Written by Louis Barwell
September was a month in which health technology assessment (HTA) policy and HTA decisions were closely linked. In the UK, the pricing agreement with the US came under parliamentary scrutiny not long after the agreement was formalised, most visibly in the National Institute for Health and Care Excellence’s (NICE) decision reversal on trastuzumab deruxtecan (Enhertu®) (initial rejection for TA992 in July 2024). Across the Atlantic, the expansion of Most Favoured Nation (MFN) pricing raised the stakes of international price referencing. In Europe, early Joint Clinical Assessments (JCA) are starting to show what comparative evidence assessors expect. Here’s our summary of key September HTA updates, and what they mean for evidence generation and submission strategies.
UK-US pharmaceutical pricing:
Life sciences industry competitiveness and parliamentary scrutiny in the UK
In a letter sent to the National Audit Office (NAO) on 15th September 2026, the chairs of six House of Commons parliamentary committees have asked for a ‘rapid investigation’ into the cost to the UK public sector of the UK-US pharmaceuticals agreement announced by the Government in December 2025.
As part of that deal, the US Government committed to imposing zero tariffs on UK manufactured pharmaceuticals exported to the US for at least three years. In return, the UK Government agreed to increase NICE cost-effectiveness thresholds from £20–30k to £25–35k per quality-adjusted life year (QALY) and increase National Health Service (NHS) spending on new medicines from 0.3% of gross domestic product (GDP) to at least 0.6% by 2036.
The Health and Social Care Committee had repeatedly requested a long-term impact assessment for these agreements to enable ‘appropriate parliamentary scrutiny’, and to further understand the long-term implications on NHS budgets and wider public expenditure. In the absence of this, the six chairs have called for a comprehensive assessment covering future years in their joint letter.
At the same time, a UK pharmaceutical competitiveness report by the Association of the British Pharmaceutical Industry (ABPI) has stated that in the three months following the UK cost-effectiveness threshold increase in April 2026, nine additional medicines were recommended for use in the NHS, which would have been denied to patients if the threshold had not been raised. The new NICE cost-effectiveness threshold and EQ-5D-5L value set changes have also reopened a previously rejected appraisal (See NICE section below).
Medicaid and MFN: expansion of the US MFN pricing
The US Government announced in September that MFN drug prices agreed with pharmaceutical companies are being expanded to Medicaid programmes in all 50 US states, Washington DC, and Puerto Rico.
Under these plans, state Medicaid programmes will receive rebates from pharmaceutical companies through the GENEROUS Medicaid payment model. The White House stated that rebates will ensure the final price to Medicaid ‘does not exceed the MFN price’ on prescription drugs.
Since September 2025, deals have been reached between the US Government and 26 pharmaceutical companies (around 90% of the branded US market) as part of the MFN agreements. As the MFN benchmarks US prices against other developed countries such as the UK, list prices, launch timing and discounts in those countries are of high importance.
NICE: four different ways to achieve HTA success
September’s positive NICE recommendations illustrate four different ways to achieve reimbursement success, and highlight the importance of recent changes to evaluation methods:
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- NICE’s final draft guidance recommended trastuzumab deruxtecan (Enhertu®) for adults with HER2-low advanced or metastatic breast cancer whose disease has progressed after chemotherapy. Previously (TA992 in July 2024), NICE had been unable to recommend it at the proposed price, with Enhertu® being the only breast cancer drug that NICE hadn’t recommended since 2018. NICE recently acknowledged that a commercial solution had now been agreed for use in this patient population following changes made to NICE methods as part of the UK-US pharmaceutical pricing agreement, and the new quality-of-life method published on 27th August 2026.
- NICE recommended ibrutinib (Imbruvica®) with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisolone (R-CHOP) for untreated mantle cell lymphoma (MCL) as a transplant sparing strategy in eligible patients. NICE recognised the efficacy gains as well as the avoidance of autologous stem cell transplant (ASCT) toxicity and resource use, with clinicians therefore noting the potential ‘step change’ in management of first-line MCL.
- NICE recommended durvalumab (Imfinzi®) in combination with platinum-based chemotherapy followed by maintenance with durvalumab in untreated advanced or recurrent endometrial cancer that is mismatch repair deficient (dMMR). However, NICE did not recommend durvalumab then maintenance durvalumab plus olaparib for endometrial cancer that is mismatch repair proficient (pMMR). The committee highlighted that the clinical benefits of the durvalumab-based strategy were clearer in dMMR. The committee also noted that only the dMMR subgroup without olaparib maintenance demonstrated acceptable cost effectiveness, highlighting NICE’s increasing use of biomarker-defined decision making.
