This ISPOR poster explores policy options to address challenges faced by chronic treatments where improving survival negatively impacts cost-effectiveness.
Strategies for addressing this issue were tested using an adapted model. Different policy options were considered including discounts to QALYs or costs, applying QALY weights, and removing certain costs associated with improved survival. The most impactful scenario was applying an uncapped QALY weight based on life years gained.
Decision modifiers currently used by policymakers in many cases restrict treatments to the most cost-effective subgroups of patients. We identified alternative policy options that can improve cost-effectiveness without limiting patient access to treatment.
Download PDF Value-based agreements (VBAs) are increasingly seen as essential in the US to address high drug spending by linking pharmaceutical payments to real-world clinical and economic outcomes. A literature review and expert survey found growing interest in VBAs, particularly for costly therapies like oncology and gene therapies, but uptake is limited by administrative burdens, data tracking challenges, and regulatory hurdles. While most surveyed industry experts anticipate greater VBA adoption in the future, success will depend on investments in data infrastructure and streamlined processes. VBAs are poised to grow but may not suit all therapies.
Download PDF International Reference Pricing (IRP) has become a central policy debate in the US, with renewed attention following the Trump administration’s recent revived interest in the Most Favored Nation (MFN) model.
This ISPOR poster reviewed the history and challenges of IRP in the US, drawing on European case studies and an expert survey. While IRP could reduce US drug prices, industry experts highlighted risks to innovation, drug launch timelines, and administrative burden. European experience shows that transparent, strategic IRP policies are essential, but implementing IRP in the US would be highly complex and carry significant implications for the global pharmaceutical industry.
Download PDF The EU Joint Clinical Assessment (JCA) is a vital first step toward successful national pricing and reimbursement procedures in Europe, running in parallel to the EMA process. With tight timelines and complex demands, manufacturers must plan early, align cross-functional teams, and develop high-quality dossiers that address the requirements of member states. Cogentia offers end-to-end JCA support—from PICO planning and joint scientific consultation to dossier development and post-submission engagement. With deep expertise in European HTA, a wealth of experience in scientific communication (including over 150 NICE submissions), and expert knowledge of the JCA documentation, Cogentia helps manufacturers prepare for JCA efficiently and effectively, ensuring readiness at every stage and maximising the value of their clinical evidence package.
Download PDF "By Any Genes Necessary: From Pipeline to Pricing and Reimbursement: Trends in the Gene Therapy Landscape and Implications for the Future" dives deep into the seismic shifts shaping this revolutionary field. This whitepaper emphasises the critical importance of robust value demonstration, strong pricing models, and early stakeholder engagement to navigate the evolving gene therapy landscape successfully.
Download PDF Our analysis of 59 orphan medicines reimbursed in Spain from January 2021 to June 2024 highlights the frequent use of funding conditions, with each drug subject to at least two on average. Common conditions included population restrictions (47.5%), maximum cost per patient (25.4%), and early access with rebate commitments (23.7%). While clinical protocols and Valtarmed usage declined, early access and trial-at-no-cost agreements increased. These trends suggest Spanish payers are leveraging funding conditions to manage costs while accelerating access to orphan drugs for eligible patients.
Download PDF Our analysis of 31 Highly Specialised Technologies (HSTs) evaluated whether these ultra-rare, “severe and disabling” conditions would qualify for NICE’s STA severity modifiers. Of 16 evaluable HSTs, 50% achieved a 1.7 severity weight, 19% a 1.2 weight, and 31% no modifier. Notably, 1 of 7 HSTs with a QALY weight assigned by NICE did not qualify under STA criteria. These findings suggest that NICE’s use of discounted QALYs disproportionately affects chronic conditions, which contrasts with HST methods that consider undiscounted QALYs, potentially disadvantaging long-term conditions under STA evaluations.
