This report distils the patient-relevant content of the EHA 2026 sessions into plain language for patient advocates. Each session opens with a short summary of what was presented, followed by a Key Messages for Patient Advocates box highlighting the points most useful for advocacy, patient communication, and engagement with regulators, researchers and industry.

Session 1 · One year of EU HTA Regulation: delivering for patients and clinicians?

A joint EHA–Patient symposium marking the first year of the EU Health Technology Assessment Regulation (HTAR). Under this regulation, EU countries now carry out a single Joint Clinical Assessment (JCA) of a new medicine’s clinical evidence, instead of each country repeating the work. Patients, carers and clinicians can feed into these assessments. The session brought together the patient, rare-disease, clinician, industry and national-authority viewpoints on how the first year has gone.

Introduction and patient’s perspective

Jan Mol, Lymphoma Coalition (Houten, The Netherlands)

Patients still hit three main barriers to getting new medicines: rules that differ from country to country and slow approval; high prices and restrictive reimbursement; and supply-chain shortages. The new EU HTA system is meant to help by having countries assess the clinical evidence together.

One year in, 16 Joint Clinical Assessments have been started (15 ongoing, 1 stopped) and 7 Joint Scientific Consultations (early advice to developers) launched. Patients and carers made up a large share of the experts involved — 41% of contributors to clinical assessments and 50% of contributors to scientific consultations.

Key messages for patient advocates Patient involvement in HTA is now built into the EU process, and patients are turning up in real numbers — but the job is to make sure that input actually shapes decisions, not just fills a seat.What patients want is consistent and clear: the best available treatments, real effectiveness at an affordable price, faster access, transparency of the data, and outcomes that reflect quality of life — captured through patient-reported outcomes and patient experience data.New pricing approaches (pay-for-performance, risk-sharing) are part of the patient ask, not just faster approvals.

The rare-disease patient’s perspective

François Houÿez, EURORDIS (France)

EURORDIS helps find and prepare patient experts for assessments. Of 29 procedures so far, 21 involved rare diseases or rare cancers; EURORDIS was involved in 18 and contacted 135 patients — but for 6 procedures no suitable patient could be found, showing how thin the patient pool can be in rare conditions.

The positives after year one: countries are genuinely sharing the work (15 HTA bodies actively assessing), the first assessment report was delivered on time, and countries are adapting their national methods — including patient involvement. The concerns centre on transparency: who sits on the expert subgroups is kept secret, experts contribute only remotely and in writing, and it is unclear whether their input is actually used. For roughly half of countries, the joint report will not contain the economic information they need, so their national process may barely change.

Key messages for patient advocates Advocates should push hardest on transparency: secret expert lists and closed subgroups make it impossible to know whether patient input counted.Watch the legal 180-day deadline for decisions (set by the Transparency Directive) — faster, on-time decisions are a concrete, measurable win to hold the system to.Capacity-building for patient experts must keep being funded (e.g. EUCAPA), because the process is still on a learning curve and rare-disease patient experts are scarce.The bigger question is whether this cooperation grows into a genuine EU HTA agency — advocates have a stake in pushing countries to declare their appetite for more and better cooperation.

The doctor’s perspective

Bernhard Wörmann, DGHO & Charité

Access to new drugs across Europe was very uneven in 2015–2025, and that has to change. Where no dossier is submitted in a country, doctors have increasingly resorted to off-label use to treat patients. The hope is that a more effective EU HTA reduces the incentive for companies to launch only in the most profitable markets.

Key messages for patient advocates Uneven access is not abstract — it pushes clinicians toward off-label prescribing when medicines aren’t formally available, which carries its own risks for patients.The doctor’s core point for advocates: regulatory approval (EMA) and real-world access (HTA, pricing, market decisions) are two different things, and a medicine being approved does not mean patients can actually get it.

