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To be announced.
To be announced.
Reserved for NORD.
Sponsored by Sciensus.
Afternoon break before pre-conference sessions resume.
To be announced.
To be announced.
To be announced.
To be announced.
To be announced.
To be announced.
To be announced.
Reserved for supporting partner.
To be announced.
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Expo hall & registration opens
Reserved for Title Sponsor
The pharmaceutical and biotech sectors allocate billions of dollars annually to developing new therapies. However, some assets face discontinuation not due to scientific or medical reasons but due to marketing and strategic priorities. These 'shelved assets' frequently remain underutilized despite their potential medical and commercial value.
This panel brings together Freda Lewis-Hall, former Chief Medical Officer at Pfizer and a renowned expert in drug repositioning, Andrew Lo, an MIT finance professor specializing in innovative financial models, and a policy expert (TBD). Moderated by Annette Bakker, CEO of the Children's Tumor Foundation and a firm advocate for releasing shelved assets for NF, the discussion will focus on crafting frameworks that align medical needs with economic incentives. Together, the panel will present opportunities to transform overlooked assets into lifesaving treatments for patients with low-incidence or low-market diseases.
By addressing this pressing issue, the panel will spotlight actionable solutions to create a win-win scenario—improving healthcare outcomes while driving economic growth in the pharmaceutical and biotech industries globally.
Discussion to focus on immediate outcomes of Rare Disease Innovation Hub as a new model of collaboration between CDER and CBER. While the extent to which this initiative will translate into influencing policy or decision making is unknown, it has the potential significance on the scale of Patient Focused Drug Development 2.0 – representing an opportunity for a systematic approach to prioritize and streamline rare disease drug development. with. Discussion sub-topics:
Networking, poster, and expo break
Sponsored by HAVAS.
We will discuss what technology can bring to healthcare from three perspectives: patient, pharma and healthcare. How AI gives us capabilities to shine a new lens on understanding patients and the personalisation of care. Uncovering its impact on decision-making forall stakeholders.
Sponsored by Caidya.
Sponsored by Ergomed
Sponsored by Premier Research.
Topics to be discussed include:
Sponsored by Bionical Emas.
Sponsored by Ergomed
Sponsored by Sanofi.
Sponsored by Parexel.
Sponsored by Onocross.
Sponsored by WEP Clinical.
Please re-join us as the keynote stage for our lunch keynote panel.
Sponsored by Eversana.
Networking, Poster, & Expo Break.
Sponsored by Alfasigma.
To be announced
Join us for an engaging roundtable discussion hosted by AscellaHealth, where we’ll aim to demystify common misconceptions surrounding exclusive distribution models for specialty pharmaceuticals. Throughout this interactive discussion, participants are invited to share insights and experiences while we explore the real-world impact of exclusive distribution on treatment access and advancing patient-centered care. Together, we’ll discover the truth about how exclusive distribution drives better outcomes for manufacturers, healthcare providers, and, most importantly, patients with complex, chronic and rare diseases—showcasing the crucial role it plays in ensuring that the right therapies are delivered to the right patients at the right time.
This roundtable discussion will look to discuss:
Sponsored by Thermo Fisher Scientific.
This roundtable will discuss key considerations for building integrated evidence at the earliest stages of development to satisfy the needs of multiple stakeholders.
Sponsored by Parexel.
Sponsored by Bionical Emas.
Sponsored by Sanofi.
Roundtables end. Short break before track sessions resume.
This session will explore:
Sponsored by Allucent.
Reserved for supporting partner.
Sponsored by Johnson & Johnson.
Reserved for supporting partner.
Sponsored by Sciensus.
Sponsored by GeneDx.
To be announced.
Please follow signage to the keynote stage.
Reserved for supporting partner.
The rare disease community has seen an increased uptick in collaboration between scientists around the world to find solutions for rare disease patients. The panel for this unique fireside chat will discuss the perspective of a rare disease caregiver who has a daughter with ARSACS and her intensive search to find a treatment or cure. After more than twenty years of seeking a diagnosis, she stumbled upon the work of world renowned neurologist, Dr. Jeremy Schmahmann of Massachusetts General Hospital, and he diagnosed her daughter during a tele-visit in 2020. Subsequently, the two have partnered together with the ARSACS Foundation, based in Montreal Canada, to embark upon various research protocols, fundraising initiatives, and collaborative efforts to rapidly advance science forward for this rare disease.
