ATMP Horizons at the Health Data Forum Global Hybrid Summit 2025

Advanced therapy medicinal products (ATMPs) are no longer a distant promise. Using genes, cells, or tissues—often as one-time interventions—they aim to correct underlying disease, not just manage symptoms. The session opened with a crisp primer from Dr Hannah Crocker (ATMP Outcomes Programme Manager, NHS Wales Joint Commissioning Committee), setting a shared vocabulary for the rest of the afternoon:

• Gene therapies

  • In vivo: the genetic change happens inside the body (e.g., liver-targeted vectors to correct a faulty gene).

  • Ex vivo: cells are modified in a lab and re-infused (e.g., CAR-T for certain blood cancers).

Spotlight 1 — Individualized medicines at scale (Andrew Roddam, EveryONE Medicines)

How do you scale therapies that may treat a handful—or even one—patient? Dr Andrew Roddam outlined a model for antisense oligonucleotides (ASOs) tailored to ultra-rare, often pediatric, genetic diseases:

• From diagnosis to drug in ~2 years (and shrinking), focusing first on tractable mechanisms (e.g., splice switching, allele-specific knockdown).

• Regulatory evolution is key: the UK's MHRA is advancing pathways for highly personalized medicines; in Europe, platform/"fixed-backbone" marketing authorizations are being explored to reuse prior knowledge across variants.

• Master "basket" protocol: a multinational trial framework that admits different patients, variants, and ASOs under one umbrella—starting at Great Ormond Street Hospital and expanding via partners.

Takeaway: technology isn't the bottleneck; ecosystems are—from regulation and reimbursement to patient-finding and data standards.

Spotlight 2 — Population genomics as ATMP fuel (Tiago Magalhães, M42, Abu Dhabi)

M42's Emirati Genome Program has already sequenced ~750,000 people, pairing long-read and short-read data with 10-year longitudinal EHRs. Dr. Magalhães showed how family structures and founder effects can accelerate discovery and trial-readiness:

• A nationwide scan of inherited retinal disease (IRD) genes revealed high-penetrance variants to prioritize—and a sobering number of zero-penetrance "known" variants that shouldn't drive screening in this population.

• By linking pedigrees, genomes, and records, the team identified novel, likely causal variants (e.g., in ABCA4 and BBS12) across multiple families—shortening routes to precision trials or ATMP referral.

The kicker: population-scale resources serve both ends of the R&D continuum—new discovery and precise trial recruitment.

Panel highlights — Rules, reality, and representation

Joining Roddam and Magalhães, Dr Michelle Lam (MHRA) and Linda Galbraith (patient leader) surfaced the fault lines—and the fixes.

• Regulatory fit-for-purpose: Some modalities (e.g., ASOs, mRNA immunotherapies) don't map cleanly to legacy categories. MHRA is updating classifications and, through a new rare disease consortium, co-designing evidence pathways for "n of few". Global harmonization (ICH/ICMRA dialogues with EMA/FDA) matters for small populations spread across borders.

• Equity and access: ATMPs are expensive, and genomic datasets are uneven globally. Momentum from companion diagnostics could normalize sequencing as standard of care for rare disease—if we invest in patient advocacy, diverse data, and sustainable payment models.

• Patient voice: Godraith reminded us that real-world data (beyond narrow trials) can better reflect lived experience—and that ultra-rare often means ultra-isolated without multinational data networks.

What this means for Wales—and beyond

This session crystallized a playbook any health system can adapt:

1. Start with outcomes: define the measures (clinical + QoL) that matter, then embed capture in care.

2. Invest in the data plumbing: dashboards, data-sharing, TREs—and design for reuse across commissioning, safety, and research.

3. Co-evolve regulation and evidence: align platform approaches (science) with platform authorizations (policy).

4. Make equity a design constraint: recruitment, datasets, and payment models must default to inclusion—or we'll widen the gap ATMPs aim to close.

These principles echo Health Data Forum's broader "Data First, AI Later" doctrine—trustworthy innovation sits on trustworthy data—and the 2025 Wales Summit theme, "Driving Change through Data Insights."

WATCH LIVE – ATMP Horizons (Trends House Session)
Date: Wednesday, 16 October 2025

Slot 1 – Prime Overlap
12:30–13:45 London /Lisbon (UTC+1)
13:30–14:45 CEST (Madrid/Paris/Berlin, UTC+2)
14:30–15:45 Riyadh/Doha (UTC+3)
15:30–16:45 Dubai/Abu Dhabi (UTC+4)
Join live → On LinkedIn here

WATCH LIVE – ATMP Horizons (Trends House Session)
Date: Wednesday, 16 October 2025

Slot 2 – After-Work Middle East / Late Afternoon Europe
16:30–17:45 London/Lisbon (UTC+1)
17:30–18:45 CEST (Madrid/Paris/Berlin, UTC+2)
18:30–19:45 Riyadh/Doha (UTC+3)
19:30–20:45 Dubai/Abu Dhabi (UTC+4)
Join live → On LinkedIn here