Download PDF Our analysis of NICE’s updated methods and processes guide (February 2022) shows that rare disease STAs still face delays in access timelines. Comparing six rare and six non-rare disease STAs published after January 2023, rare disease STAs averaged 514 days to access versus 280 days for non-rare STAs. These delays mirror pre-update timelines (504 days), with no change in the average number of ACMs (2). However, the time in NICE for rare disease STAs has decreased by 178 days since the update, suggesting partial improvement, though access delays persist.
Download PDF Our analysis of NICE’s Innovative Medicines Fund (IMF) revealed that, despite its launch in 2022 with £340M to fast-track non-oncology drugs, it remains unused for managed access. Of 20 technology appraisals (TAs) analysed, 11 achieved reimbursement, but none used managed access. Barriers included lack of manufacturer engagement (12 TAs), cost-effectiveness issues (3), and data challenges (3). Manufacturers often prioritized routine commissioning, avoiding IMF's risk of providing drugs free if post-access agreements fail. These findings suggest that concerns over financial commitments may deter IMF uptake.
Download PDF NICE’s highly specialised technology (HST) routing has restrictive criteria, leading many rare disease treatments to be evaluated via the single technology appraisal (STA) route. Our analysis of 13 public HST checklists found only 5 routed to HST, 6 to STA, and 2 suspended. HST criteria were met 69% of the time, but the key criterion—benefit over existing treatments—was unmet 83% of the time for STA-routed products. HST reviews take 1.5 years longer on average. The findings highlight the importance of innovation in meeting HST criteria and suggest consistent, polar decision-making by TSOP.
Download PDF In 2023, a record seven gene therapies were FDA-approved, with high expectations for 2024. Our study analysed 124 gene therapies, revealing positive developments for 52, limited/no developments for 48, and negative developments for 24. Key themes include a shift toward prevalent conditions, improved safety, and competitive de-prioritization. Rare diseases still dominate, with high prices like Lenmeldy at $4.25M posing affordability challenges. Therapies for more prevalent conditions along with technological advances are likely to further influence pricing and reimbursement, with budget concerns remain significant for payers.
Download PDF Our analysis evaluated patient involvement initiatives (PIIs) in HTAs across England, Germany, Spain, and Poland. Using a five-domain scoring system, England excelled with high scores across all domains, while Germany, Spain, and Poland showed varying gaps, particularly in patient inclusion and process consideration. A case study highlighted England's effective PII framework, where the Sickle Cell Society successfully appealed a NICE decision, leading to voxelotor's approval. To enhance PIIs, we recommend legal frameworks for patient involvement, mandatory reviews of engagement processes, plain language training, and broader outreach to capture diverse patient perspectives.
Download PDF Many markets currently lack comprehensive evaluation processes for digital health products (DHPs), which risks limiting reimbursement, access, and adoption. Our analysis examines market access, evaluation processes, and recent reimbursements of DHPs across US, UK, and EU4 markets. Germany and the UK have mature DHP landscapes with clear reimbursement processes, while France and the US require more functional processes. For example, around 30% of digital companies abandoned launches in France due to regulatory hurdles. Dedicated evaluation pathways are not available in Italy and Spain, emphasising the varying reimbursement processes present across markets, and potential for future harmonisation of digital health assessments.
Download PDF France’s Early Access Programme (AAP), introduced in July 2021, enables early access to medicines with free pricing upon scheme entry. This study examines the price changes for innovative medicines in the AAP between 2021-2024, focusing on the impact of reference pricing. From 2021-2024, 78 orphan medicines entered the AAP, with 39 fully reimbursed and an average price reduction of ~16%, mainly driven by reference to Germany’s post-benefit assessment prices. Our analysis highlights the importance of evaluating pricing implications in early access programmes, particularly in France, where manufacturers may face retrospective rebates if prices during AAP differ significantly from launch.
Download PDF This analysis of HST vs STA topic selection decisions, based on data obtained through a Freedom of Information request to NICE provides the most comprehensive review of NICE topic selection to date.