- NICE also recommended mirdametinib (Ezmekly®) for NF1-associated plexiform neurofibromatosis type 1 in people aged 2–17 years using a cost-comparison approach, concluding that mirdametinib was clinically and economically comparable to selumetinib (Koselugo®). NICE recommended using the least expensive option of the suitable treatments available.
Taken together, these decisions highlight the importance of recent changes to evaluation methods, and reinforce that NICE’s focus remains firmly on delivering value whether through improved outcomes, reduced healthcare resource use, or lower overall system costs.
EU HTA: from eligibility to evidence in early JCA
Source’s August HTA update covered the tools for successfully approaching a JCA; key to September news is that the first advanced therapy medicinal product (ATMP) onasemnogene abeparvovec (Itvisma®) has now gone through the JCA process. Assessors repeatedly flagged uncertainty arising from indirect comparisons, residual confounding and limited comparator data. This sits alongside the tovorafenib (Ojemda®) submission, where seven of eight PICOs could not be assessed because of missing comparative data. Early JCAs have highlighted that comparative evidence generation, particularly where submissions rely on single-arm studies or external comparators may be one of the biggest determinants of JCA success. The new Joint Scientific Consultation (JSC) window (23rd September to 21st October 2026) provides an opportunity for developers to address these challenges before pivotal evidence plans are final.
This month, the Commission has also released three new templates to support the written input of patients, carers and clinicians in JCA.
SMC: data collection
This month, the Scottish Medicines Consortium (SMC) published advice on eight medicines. All were accepted, with two under restricted use:
Accepted:
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- Atidarsagene autotemcel (Libmeldy®) for metachromatic leukodystrophy, having been available previously through the ultra-orphan pathway
- Lorlatinib (Lorviqua®) for advanced anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) having been accepted on an interim basis in 2020
- Elacestrant (Korserdu®) for a type of advanced breast cancer in post-menopausal women and in men
- Epcoritamab (Tepkinly®) for follicular lymphoma after two prior lines of treatment
- Pembrolizumab (Keytruda®) for resectable locally advanced head and neck squamous cell carcinoma
- Seladelpar (Livdelzi®) for primary biliary cholangitis
Restricted use:
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- Capivasertib (Truqap®) with fulvestrant for a type of advanced breast cancer
- Nusinersen (Spinraza®) for pre-symptomatic 5q spinal muscular atrophy (SMA)
Two of the eight technologies will now be available for routine use following a period of further evidence collection (Libmeldy® and Lorviqua®), highlighting that data gathered during interim or managed access periods can convert to routine reimbursement.
NCPE
September has been another busy month for completed assessments by the National Centre for Pharmaeconomics (NCPE). Three full HTAs were completed:
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- Trastuzumab deruxtecan (Enhertu®) for HR-positive, HER2-low metastatic breast cancer after at least one endocrine therapy
- Durvalumab (Imfinzi®) for muscle invasive bladder cancer
- Blinatumomab (Blincyto®) for acute lymphoblastic leukaemia.
The NCPE recommended Enhertu® not be considered for reimbursement unless cost-effectiveness can be improved relative to existing treatments, whereas the NCPE recommended that Imfinzi® and Blincyto® be considered for reimbursement if cost-effectiveness can be improved relative to existing treatments. Seven rapid reviews were also completed in September, with four proceeding to full HTA review.
Conclusion and looking ahead:
September’s developments point to three clear learnings when considering evidence and market access strategies:
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- Value remains the defining factor in reimbursement decisions. While changes to NICE methods and cost-effectiveness thresholds may create new opportunities, positive recommendations still depend on demonstrating a robust economic case and clear value relative to relevant comparators.
- Comparative evidence is becoming increasingly important. Early experience from EU JCA suggests that evidence gaps remain a key barrier to successful assessment and will need to be addressed early in drug development.
- Pricing and market access decisions are becoming more globally interconnected. From the UK-US pharmaceutical agreement to the expansion of US MFN pricing, decisions made in one market are increasingly influencing reimbursement opportunities and commercial strategy elsewhere.
As we move into October, further insights are expected from ongoing NICE appraisals, NCPE assessments and the next wave of EU HTA activities, all of which will continue to shape the HTA landscape.
If you would like to learn more about our HTA submissions (including systematic reviews, health economic modelling, and medical writing), please contact us at Source Health Economics, a HEOR consultancy specialising in evidence generation, health economics, and communication.