An industry perspective

Tanja Podkonjak, EFPIA (Switzerland)

Preparing for the centralised EU process has meant major internal change for companies — new roles, new capabilities, and planning for the assessment as early as the trial-design stage rather than after results are in. A key sticking point is the PICO: the framework (Population, Intervention, Comparator, Outcome) that defines what question the assessment answers. A simulation of three assessments produced anywhere from 6 to 52 PICOs, because clinical practice varies so much across countries and there was no clear way to consolidate them.

Companies report improvements but also a heavy increased burden — aligning EU HTA with FDA and EMA timelines at the same time, tight deadlines, and little dialogue with assessors, so submissions rely on best guesses. About a third of developers are small companies or academic groups that have never even submitted an EMA dossier, and half of assessments are in rare diseases where patients and data are limited.

Key messages for patient advocates A ballooning number of PICOs (up to 52) risks reports full of thin, fragmented evidence rated as ‘weak’ — which helps no one, least of all patients waiting for a decision.Industry and advocates actually align on several asks: meaningful stakeholder involvement (right now often just one patient and one clinician per assessment), ongoing dialogue with assessors during the process, and reports that capture context — not only statistics.The shared goal advocates can champion: make the Joint Clinical Assessment an enabler of innovation in Europe, not a new barrier, measured by faster patient access and quicker decisions.

The national authority perspective

Niklas Hedberg, TLV, Co-Chair of the Member State HTA Coordination Group (Sweden)

This talk laid out who does what: the EMA licenses medicines centrally; the EU HTA Regulation provides a shared clinical assessment; and each country still decides on price and reimbursement independently. The HTA Stakeholder Network deliberately balances one-third patients, one-third industry and one-third clinicians. The first joint report — on tovorafenib (Ojemda) — was published on 9 June 2026.

Sweden’s agency (TLV) has restructured to take part: new confidentiality rules, updated guidance for developers, a dedicated HTA office, and roles as assessor or co-assessor on three assessments. Notably, Sweden involves patients in developing its national PICO. The expectation is more work up front but reduced workload long-term, and less dependence on FDA requirements.

Key messages for patient advocates The critical thing to remember: the joint clinical assessment does NOT decide price or reimbursement — those stay national. Access still depends on each country’s own decision.Sweden’s practice of involving patients in shaping the national PICO is a model advocates can point to and ask other countries to copy.A live challenge for the whole system: how do we actually measure whether EU HTA is improving patient access? Advocates should press for that impact to be tracked and published.

Session 2 · How Patient Experience Data (PED) can inform the approval of new medicines

Patient Experience Data (PED) is systematic information about what matters most to patients — symptoms, the impact on daily life, side-effects and treatment burden, patient preferences, and how much risk patients are willing to accept. This session examined why that data is essential to drug approval, why it is still under-used, and how patients, academics, industry and regulators can each strengthen it.

From surviving to living: why PED must shape acute leukaemia drug approval

Samantha Nier, ALAN (Acute Leukemia Advocates Network)

PED captures what living with the disease is actually like — reported toxicities, patient-reported outcomes and patient preferences. Regulators such as the FDA and EMA increasingly value patient input, but in practice it is often gathered too late, can be anecdotal, and the outcomes measured may not reflect patients’ daily reality.

Key messages for patient advocates The core warning: without systematically collecting PED, we risk approving medicines that extend life but ignore how that life is actually lived.’From surviving to living’ is the frame — advocates should insist patient experience is built in from the start of development, not bolted on at the end.

Incorporating patient preferences into academic developments

Julio Delgado (Barcelona, Spain)

Even skilled clinicians can miss patient preferences, because patients may feel intimidated by doctors or fear that voicing their true wishes could exclude them from a therapy. The speaker suggested advanced-practice nurses can help bridge that gap, and argued there is no valid excuse to leave patient preferences out of academic research: if patients are hard to find, try harder; if they don’t understand, communicate better.