Our analysis addresses four key objectives:
- To explore the impact of routing decisions on the ultimate appraisal outcome
- To establish the key contributing factors to treatments being routed via HST or STA
- To better understand the topic selection decision-making process, including the interpretation of each criterion and the use of external sources to inform it
- To provide recommendations to manufacturers with orphan medicines that they believe may be eligible for HST routing.
Our findings support the conventional wisdom that for manufacturers targeting ultra-rare conditions, there is no more critical step in a successful UK market access strategy than qualifying for HST.
Download PDF We reviewed studies that reported on the number of varicella cases, doctor visits, hospitalizations, and costs due to varicella and associated complications, and developed an analysis of the overall cost burden. Even though less than 1% of varicella patients need to be hospitalized, costs remain high because varicella is so common. Furthermore, if the number of workdays lost are counted as well, then varicella-related costs are even higher. We found that Varicella-related primary healthcare visits account for 30% to 80% of total direct costs, and Varicella-related hospitalizations contribute between 2% and 60% to total direct costs. Varicella-related hospitalizations may occur both in children and adults, with costs ranging between $1,308 and $38,268 per episode depending on severity, complication type and length of stay.
Download PDF Sub optimal targeting in early development is costly, and it is increasingly important to start the development with the end in mind, and considering it’s potential value. Identifying what outcomes need to be achieved, whether sub populations should be targeted and evidence requirements to maximise the value of the asset is the aim. In this case study we highlight how the Target Product Profiles (TPPs) can be optimised through leveraging an early economic model and a forecast model, and a multidisciplinary team review.
Download PDF Healthcare inequality can take many forms, from geographical differences in
access between countries, to funding challenges for rare disease treatments,
and even a disparity in health burden between different ethnicities. As part of her talk at the World EPA Congress 2024, Abbey highlighted some key initiatives which have aimed to level the
playing field. To find out more download your copy.
Download PDF In this paper, Cogentia reviews one of the newest advanced therapies market segments: stem cell therapeutics. Following a review of the pipeline, we highlight the most relevant market access challenges and principles for manufacturers to consider for a successful commercialisation of these assets. These principles can also be applied by manufacturers of ATMP assets to plan market access strategy for their assets.
Download PDF As Health inequalities are becoming increasingly relevant to Health Technology Assessment (HTA), it will be ever more important to assess inequalities systematically. DCEA is a tool for the job. However, a full DCEA is more time demanding than CEA. We believe an aggregate DCEA provides balance between company analytical resource-time constraints and the systematic evaluation of health inequalities. Irrespective, HTA agencies need to develop clear reference-cases to enable DCEA’s future in HTA submissions.
Download PDF In the UK, Highly Specialised Technologies (HSTs) are interventions used for treatment of very rare conditions. Technologies eligible for HST routing are evaluated against strict criteria. In fact, evaluation of criteria for HST routing are polar extreme; HST routing approval is only achieved when all checklist criteria are met. But the biggest determination for HST routing success is product innovation in a sparse competitor landscape. Without benefit over existing treatments, products are rerouted to Single Technology Appraisal (STA).
Download PDF The European Union (EU) has proposed a new Regulation to address the
challenge of antimicrobial resistance by introducing a transferable data
exclusivity voucher (TEV) as an incentive for priority antimicrobial
development. The objective is to encourage pharmaceutical companies to invest
in this area despite limited market prospects. This conceptual exploration
examines the pros and cons of the TEV scheme through a targeted literature
review. While TEVs may incentivize innovation, they may also result in higher
drug prices, lack of guaranteed access to antimicrobials, and a potential shift
away from preventive measures. The forthcoming implementation has generated
mixed opinions, with critics expressing concerns about higher drug prices and
limited access, while the research-based pharmaceutical industry generally
supports the concept. The TEV has the potential to generate significant
revenues and could be an initial step in combating antimicrobial resistance.
