We understand that your journey to health technology assessment (HTA) is not always a smooth one, that one size does not fit all, and that patient access to treatments faces many challenges.
At Tolley,
we take a bespoke approach to each project and develop decision-focused materials that meet your UK and Republic of Ireland (RoI) HTA needs and requirements for Joint Clinical Assessment (JCA) in the EU. We can also support adaptations of economic models to the needs for HTA bodies in other countries, including the US, wider Europe, and globally.
Early HTA preparation ensures a smooth, efficient pathway
to product launch and reimbursement with activities focused on two strands:
Core HTA relevant evidence generation
We divide activities around
HTA evidence preparation
for pharmaceuticals into
three key components:
…is best started in the pre-phase 3 trial period approximately 2-3 years prior to UK launch.
This includes general feasibility and preparation activities such as precedence analysis of prior HTA submissions; HTA Evidence Needs Assessment/Gap Analysis; and the writing of Briefing Books for NICE Advice, plus planning of strategic elements (Value Messages Development), and formation of an HTA Roadmap for the future.
…early clinical and economic SLRs, early economic modelling, full HTA ready economic models, indirect treatment comparisons, healthcare cost and resource use studies, structured expert elicitation (SEE) of key data, health-related quality of life (HRQoL) and patient-reported outcomes (PRO) strategy, utility studies, burden of disease and carer burden data collection, and input to out-of-trial real world evidence generation planning and analysis.
The aim is to generate HTA-ready deliverables, along with continued HTA stakeholder engagement, for preparation of a robust NICE/SMC/NCPE submission dossier and HTA-ready cost-effectiveness model, to facilitate a positive HTA recommendation.
Component 3: Influencing the HTA environment…
…through engagement with key HTA stakeholders including patient advisory groups (PAGs), clinical KOLs, and HTA bodies/NHS England, and associated groups, often runs adjacent to pivotal trial data collection and involves implementation of strategic ideas and further evidence generation and collection based on the HTA Evidence Needs Assessment/Gap Analysis in Component 1.
Multi-stakeholder panels and advisory boards can be created to accompany the HTA journey. This also involves communications through a publication strategy to disseminate key HTA relevant evidence, via conference abstracts and publications in peer-reviewed journals.
Evidence needs and gap analysis assessment
To secure reimbursement by the UK National Health Service (NHS) and UK and Republic of Ireland HTA bodies requires robust and appropriate clinical and economic evidence with optimal value propositions.
At Tolley, we perform Evidence Needs and Gap Analysis Assessment for UK and RoI HTA based on applying a unique framework comprising four key pillars of evidence. The pillars of evidence are designed to capture the four essential components of evidence required for optimal HTA submissions to NICE, SMC, AWMSG, and NCPE in Ireland, and are based on the value framework and evidence needs set out in the 2022 NICE health technology evaluations process and methods manual.
The Evidence Needs and Gap Analysis Assessment is bespoke to each treatment and disease area but
would be employed to assess existing evidence, inform further evidence generation plans, and make preliminary recommendations of how necessary evidence can be generated within appropriate timeframes to support value propositions for UK HTA submissions. Along the HTA journey for a new therapy, it can be applied early, pre-phase 3 trials, to assess evidence needs in terms of trial design and endpoints, and complementary out of trial evidence generation activities to support HTA needs across the four Pillars, and/or later (e.g. post-phase 3), to assess remaining key evidence gaps across the four pillars.
A four pillars Evidence Needs Assessment for UK HTA would include:
HTA Evidence Generation
Disease burden and unmet need
Clinical effectiveness
Cost effectiveness
Innovation and health inequalities
Our systematic reviews follow a transparent process and are undertaken according to the principles of systematic reviewing published in the Cochrane Handbook, the Centre for Reviews and Dissemination (CRD), and the NICE health technology evaluations manual, in line with the PRISMA-P checklist, and reported according to PRISMA. This transparency is crucial for HTA submissions.
Systematic reviews play a pivotal role in supporting evidence-based decision-making by assessing the available clinical and economic evidence for specific interventions. We conduct a feasibility assessment and partner with an experienced Information Scientist to understand all elements of the PICO framework and to formulate a research question for your review. We think ahead to your potential indirect treatment comparisons such as network meta-analyses (NMAs) and matching-adjusted indirect comparisons (MAICs) by partnering with Estima (www.estima-sci.com) for all your statistical needs.
All our reviews are registered with PROSPERO to enhance transparency and contribute to the integrity of systematic reviews. We encourage you to publish your SLRs through abstracts at leading clinical and HEOR conferences, and through peer-reviewed journals.