His ‘wish-list’ example: a trial where patient-reported outcomes are the primary endpoint, the experimental drug wins because patients prefer it, and survival is simply comparable — reversing the usual hierarchy that puts survival first and quality of life last. A real example (var-cel in relapsed/refractory ALL) showed global health scores rising from 1.92 at screening to 5.52 at 12 months; after consulting ALAN, the trial switched to questionnaires patients preferred and that regulators recommend.

Key messages for patient advocates Patient advocates can and should sit on trial steering committees, help design trials, and help interpret results — not just be consulted afterwards.Advocacy input can directly change which quality-of-life questionnaires a trial uses, making the data more meaningful and more likely to be accepted by regulators.Ask for regulator support to make patient-preference endpoints count — the barriers are practical, not principled.

How can PRO data collection lead to informative evidence?

Maria Teixeira, EORTC (Brussels, Belgium)

Patients sometimes doubt whether patient-reported outcome (PRO) questionnaires are worth it — they can be long and burdensome, and patients often don’t feel the data changes their care. Yet everyone agrees collecting it matters. The problem is a disconnect between generating data and actually using it.

The numbers show the gap: across 2021–2025 oncology approvals, PROs were the primary endpoint in only 0.6% of cases (1 study). Only 3 (1.8%) resulted in a patient-reported outcome claim on the product label. Known limitations — data quality, weak endpoint definitions, analysis and missing data — keep blocking PRO findings from reaching the label. The MOMENTUM study in myelofibrosis was shown as a positive example, where PRO measures demonstrated meaningful improvements in symptoms like fatigue that matched patient priorities.

Key messages for patient advocates More PRO data is not the goal — better, informative PRO evidence that can genuinely change decisions is. Weakness at any single step (objective, design, analysis, interpretation, reporting) undermines the whole thing.This is a shared responsibility: patients advocate for data that matters, academia protects the methods, industry designs trials that generate meaningful data, and regulators integrate it into decisions.Fatigue in myelofibrosis is a concrete example of an unmet need that only shows up clearly when you measure what patients actually experience.

Integrating PED into haematology and oncology drug development: the US FDA perspective

Margret Merino (FDA, United States)

The FDA’s 2024 guidance recommends a core set of patient-reported outcomes in cancer trials: disease-related symptoms, symptomatic side-effects, an overall side-effect impact measure, and physical and role function. These can and should be measured throughout development — not just in late trials. The FDA’s ‘Project Patient Voice’ is an online platform giving patients, carers and clinicians direct access to patient-reported symptom data from cancer trials.

The belantamab mafodotin case (a myeloma antibody-drug conjugate) showed why this matters: over 90% of patients experienced some eye toxicity and more than 75% had severe (grade 3–4) events. Patient-reported tools captured how this hit daily life — for example, difficulty driving at night rose from 9% of patients at baseline to 47% at week 13 in the treatment arm. That patient-reported evidence made it onto the US label.

Key messages for patient advocates Patients’ own reports of tolerability can be captured in every oncology trial, including the earliest dose-finding stages — advocates can ask for this as standard.’Project Patient Voice’ is a real transparency tool advocates can point patients and clinicians toward to see trial symptom data directly.When side-effects are severe (as with eye toxicity here), patient-reported data is what translates a clinical grade into something patients recognise — like no longer being able to drive at night — and can shape the official label.

The EU regulatory position on using PED in medicines development and evaluation

Juan García Burgos (EMA)

The EMA’s message: PED is essential, not optional, for patient-centred regulation. Its reflection paper offers a general framework (not step-by-step methodology) to encourage systematic use of PED across the medicine lifecycle, and complements international (ICH) work. PED should be built in early, with developers encouraged to talk to the EMA through scientific advice.

A public consultation (Sept 2025–Jan 2026) drew strong support from 112 stakeholders — patient organisations were the largest group (39), ahead of industry (37). Feedback called for shared principles, harmonised terminology, and practical guidance, and asked the paper to state even more strongly that PED is a core part of patient-centred regulation.