Download PDF NICE launched the Innovative Medicines Fund in June 2022, with the objective of levelling the playing field for non-oncology drugs, following in the footsteps of the Cancer Drugs Fund. However, utilisation of the IMF has been limited to-date. This poster presents research conducted to analyse the reasons behind the lack of uptake, which appear to be driven primarily by a lack of willingness to engage on the manufacturer’s part. Interestingly, manufacturers preferred to offer larger discounts to secure routine commissioning rather than pursue managed access.
Download PDF Exploration of the evolving digital health landscape in EU5 and US markets, the core focus being reimbursement challenges. Germany, France, and the UK appear to be leading in the digital space with standardised evaluation frameworks for these products. Despite this, reimbursement may still be achieved in markets without comprehensive frameworks although decentralised evaluations and decision-making processes can restrict widespread adoption and subsequently the extent of accessibility.
Download PDF In England and Wales, patent protection spans 20 years. Despite superior clinical effectiveness, numerous technologies faced rejection by NICE since 1999 due to unattainable cost-effectiveness from high expenses. We have studied Abiraterone as a case, which received a negative NICE recommendation. We anticipated a cost reduction post-patent expiration in September 2022, which actually happened and led to the introduction of generic versions with cost-effective prices.
Download PDF Interest in health technology assessment (HTA) by global markets increased between 2015 and 2021. Majority of largest healthcare markets by region have already implemented HTA processes to support decision-making in healthcare. Cost-effectiveness assessments (CEA) are very common in HTAs, with over 70% of the largest markets using them. Future research is needed to understand the progression of HTA implementation across global markets, as well as the level of influence established CEA HTAs have on the newly developed HTAs processes on other global markets.
Download PDF Cogentia have recently conducted a global market review to assist our client in planning for a successful relaunch of their pharmaceutical product across a total of 40 scope markets.
Our expertise in market understanding and strategy development can assist successful product commercialisation. Check out our case study below for more details.
Download PDF As we acknowledge the International Day of Women and Girls in Science today, we thought we would share our journeys in the world of science, technology, engineering and mathematics (STEM).
Download PDF Biosimilars are an ever-increasing part of the treatment landscape, with their use being driven by multiple and complex factors. With the opportunity for reduced healthcare costs, is this the year for Biosimilars?
Download PDF NICE’s revised methods introduced a severe disease QALY modifier. We applied the new modifier criteria to HSTs, which must be considered “severely disabling” by NICE and found that one-third of HSTs did not achieve a QALY weighting. We believe that a key determinant is the use of discounted QALYs in assessing shortfall, which penalizes chronic diseases where morbidity and mortality accumulate over a longer time.
Download PDF There is a mismatch in resourcing between high morbidity and high mortality conditions. This is especially apparent when comparing two diseases of comparably high burden: Migraine and Multiple Sclerosis. This research interrogates the discrepancy in market size, prevalence, and pipeline between these diseases, while quantifying the remarkably similar burden’
Download PDF Clever or innovative technologies do not always translate to innovative products. In this work we developed a tool to assess new technologies, to prioritise product development, and support the selection of an optimum 1st target test, indication and therapy area. The methodologies and outputs are discussed.
Download PDF Antimicrobial resistance (AMR) is a threat to global health, here we reviewed the policies and incentives globally that aim to address the health economic challenges of antimicrobial development. Can we have varied approach or does there need to be real change at an international level?
Download PDF NICE have reduced the number of scoping criteria for products entering HST from seven down to just four with the aim of making “routing decisions clearer, consistent, more transparent and precise to provide greater clarity, precision and predictability for the routing of topics”. This research investigates whether the new scoping criteria enable greater predictability for the routing of topics compared to the former criteria, and the potential impact on patient access in the UK.
Download PDF Minimally invasive paddle-type spinal cord stimulation (MI-SCS) harnesses ideas from the fields of bioelectronics and soft robotics and combines the benefits of conventional SCS devices. In this research, we developed an early economic model exploring the cost-effectiveness of MI-SCS vs. conventional forms of SCS from the perspective of the UK NHS.
