World Orphan Drug Congress Europe 2026: Previewing the discussions on evidence, HTA and access

From patient involvement in joint clinical assessments to real-world evidence, study design and access strategies, we preview selected sessions at World Orphan Drug Congress Europe 2026 relevant to the HEOR, RWE and market access communities.

Continuing our coverage of events taking place in October 2026, we turn our attention to World Orphan Drug Congress Europe 2026, taking place in Amsterdam on October 26–28, 2026.

With more than 1500 attendees and 250 speakers expected, the meeting brings together researchers, patient organizations, policymakers and industry to discuss the development, assessment and availability of treatments for rare diseases. A workshop day on October 26 precedes two days of conference sessions and an exhibition.

The program combines plenaries and working groups with eight tracks: Patient Centricity, Access & Pricing, Clinical Development, Real-World Evidence, Investment & Funding, Science & Strategy, Cell & Gene Therapy, and Precision Medicine.

As a media partner for the event, The Evidence Base previews selected sessions of interest to our audience.

EU HTA: Lessons from the first assessments

Unsurprisingly, EU health technology assessment (HTA), particularly Joint Clinical Assessment (JCA), features prominently in this year’s program. With the first phase covering new oncology medicines and advanced therapy medicinal products (ATMPs), including those with orphan designations, the meeting offers a timely opportunity to consider lessons emerging ahead of the broader inclusion of orphan medicines in 2028.

For developers preparing for this expansion, sessions will explore how early assessment experience can inform evidence planning, the role of Joint Scientific Consultations in development strategies, and the implications for national decision-making and patient access.

Another important focus is how patient and caregiver involvement can translate into meaningful influence on assessments. Discussions will consider how their perspectives are represented, how their impact can be demonstrated, and what could strengthen their involvement in assessments of treatments for rare diseases and pediatric conditions.

Selected sessions include:

Invisible patients or visible impact? Examining patient involvement and influence in Europe’s Joint Clinical Assessments (JCAs) of new medicines
Date and time: Tuesday, October 27, 11:30
Speakers: Manca Povsic and Sarah Mohamad (Amiculum)

From evidence gaps to patient access: Lessons for EU JCA readiness
Date and time: Tuesday, October 27, 14:40
Speaker: Emityaz Chowdhury (Parexel)

EU Joint Clinical Assessment for orphan medicines: Insights from the first reports, methodological learnings and implications for evidence generation strategies
Date and time: Tuesday, October 27, 16:10
Confirmed speaker: Louise Hartley (RTI Health Solutions)

Keynote panel: Redefining clinical value: Navigating the scientific and strategic era of EU Joint Clinical Assessments and Joint Scientific Consultations
Date and time: Wednesday, October 28, 09:00
Moderator: Karen Facey (University of Edinburgh)
Speakers: Niklas Hedberg (Swedish Dental and Pharmaceutical Benefits Agency, TLV), Anne Willemsen (Zorginstituut Nederland), Alexander Natz (EUCOPE), Stephanie Said (G-BA) and Tanja Podkonjak (Takeda)

Panel: JCA, the first reports: Implications for patient access for orphan medicines and ATMPs
Date and time: Wednesday, October 28, 13:45
Moderator: Patrick Hopkinson (independent HTA expert)
Speakers: Lorenzo Capretto (Chiesi Farmaceutici), Lydia Shotton (Alliance for Regenerative Medicine) and Peter Pemberton-Ross (Biogen)

Panel: Implications of EU HTA Joint Clinical Assessments to patient engagement practices in rare disease
Date and time: Wednesday, October 28, 13:45
Moderator: Veronica Lopez Gousset (Harvard T.H. Chan School of Public Health)
Speakers: Alexander Natz (EUCOPE), Sheela Upadhyaya (Rarely Ordinary) and Valentina Strammiello (European Patients Forum)

Market access: Pricing pressures and launch decisions

Beyond evidence assessment, the program considers how pricing policy and national access pathways shape the availability of rare disease treatments. US Most-Favored-Nation (MFN) pricing is a particular focus, with sessions examining its potential implications for investment, pricing and launch decisions in Europe and Japan.