Key messages for patient advocates The EMA has publicly framed PED as essential — advocates now have an official statement to hold regulators and developers to.Patient organisations were the single biggest voice in the consultation, showing the sector’s collective input carries real weight.PED is especially important in cancer, where quality of life may matter more to patients than established endpoints like overall survival — and where patient-reported outcomes can inform decisions when ‘harder’ survival data isn’t yet mature.

From patient experience to regulatory action

Susan Frade (Novartis)

PED is increasingly seen as essential to align medicines with real-world patient needs. The proposed approach threads PED through the whole development lifecycle — from choosing candidate drugs to launch — with three pillars: a development strategy informed by patient needs, clinical study design reflecting diverse patient perspectives, and regulatory/value dossiers that incorporate PED. Real examples were cited, including patient input factoring into 46% of FDA 2025 approvals and the EMA’s use of PED in benefit-risk assessments.

Key messages for patient advocates Patient input is already influencing a large share of approvals — the direction of travel is set; the task is consistency.The call to action is collaboration: industry, regulators and patient groups working together so patient voices consistently shape development and approval — not occasionally.

Rethinking clinical trials and treatment decisions

Day 2 turned to how trials are designed and how treatment choices are made — from new statistical thinking, to using biology and disease-response to tailor leukaemia treatment, to two live debates on the standard of care.

How to design, conduct and analyse clinical trials in the future

Jérôme Lambert (France)

The ‘old thinking’ was a fixed, two-arm randomised trial with one outcome and one final analysis, designed above all to protect against a false positive — appropriate when diseases were common, treatments scarce and patients not split into molecular subgroups. That context has changed. ‘New thinking’ includes adaptive and platform trials, pragmatic trials, external/historical control arms, and modern statistics (Bayesian methods, indirect comparisons, surrogate outcomes).

The caution: not all new methods are equally good. Simple-but-wrong approaches can mislead; some newer techniques (like generating a control arm from historical data) add no real information, while others work only with complex methods to compensate for data that isn’t shared. The guiding principle stays ‘first, do no harm’ — give patients the most effective, safe treatment at the highest level of evidence.

Key messages for patient advocates Newer, faster trial designs are coming and can speed access — but advocates should ask whether a novel method genuinely adds reliable evidence or just looks modern.The patient-protection purpose of trials hasn’t changed, even as the tools do; ‘faster’ must not mean ‘less certain that it actually helps.’

How to deliver biologically informed treatment decisions in acute leukaemia

Anthony Moorman (Newcastle upon Tyne, United Kingdom)

Survival for children and young people with acute lymphoblastic leukaemia (ALL) has improved dramatically across successive UK trials. The next step is precision: using the biology of each patient’s leukaemia plus how well it responds to early treatment to decide who needs more intensive treatment and who is being over-treated.

Two tools drive this. Measurable residual disease (MRD) — how much leukaemia remains after initial treatment — sorts patients into favourable, intermediate and high risk. Genetic subtype adds another layer (some genetic changes carry good prognosis, others poor). Combining response and genetics lets teams reduce treatment for low-risk patients and escalate (targeted therapy, CAR-T, transplant) for high-risk ones. Future challenges include newer therapies like blinatumomab, toxicity-free survival, and AI.

Key messages for patient advocates ‘Biologically informed’ care means some patients can safely have LESS treatment — sparing them toxicity — while others get more. Advocates can help patients understand that de-escalation, when evidence-based, is a benefit, not a shortcut.MRD and genetic testing are central to modern risk-based treatment; access to good-quality testing is itself an access-and-equity issue worth advocating for.A meaningful emerging goal is ‘severe toxicity-free survival’ — living longer AND better — which aligns closely with what patients say matters.

Debate · Is HMA-Venetoclax the new standard of care for induction in fit older AML patients?