"Driving the Future of Rare Disease R&D: A Multi-Stakeholder Perspective" brings together a diverse group of experts to discuss the evolving landscape of research and development in rare diseases. Featuring insights from pharmaceutical companies, healthcare providers, patient advocacy groups, and policymakers, the panel will explore the challenges and opportunities in accelerating breakthroughs for rare diseases. Key topics will include the role of innovation, patient-centered approaches, regulatory frameworks, and collaborative efforts across sectors. The discussion will highlight how multi-stakeholder engagement can drive more efficient, targeted solutions to meet the unique needs of rare disease patients, while addressing the financial, logistical, and ethical complexities of rare disease research.
In this panel discussion, experts will share best practices for successfully commercializing orphan drugs and scaling them to global markets. The session will cover the entire lifecycle of orphan drug development, from initial launch strategies to navigating regulatory hurdles and building capacity in international markets. Panelists will explore key challenges such as pricing and reimbursement, market access, and distribution, while also highlighting successful case studies and lessons learned. Attendees will gain practical insights into creating sustainable commercialization strategies that ensure access to life-changing therapies for rare disease patients worldwide.
Reserved for Alexion Pharmaceuticals.
In this panel, patient advocates, caregivers, and community leaders will discussthe special sauce. This panel will leave you with actionable, tangible take-aways to improve therapeutic development, strengthen pipelines, build patient-driven company cultures, impact patients in need of treatment, and efficiently meet business goals through authentic and powerful patient partnerships.
This stand out panel will be an open dialogue with ideas to integrate the patient community into biotech process, and how new advocacy organizations can galvanize as a community to de-risk the orphan drug development process… IF we take this beyond the panel and into the process. Today.
With the tremendous progress in recent years of genetic screening and more sophisticated clinical diagnosis and biomarker approaches, broader populations of patients who share common pathologies and/or molecularetiologies are being stratified into subpopulations of orphan and rare diseases. This has enabled precision medicine approaches that, for example, can selectively target the underlying genetic driver of a disease. Moreover, in the past, a popular drug development strategy has been to test novel therapies in a subpopulation before expanding into the larger disease population. This expert panel will offer diverse perspectives on indication selection considerations and challenges for novel orphan drugs that have broad therapeutic potential for several rare disease patient subpopulations, such as clinical developmentstrategies,regulatory landscape, patient accessibility and the impact of the IRA on commercial models.
Unlocking the Future: The Convergence of AI, Personalized Medicine, and Genomics
AI and genomics are revolutionizing healthcare, accelerating drug discovery, optimizing treatments, and transforming patient outcomes. But how do we bridge the gap between innovation and real-world implementation? Join leading experts as they explore how AI-powered insights and multi-omics are driving precision medicine forward—while tackling critical challenges in regulation, access, and adoption. Don’t miss this dynamic discussion on the breakthroughs shaping the future of healthcare!
Experienced panelists will share their experiences in designing, operating and closing out EAPS, with an in depth look at the success and failures encountered along the way. To be moderated by an expert EAP Ethicist that can provide additional context and situational learnings from her experience supporting a vast number of small and large pharmaceutical companies running EAPs.
In this session, we will evaluate the investment potential within the rare disease and orphan drug markets, exploring the unique opportunities and challenges these sectors present. Attendees will gain insights into the growing demand for innovative treatments for underserved patient populations and the financial implications of developing therapies for rare conditions. We will also discuss the regulatory landscape, funding trends, and key factors influencing investment decisions. By examining the current investment climate and forecasting future trends, this session aims to provide a comprehensive understanding of how to navigate and capitalize on the evolving rare disease and orphan drug landscape.
The panel discussion will center around the increasing number of Gene and cellular therapies that are entering clinical trials. Specifically in rare diseases the number of eligible patients is highlighted as a rate limiting issue for execution of the trial. While this is true, the following other issues will be discussed.
Rare diseases, particularly ultra rare, require special considerations during clinical development. The Rare Hub has been a long awaited answer to the question "How do we improve the process for all stakeholders in developing therapies for the rare community". Spend time withlong time rare patient advocate Jenn McNary andAmy Comstock Rick, Director of the Rare Hub, moderated by Susan Winckler, CEO of Reagan-Udall and as they discuss near and long term goals and activities of the Hub. Learn how all stakeholders can continue to weigh in on Hub priorities and what we can expect over the coming year.