Discussions will also examine the UK’s position as a potential first-launch market and the access challenges facing ATMPs in the Netherlands. Alongside these strategic questions, speakers will consider practical ways to address delays between diagnosis and treatment, manage uncertainty when evidence is limited, and improve coordination between regulators, HTA bodies, industry and patient organizations.

Selected sessions include:

Panel: MFN: A regime change for Europe?
Date and time: Tuesday, October 27, 14:10
Moderator: Alexander Natz (EUCOPE)
Speaker: Sarah Pitluck (SP Consulting)

Japan as a test case for US MFN: Implications for orphan drug investment, pricing and launch decisions
Date and time: Tuesday, October 27, 14:10
Speaker: Tosh Nagate (e-Projection K.K.)

Is the Dutch innovative healthcare system falling behind vs other EU countries? ATMP access and HTA challenges
Date and time: Tuesday, October 27, 16:10
Speaker: Angela Borghouts-de Ruijter (Gilead Sciences)

Panel: Is the UK now the smarter first launch market for rare disease therapies?
Date and time: Tuesday, October 27, 16:40
Moderator: Sheela Upadhyaya (Rarely Ordinary)
Speakers: Daniel O’Connor (ABPI), Brad Groves (NICE) and Ben Doak (independent adviser)

Panel: From different perspectives to a shared goal: Improving timely patient access
Date and time: Wednesday, October 28, 13:45
Moderator: Matthias Heck (EUCOPE)
Speakers: Kwee Lan Tan and Iroda Jurabekova (Boehringer Ingelheim), Tina Wang (Center for Innovation in Regulatory Science) and Philipp von Gallwitz (admedicum Patient Driven Solutions)

Evidence generation: From study design to decision-making

With a track dedicated to real-world evidence (RWE), the conference will explore how data can support clinical, regulatory and access decisions in rare diseases. Across this track and the wider program, sessions will consider how study design and evidence planning can address decision-makers’ requirements while reflecting patients’ needs and priorities.

Discussions will examine the design of natural history studies and their potential use as historical controls, alongside practical approaches to improving real-world data (RWD) quality without increasing collection burden. National data infrastructure, patient-centered RWE and the use of AI to support earlier identification of rare diseases also feature. Contributions from the IHI RealiseD project and the HTAi Rare Disease Interest Group will connect developments in evidence generation with approaches to assessment and their implications for patient access.

Selected sessions include:

Considerations for design of natural history studies
Date and time: Tuesday, October 27, 14:10
Speakers: Patricia Murphy, Tanya Binette and Jana Benesh (ICON)

It is what it is (no, it isn’t): Raising the bar without raising the burden
Date and time: Tuesday, October 27, 15:10
Speaker: Pamela Dobay (Rivia)

Panel: Patient centred real-world evidence for rare disease decision-making
Date and time: Tuesday, October 27, 16:10
Moderator: Karen Facey (University of Edinburgh)
Speakers: Elizabeth Vroom (World Duchenne Organization), Maurizio Scarpa (MetabERN), Stephanie Said (G-BA) and Alessio Amadasi (Chiesi Farmaceutici)

Generating regulatory-ready evidence for rare diseases: Insights from the French national rare disease ecosystem
Date and time: Tuesday, October 27, 17:10
Speaker: Solange Roumengous (Banque Nationale Maladies Rares, AP-HP)

Relevance and reality of real-world data generation and utilization in rare diseases
Date and time: Tuesday, October 27, 17:25
Speaker: Tom Pulles (Acadia Pharmaceuticals)

Panel: Improving access through innovation of evidence generation and assessment
Date and time: Wednesday, October 28, 11:15
Moderator: Karen Facey (University of Edinburgh)
Speaker: Alicia Granados (Sanofi)

Beyond AI predictions: Identifying early rare disease patients through real-patient-evidence data
Date and time: Wednesday, October 28, 11:15
Speaker: Arndt Rolfs (subrosa Health & Capital)

Building decision-ready RWE around patient needs and access decisions
Date and time: Wednesday, October 28, 14:15
Speaker: Batyrkhan Kuatov (CSL)