YES: Paresh Vyas (Oxford, UK)   ·   NO: Christoph Röllig (Dresden, Germany)

The question: for older-but-fit patients with acute myeloid leukaemia (AML), should first treatment be the gentler azacitidine-plus-venetoclax combination (Aza/Ven) or traditional intensive chemotherapy (IC)? The PARADIGM trial (172 patients, median age 65) found Aza/Ven roughly doubled event-free survival (14.6 vs 6.15 months) and let more patients reach a stem-cell transplant (61% vs 40%), with similar overall survival between the arms.

The YES case: most older fit patients (about 87%) still do poorly on intensive chemo and would benefit from transplant; Aza/Ven has lower early mortality and better quality of life, helping patients reach transplant fitter. The NO case: intensive induction has cured thousands of patients over decades, and for specific groups — such as core-binding-factor AML and younger patients with an NPM1 mutation, both of which PARADIGM excluded — response rates with HMA-Ven are clearly worse (69% vs 94% complete response). Practice, for now: enrol in a trial if possible; otherwise Aza/Ven for most who’ll go to transplant, but intensive chemo for defined subgroups.

Key messages for patient advocates For older fit AML patients, a gentler induction that gets people to transplant in better shape is a genuine, patient-relevant option — lower early mortality and better quality of life matter as much as the survival curve.One size does not fit all: specific genetic subgroups still do better with intensive chemotherapy, so advocates should stress that treatment choice must be individualised to the patient’s biology.PARADIGM ‘informs but is not practice-changing’ — advocates should be wary of over-claiming from a single trial, and support continued trial enrolment in a fast-moving field.

Debate · Do we need stem-cell transplant in first-line Ph+ ALL?

YES: Adele Fielding (York, UK)   ·   NO: Sabina Chiaretti (Rome, Italy)

A debate on whether patients with Philadelphia-chromosome-positive ALL still need an allogeneic stem-cell transplant as part of their first treatment, now that targeted therapies have improved. (Presented as opposing viewpoints.)

Key messages for patient advocates The very fact this is now debated is good news for patients: better targeted drugs are raising the question of whether some patients can be spared a demanding transplant and its long-term risks.Advocates can watch this space closely — the answer will directly affect treatment burden and long-term quality of life for Ph+ ALL patients.

EHA–EMA–FDA Session on Global Trials

A joint session with both European and US regulators on why medicine research is increasingly global, what that means for whether trial results apply to your patients, and how to make sure patients everywhere are genuinely represented — not just recruited.

Global clinical trials: a necessity for progress and a priority for EHA

Tarec El-Galaly (Aarhus, Denmark)

Research is increasingly happening outside the US and Europe. Europe still takes part in global research but no longer reliably leads it, held back by complex regulation, high costs, restrictive post-approval access policies, and weak incentives for academic innovation. Global trials matter because progress can come from anywhere, disease biology and patient characteristics differ across regions, and haematological diseases are increasingly rare (thanks to finer molecular subtypes) — so recruiting enough patients often requires going global.

EHA is actively pushing for better, more predictable regulation — including through an EHA-led Coalition for Reducing Bureaucracy in Clinical Trials — so Europe stays an attractive place to run high-quality, patient-centred research.

Key messages for patient advocates Global trials are how patients get access to innovation and how rare-disease trials become possible at all — but clinicians need confidence that the benefit and risk seen in a trial actually apply to their own patients.Europe’s competitiveness for trials is a patient-access issue: if trials leave Europe, so can early access to new treatments. Advocates have a direct interest in simpler, more predictable EU trial regulation.

The patient perspective on global trials

Angelo Loris Brunetta (Genova, Italy) — Vice-Chair, EHA Patient Advocacy Committee

The central argument: opening recruitment worldwide is not the same as achieving meaningful representation, and one patient perspective is not enough. Patients with the same diagnosis can have very different realities depending on their healthcare system, culture, access to treatment and finances. A treatment developed globally must also be designed globally — with patients at the table from the start.