Sponsored by Somebody to Talk To.
To be announced
This concludes Day 1 of the congress. Please join us in the Expo Hall for our cocktail reception.
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Expo hall & registration opens
Sponsored by Johnson & Johnson.
In this illuminating keynote panel, we will explore real experiences of navigating the regulatory and reimbursement framework at an EU and global level to bring innovative therapies to people with rare conditions. Leading stakeholders will share their insights on creating sustainable and effective models that ensure equitable access to life-saving therapies. Join us for this engaging session as we seek to redefine the future of orphan drug development and delivery, focusing on sustainability, equity, and innovation in the quest for better outcomes for patients with rare diseases.
Networking, poster, & expo hall break
To be announced.
In this panel discussion, experts will share perspective best strategies for optimizing market access, pricing and reimbursement of orphan drugs. The session will cover considerations from early-stage drug development through commercial launch. Panelists will explore key challenges to developing and executing a multi-stakeholder strategy across multiple orphan therapeutic areas. Attendees will gain insights onto creating value-added market access and reimbursement strategies for investor and strategic stakeholders that bolster access to novel, life-changing therapies while ensuring corporate viability.
Sponsored by Genome Medical.
This presentation showcases innovative methods validated against the newly assigned ICD-10 code for Activated PI3K Delta Syndrome.
Sponsored by EVERSANA.
Sponsored by Volv.
Sponsored by Lumanity.
Sponsored by 3H Medi Solution Inc.
Reserved for supporting partner.
Reserved for supprting partner.
Sponsored by Johnson & Johnson.
This session will examine the critical role of early-stage collaboration between advocacy groups and industry stakeholders in the development of orphan drugs and treatments for rare diseases. With the unique challenges posed by these conditions—including small patient populations, high development costs, and regulatory complexities—early engagement between patient advocates and pharmaceutical companies is essential to drive innovation, expedite regulatory pathways, and improve access to life-saving treatments.
Key topics will include the role of early dialogue in shaping clinical trials, the impact of advocacy efforts on regulatory approvals, and strategies for addressing the financial and logistical barriers that often impede progress in rare disease treatments. Participants will leave with practical strategies for fostering effective partnerships and lessons on how to leverage collaboration for meaningful impact in the orphan drug space.
Reserved for supporting partner.
Reserved for Sanofi.
Sponsored by 3H Medi Solution Inc.
Reserved for supporting partner.
This panel will be focused raising awarness about adult onset, genetic, neurodegenerative diseases. We will discuss roadblocks leading to delayed diagnosis and areas of opportunity to change this pattern with a focus on genetic testing and differential diagnosis. We will highlight and explore the critical importance of recognizing the unique challenges for those impacted by diseases that hit adults in the prime of their lives and specifically those with limited or no treatment options that may lead to cognitive decline and dementia. We hope this panel will encourage further discussion resulting in a tighter bond, strength in numbers, louder voice in the rare community and a strengthening coalition and call to action.
Navigating the regulatory landscape is one of the most complex and critical aspects of bringing new therapies to market, especially in the context of innovative treatments, rare diseases, and cutting-edge technologies. This panel will delve into strategies for finding the optimal regulatory pathway to bring life-saving drugs and biologics to market faster and more efficiently, while maintaining the highest standards of safety and efficacy.
The discussion will explore the evolving regulatory frameworks, from accelerated approval programs to adaptive trial designs and breakthrough therapy designations, and how these pathways can be leveraged to speed up the development of groundbreaking treatments. Panelists, including regulatory experts, industry leaders, and policymakers, will share insights on how regulatory agencies are evolving to meet the challenges of rare drug development, and how companies can navigate these changes to achieve faster approvals.
Effective patient engagement is essential for the success of any healthcare innovation, but smaller biotechs, providers, and payers often face unique challenges in building meaningful, lasting relationships with patients. This panel will explore the practical strategies and tools these stakeholders can use to directly engage patients, gather insights, and ensure that their needs and preferences are integrated into the development, delivery, and reimbursement of orphan drugs.
Panelists will share real-world examples and actionable advice on how smaller companies and organizations can break through the noise and create a patient-centered approach that resonates with the communities they serve. Discussion will focus on building trust, ensuring transparency, and fostering collaboration with patient advocacy groups to drive better outcomes for patients, while also meeting business and regulatory objectives.
