) development landscape, business development plays a critical but often underappreciated role — not in asset origination, but in enabling sponsors to navigate complexity, align stakeholders, and execute a coherent development strategy. This session contributes a business development perspective focused on how BD professionals support sponsors throughout the lifecycle of a 505⁠(b)⁠(2) program by recognizing strategic potential, assembling the right expertise, and facilitating cross-functional and external alignment.

As part of a broader two-day seminar on 505⁠(b)⁠(2) development, this presentation will highlight how business development functions as a strategic connector between sponsors, scientific teams, regulatory advisors, and external partners. Emphasis will be placed on BD's role in helping sponsors navigate procurement decisions, engage appropriate vendors and collaborators, and align nonclinical, clinical, and regulatory strategies to support efficient development and a successful regulatory submission.

The session will also underscore how early scientific and nonclinical insights inform business decisions, reduce execution risk, and strengthen interactions with regulatory agencies. By ensuring that development plans are not only scientifically sound but also operationally and strategically aligned, business development helps position 505⁠(b)⁠(2) programs for timely progression toward market entry.

This presentation is intended for attendees involved in sponsor support, program leadership, and cross-functional development — offering practical insight into how effective business development engagement contributes to clarity, coordination, and success in 505⁠(b)⁠(2) drug development.

Our role in this seminar is to connect the scientific and regulatory mechanics of 505⁠(b)⁠(2) development with the commercial strategy that ultimately determines success. Drawing on business development experience that spans opportunity assessment, cross-functional planning, and partnering, we will focus on how 505⁠(b)⁠(2) functions not only as a regulatory pathway, but as a business model.

Specifically, our contribution will address: 505⁠(b)⁠(2) as a business strategy — how the pathway enables faster time to market, lower capital intensity, higher probability of technical success, and the creation of differentiated, partner-ready assets; cross-functional alignment — how business development works alongside nonclinical, clinical, CMC, and regulatory teams to define data gaps, select efficient bridging strategies, and build development plans that are both scientifically credible and commercially realistic; and partnering and deal-making considerations — what investors, licensors, and acquirers look for in 505⁠(b)⁠(2) programs, how to package the regulatory and development story, how to frame residual technical risk, and how early scientific insights influence valuation and deal structure.

We can illustrate these points through non-confidential examples demonstrating how early alignment between science, regulation, and commercial strategy can significantly increase asset value and reduce downstream risk.

Constance currently works as a Value Added Medicines Policy Manager at Medicines for Europe. In her position, she manages the Value Added Medicines Sector Group workstream, ensures tight collaboration with both institutional and non-institutional stakeholders, and advocates for better value-added medicines uptake in Europe. With experience at national and European levels, Constance actively supports policies enabling timely, equitable, and sustainable off-patent medicines for European patients.

The U.S. healthcare market presents significant opportunities for European pharmaceutical developers pursuing the 505⁠(b)⁠(2) regulatory pathway, yet many programs underestimate the influence of managed care on product adoption, pricing, prescribing behavior, and long-term commercial viability. While the 505⁠(b)⁠(2) pathway can reduce development timelines and clinical risk, commercial success in the United States increasingly depends on aligning clinical differentiation with payer expectations early in development.

This presentation will provide a practical framework for understanding the structure and dynamics of the U.S. managed care ecosystem. The session will de-mystify how coverage, reimbursement, formulary placement, and utilization management decisions are made and why these decisions can dramatically influence uptake even after regulatory approval.

The presentation will examine why market access considerations should be integrated during early clinical and commercial planning for 505⁠(b)⁠(2) assets, particularly when differentiation versus generic or branded standards of care may appear modest from a payer perspective. Attendees will gain insight into how payers evaluate value propositions across therapeutic classes. Specific attention will be given to how reimbursement expectations differ among therapeutic areas.

Key topics will include: understanding the U.S. managed care landscape and key decision-makers; how PBMs and payers influence prescribing and access; formulary positioning, step edits, prior authorization, and coverage dynamics; how therapeutic class shapes coverage decisions; criteria used by payers to evaluate 505⁠(b)⁠(2)s; similarities and differences between U.S. payer evaluations and HTA; and designing your development program to maximize payer coverage.

Through real-world case studies and examples, the session will highlight how early understanding of managed care can improve development strategy, optimize positioning, reduce commercialization risk, and enhance investor confidence for emerging biopharma companies entering the U.S. market.

This presentation is intended for biotechnology executives, clinical development leaders, regulatory strategists, investors, and commercial teams involved in developing or evaluating 505⁠(b)⁠(2) products for the U.S. market.

Many pharmaceutical opportunities fail to reach their full potential because their value is not properly assessed. This session will explore the key elements that determine the value of innovation based on known molecules. Practical insights will be provided to support investment decisions, portfolio prioritization, and strategic planning.

Bluepharma is committed to developing differentiated value-added medicines (VAMs) that address unmet medical needs and improve patient care. The company leverages innovative pharmaceutical platforms built on scientific innovation and advanced formulation and dosage form expertise, including the flexibility to work with high-potency molecules.

This session will provide an overview of Bluepharma’s technologies, including oromucosal delivery systems, oral solid dosage forms, and complex sterile formulations. Attendees will gain insight into its proprietary technologies, BlueOS® and BluEase™, and their application in the development of versatile drug delivery solutions designed to enhance patient adherence, optimize bioavailability, reduce side effects, and support tailored therapeutic approaches for diverse patient populations.

The presentation will also highlight Bluepharma’s expertise in fixed-dose combinations, improved solid dosage forms, lipid-based formulations, and long-acting injectable technologies, illustrating how these capabilities contribute to the development of innovative solutions that address healthcare and market needs.

The presentation will explore the key differences between product evaluation pathways in Europe and the US, highlighting the strategic aspects that shape successful Value-Added Medicines and 505⁠(b)⁠(2) projects. It will address how to build a robust business case, including partnerships, project scope, fees, distribution models, and market assumptions, while focusing on what truly creates value for potential partners. The session will also provide insights into competitive landscape assessment and strategic positioning.