Investigators ask about response, efficacy, safety and durability; patients ask different questions — will I still need treatment, can I work or study, how often must I travel, will this affect fertility, who pays for follow-up, can my local doctor handle complications? Priorities also differ by region: higher-resource settings may value convenience, fertility and preserved cognition, while lower-resource settings prioritise basic access, affordability, travel burden and treatment continuity. And trials still cluster in a few countries with strong research infrastructure — often not where disease burden is highest. Of 32 globally approved gene therapies in 2025, only 10 were approved outside high-income countries.

Key messages for patient advocates This is the advocacy centrepiece of the congress: patients are partners, not just participants — they should shape endpoints, visit schedules, consent design, acceptable risk trade-offs and trial locations, from the beginning, not review protocols after they’re written.Engage patients early and ‘not to check a box’; partner with experienced patient organisations from multiple regions so the real diversity of patient experience is captured.Global representation is about lived experience, not just geography. If trials skip the regions with the greatest disease burden, innovation risks becoming a privilege of the few.Measuring outcomes that don’t matter to patients produces therapies that improve numbers without meeting real needs — advocates should insist on culturally appropriate quality-of-life and patient-reported measures.

Global trials: perspective from an academic research group

Michael Fuchs, German Hodgkin Study Group (Cologne, Germany)

Academic groups like the German Hodgkin Study Group have run first-line trials since 1978, enrolling over 22,000 patients across thousands of sites — trials aimed at optimising therapy rather than winning marketing approval, yet still capable of changing the standard of care and even influencing drug approval and reimbursement. But they face real hurdles: securing the study drug, coordinating across EU and non-EU countries, contracts, and funding that (from public sources) usually doesn’t cover the full cost.

Key messages for patient advocates Academic, non-commercial trials often ask the questions that matter most to patients — like whether treatment can be safely reduced — but they are chronically under-funded. Advocates can champion sustained public funding and access to study drugs for these trials.Data from these investigator-led trials can increasingly support approvals and reimbursement decisions, so protecting them protects patient access to well-studied, optimised treatment.

Global trials: some regulatory considerations

Filip Josephson (Sweden)

International guidelines (ICH E5 and E17) govern multi-regional trials. They distinguish ‘intrinsic’ factors — genetics and disease biology that affect how a drug is handled by the body and how well it works — from ‘extrinsic’ factors — differences in local healthcare practice, diagnosis, monitoring, supportive care, follow-on therapies, how symptoms get reported, and patients’ willingness to stay on treatment despite side-effects. Both can change the measured treatment effect.

For a drug that works by a well-understood mechanism, regulators are fairly comfortable accepting foreign data (some cancer drugs have been approved in the EU based on China-only studies). For genuinely new mechanisms, more caution is needed. When results differ between regions, the key question is whether that difference can be explained and understood — not just noted.

Key messages for patient advocates Where a trial is run genuinely affects whether its results apply to a given patient — this is a scientific reason patient diversity in trials matters, not just a fairness argument.Differences in supportive care and follow-on treatment between regions can change a trial’s apparent results — a reminder that patient access to good overall care shapes outcomes, not just the study drug.

Multiregional trials in oncology: the FDA experience (STARGLO case)

Margret Merino (FDA, United States)

Multiregional trials speed recruitment and can support use across many countries, but they must satisfy multiple regulators and produce results that apply to each local population. The STARGLO trial (glofitamab plus chemotherapy in relapsed/refractory diffuse large B-cell lymphoma) became a cautionary example: much of its benefit came from the Asian subgroup, whose patients differed from the intended US population (younger, more early-relapse disease, most had refused transplant, different available follow-on therapies).

The FDA’s advisory committee voted that the results were not applicable to the proposed US population, and the FDA did not grant a new indication — an additional trial was required.