Networking, poster, & expo hall break
Short break before track sessions resume
To be announced.
WHO speaker to be announced.
Panelists to be announced.
In the journey of living with a rare disease, the effects often extend far beyond the individual patient, creating a profound impact on the entire family unit. This compelling talk will explore the emotional and psychological trauma experienced by families navigating the complexities of rare illness. Using the metaphor of a waterbed, I will illustrate how the ripples of trauma can shape family dynamics, relationships, and overall well-being. Join us to understand the intricate interplay of trauma and healing and discover ways to create a nurturing environment that empowers both patients and their families on this challenging journey.
Industry partners recognize the need to learn from patient and caregiver's stories of their lived experience. Patient and caregiver stories are the heart of rare disease insights. Patients and caregivers have created authentic, compelling media - from blogs to podcasts to films - offering an invaluable window into the rare disease experience. Tapping into the emerging and increasingly robust media environment specific to rare disease stories can be a first step for patient engagement. Storytelling isn't just advocacy and it can be a key asset to industry supporting and developing meaningful relationships with these creators - who have the pulse of the community. Publicly available and searchable sources of rare disease patient stories represent an under-utilized opportunity for industry. The panel will outline ways industry can make use of these media outlets as part of their social listening and pre-engagement plans to set themselves up for more meaningful connection, and authentic engagement.
Sponsored by RTI Health Solutions.
Reserved for supporting partner.
Reserved for supporting partner.
To be announced
With new and innovative treatments, better clinical and research options and emerging registry options, newborn screening has become an important discussion topic in the rare disease community. This multi-stakeholder panel will talk through the current state and federal landscape of NBS, and then turn to projections on the initiatives that could shape the future of NBS. Hear from patient advocates on the benefits of early screening and diagnosis, and from an industry partner about the work being done to support the addition of conditions to federal and state testing panels.
Sponsored by RTI Health Solutions.
As the healthcare landscape becomes more global and inclusive, ensuring diversity in clinical trials and product planning is crucial for developing orphan drugs that are safe, effective, and accessible to all populations. This panel will address the importance of incorporating diverse patient populations in clinical trials and how such efforts impact product development, regulatory approval, and commercialization—particularly in later-stage drug development.
Panelists will explore how diverse representation in clinical trials—including age, gender, race, ethnicity, and socio-economic status—can lead to more reliable data, better health outcomes, and ultimately, broader market access. The discussion will also highlight how inclusive product planning and real-world evidence collection play a critical role in supporting market entry and improving commercialization strategies, especially for products targeting underserved or minority communities.
Other speakers to be announced.
"Rare Leadership: Women Redefining Healthcare," assembles four distinguished leaders from the Rare Women podcast,which launched January 11th, 2025. Each one pioneers change to improve healthcare and address critical rare disease and biopharma needs. They are united by a commitment to innovative therapies, patient-centered care, and sustainable healthcare solutions. Their backgrounds are diverse, with expertise in healthcare, biopharma, and biotech. They have unique perspectives on resolving rare disease challenges and improving healthcare for underserved communities. Their personal stories and professional insights will inspire others to shape the healthcare of tomorrow.
We’ll discuss:
Overcoming barriers in the healthcare industry
● Creating impactful solutions for rare diseases
● AI’s usefulness in healthcare innovation
● Strategies to drive meaningful change in traditionally male-dominated fields
● The power of collaboration in healthcare innovation
As the EU Health Technology Assessment (HTA) Regulation reshapes the way health technologies are evaluated across Europe, it brings both new opportunities and challenges for patient involvement in the decision-making process. This regulation emphasizes the importance of integrating patient perspectives into the assessment of treatments, which raises key questions about what successful patient involvement looks like. Effective engagement means ensuring that patients’ lived experiences, needs, and priorities are not only heard but meaningfully influence outcomes. A successful model of involvement goes beyond consultation to co-creation, where patients actively contribute to the design and interpretation of assessments. However, the roles of individual patients and patient organizations in this process differ. Individual patients bring personal insights and lived experience, often providing powerful testimony about the real-world impact of treatments. In contrast, patient organizations represent a broader community of patients, advocating for collective interests and ensuring that diverse patient voices are considered. This panel will explore these dynamics, discussing how individual and organizational contributions can complement each other to create a more patient-centered approach to HTA, and the implications this holds for the future of healthcare policy and practice across Europe.