Want to take a more pragmatic approach?
If you want to inform your early economic model, or carry out research independent of HTA, we can conduct targeted literature reviews that meet your needs.
Case studies
Publication of the SLR and NMA for a non-statin, lipid-lowering therapy for patients with hypercholesterolaemia following successful reimbursement through NICE
Our health economics team has expertise in developing global and UK cost-effectiveness models that are designed specifically around the client’s technology.
All new healthcare technologies bring something unique to patients and have a specific story to tell. We understand the importance of developing a model around an intervention rather than fitting a technology into a model, helping to ensure that key value messages are demonstrated fully and transparently.
Whether this involves tried-and-tested modelling approaches in areas such as oncology or an entirely new approach to defining health states and patient transitions in rare diseases, our bespoke approach ensures that models provide the foundations to a successful HTA submission.
This is combined with strategic planning to make sure the models are fit for purpose for the specific needs of NICE, SMC, AWMSG or NCPE. We are able to advise on, and deliver, the most appropriate economic evaluation (from cost-utility to cost-minimisation) and model structure to meet specific HTA needs, and provide unique consultancy and advise on its optimal use for achieving a positive recommendation. We have a track record of success in doing this.
We recognise that key strategic decisions for pipeline products often need to be made based on early data readouts or target product profiles,
whether that is to inform go/no-go decisions, establish indicative pricing assumptions, or develop a business case for investment. We also find that early modelling leads to stronger HTA submissions, as it allows clients to identify key cost-effectiveness drivers, data gaps and uncertainties in time for evidence needs to be addressed through additional data gathering.
The structure and complexity of our early models are built around your needs, whether that is to explore cost-effective pricing based on a minimal set of clinical assumptions (such as median survival estimates) or to conduct a more complex analysis of key evidence gaps and cost-effectiveness drivers that can be adapted to a full HTA-ready model.
As well as developing de novo economic models, our team is experienced in quality checking existing models from other global or UK vendors and advising on or carrying out adaptations to meet the required standards for HTA submission to NICE or other UK HTA bodies.
Our extensive experience in supporting client submissions through to final guidance, and close working relationships with assessment groups, means that we are well placed to identify common pitfalls and challenges in evaluation strategies ahead of submissions.
Case studies
Development of the first HTA-ready cost-effectiveness model for epidermolysis bullosa (EB)
Utility and PRO studies
Utility elicitation is a key data need for estimating quality-adjusted life years (QALYs) in cost-utility based economic models that are submitted to UK and RoI HTA bodies, and for other countries that require cost-effectiveness evidence.
We perform robust preference-based utility elicitation studies to generate core estimates of the quality of life impact of health states included in an economic model.
Using time trade-off (TTO) and other recognised methodology, we perform rigorous studies to determine utilities relevant to patients, primary carers, and secondary carers across a range of therapeutic areas, including rare diseases. The team is experienced in advising on the inclusion of EQ-5D and other patient-reported outcome (PRO) instruments in clinical trials or as part of real-world evidence (RWE) studies, in EQ-5D/PRO data analysis, and mapping studies to support use in economic models and HTA submissions.
Starting with a thorough evidence needs assessment for HTA,
we can support all your de novo HTA evidence generation from clinical and economic SLRs, ITCs, cost-effectiveness and budget impact analyses, use of scientific advice, through to stakeholder engagement, use of multi-stakeholder panels, and strategic consultancy.
Alternatively, we can critique and adapt global materials to make them fit-for-purpose. Our adaptable HTA strategy planning approach supports the journey from phase 3 planning to HTA submission and beyond, including support with managed access agreements, and post-HTA data collection needs.
When it comes to your HTA submission, we believe that ones size does not fit all.
‘One and done’ approach
We care that patients can get early access to potentially life-saving new medicines.
Alongside the evidence generation planning for your NICE submission, we can build a bespoke HTA strategy plan framed around our four pillars of evidence.
This includes NICE and SMC process planning, stakeholder management (KOL, patient body engagement), managed access agreement/commercial (PAS) planning, critical activities pathway, and SWOT assessment of disease burden, clinical effectiveness, cost-effectiveness, and health inequalities.
We include pre- and post-submssion support for your NICE, SMC, AWMSG, and NCPE dossiers.
Our team can support with each step in the NICE process including NICE appraisal scoping, the NICE decision problem, preparing the submission dossier, Patient Access Scheme considerations, responding to NICE External Assessment Group clarification questions, preparing your objection handler, and walk you through a mock appraisal committee meeting for a one and done appraisal committee meeting approach to reimbursement and patient access.
Case studies