Key messages for patient advocates A trial can be positive overall yet still be turned down by a regulator if the results don’t clearly apply to that country’s patients — approval in one region does not guarantee it elsewhere.For patients, this underlines why trials must enrol enough people who reflect the population that will actually use the drug — and why advocates should ask about representation early in trial design.

FDA regulatory framework and design considerations for multiregional trials

Kelly Norsworthy (FDA, Division of Hematologic Malignancies, United States)

The FDA’s paramount question is whether results apply to US patients and US standard of care. That means control arms must reflect the real standard of care in each region (no outdated comparators), enough US patients must be enrolled, and site distribution should be planned in advance. A Diversity Action Plan is now required for pivotal studies. For cancers common in the US, the FDA recommends equal allocation across regions; for less common cancers, allocation proportional to disease prevalence.

Key messages for patient advocates Diversity Action Plans are now a formal US requirement for pivotal trials — a concrete lever advocates can reference when pushing for representative enrolment.Control arms must match the current best standard of care in each region — advocates can ask whether trial participants are being compared against genuinely up-to-date treatment, not an outdated one.The FDA explicitly names patient-perspective input and patient collaboration as opportunities in trial design — an open door for advocacy involvement.

Turning science into access: lessons from a global Phase 3 AML study (ADMIRAL)

Markus Vallaster, Astellas Pharma (United States)

The ADMIRAL trial tested gilteritinib (a targeted drug against FLT3, the most common molecular abnormality in AML) against salvage chemotherapy in relapsed/refractory FLT3-mutated AML. Gilteritinib improved median survival (9.3 vs 5.6 months) and more than doubled remission rates. A single global pivotal trial supported approvals across Japan, the US, the EU, Canada and Asia-Pacific — moving from first-in-human to first approval in about five years.

Interestingly, different regulators leaned on different evidence: Japan and the FDA approved first on early response (remission) data, enabling faster access, while the EMA’s approval was driven by more mature overall-survival data. The lesson: response-based endpoints can open early access where the need is high, but survival evidence remains central to global alignment.

Key messages for patient advocates A single well-designed global trial can bring a new medicine to patients across many countries in a few years — and early-response endpoints can accelerate access for patients with urgent, unmet need.Different regulators may accept different evidence at different times, which is why the same drug can reach patients on different timelines in different regions — useful context when advocates field questions about why access varies.Early, coordinated engagement between developers and multiple regulators is what makes fast, broad access possible — a process advocates can encourage from the sidelines.

Glossary of key terms

ALL	Acute lymphoblastic leukaemia
AML	Acute myeloid leukaemia
Aza/Ven (HMA-Ven)	Azacitidine plus venetoclax — a lower-intensity treatment combination
CAR-T	Chimeric antigen receptor T-cell therapy
EMA	European Medicines Agency (licenses medicines in the EU)
FDA	US Food and Drug Administration
HTA / HTAR	Health Technology Assessment / HTA Regulation (assesses a medicine’s value for health systems)
IC	Intensive chemotherapy
JCA	Joint Clinical Assessment — the shared EU assessment of a medicine’s clinical evidence
JSC	Joint Scientific Consultation — early EU advice to developers
MRCT	Multiregional clinical trial — a trial run in more than one region under one protocol
MRD	Measurable (minimal) residual disease — how much cancer remains after treatment
OS	Overall survival
PED	Patient Experience Data — systematic data on what patients experience and prefer
PFS / EFS	Progression-free survival / Event-free survival
PICO	Population, Intervention, Comparator, Outcome — the frame defining an assessment question
PRO / PROM	Patient-Reported Outcome / the measure (questionnaire) used to capture it
QoL	Quality of life
RWE / RWD	Real-World Evidence / Real-World Data
SoC	Standard of care

Report prepared for patient advocates from EHA Congress 2026 session content. Summaries are for advocacy and educational use and are not medical advice.