Download PDF Cell and gene therapies, with their anticipated pipeline boom, have been a hot topic in the pharmaceutical landscape for some time. Here, we take a deep dive into the gene therapy pipeline, analysing events over the past 12 months, and drawing out key themes emerging, considering implication for pricing & reimbursement.
Download PDF The FDA anticipate that 10-20 cell and gene therapies will be approved per year by 2025. Prevalence of target diseases has a big impact on payers, as well as manufacturers. Here, we analyse the prevalence of disease areas being targeted in gene therapy clinical trials, finding rare diseases (50%) to be the target of choice, likely driven by a balance between achievable price and prevalence.
Download PDF As a wave of gene therapies reach the market, manufacturers must consider commercial viability. Here, we aim to devise a framework of factors that should shape early decision making around which disease area to target for a gene therapy. Factors to consider include prevalence, age of administration, disease burden, resource use, current treatment options, and price precedent.
Download PDF The question of what constitutes gene therapy sustainability to developers and healthcare systems has never been more critical. Cogentia conducts an analysis to identify the economic factors that contribute towards commercial attractiveness for 10 key target disease areas within the gene therapy pipeline. Furthermore, we highlight that having a simple framework to aid targeting at the preclinical stage promotes long-term sustainability of gene therapy.
Download PDF Identification of appropriate stroke event and management costs in Atrial Fibrillation
Publication type: Publications, Articles
Project type: Economic modelling, Market Understanding, HTA Submissions, Evidence synthesis (including SLR, TLR and meta-analysis)
Complexities exist in selecting suitable stroke costs for use in economic evaluation with a nonvalvular atrial fibrillation (NVAF) population. Cogentia conducts a literature review to systematically identify and review the ischaemic stroke (IS) and intracranial haemorrhage (ICH) cost sources available. The study findings highlight that further research is warranted in this area.
Download PDF A structured review of the stakeholder comments received during the development of ‘Atrial Fibrillation: Diagnosis and Management’ (NG196) show that stakeholder views highlighting limitations and errors within the body of evaluated evidence led to a reversal in draft direct oral anticoagulant (DOAC) recommendations. This demonstrates the significance that NICE places on the views of stakeholders in developing evidence-based guidelines.
Download PDF The first gene therapy treatment was approved in 2012 in Europe and in 2018 in US. There are signs that the rate of gene therapy approvals is set to accelerate with the FDA predicting around 10-20 gene therapy approvals a year by 2025. This study describes the disease target and level of competition in the pipeline of gene therapy assets undergoing clinical studies to evaluate the sustainability of this pipeline.
Download PDF Population size is important for selection of NICE-appraised HSTs, but not all orphan indications can be routed to HST. This study observed acceptable evidence base in the context of size-based HST criteria. Although patients per trial in published HSTs do not appear to change over time, there may be a trend towards reduced sample size in pivotal trials of proposed HSTs, possibly representing tightening of size-based criteria.
Download PDF By Any Genes Necessary - Reflections on a Decade of Gene Therapy, and Key Considerations around Sustainability as we Look to the Future
Publication type: Publications, Case studies
Project type: Landscape, Opportunity assessment, Market Access Strategy, Market Understanding, Value Communication
In this paper, Cogentia reflects on the past and looks to the future, highlighting factors that will likely contribute to the commercial viability of target disease areas. We share insights from a framework exploring the commercial attractiveness of future entrants and the obstacles that they face, and share our thoughts on the future sustainability of gene therapy for payers and the industry.
Download PDF Discrete WTP thresholds for HST were introduced by NICE in April 2017, starting at £100,000/QALY up to a maximum of £300,000/QALY, on a sliding scale for undiscounted QALY gains above 10. This study examined whether introduction of these thresholds has impacted the publication of positive HST guidance. With the caveat of small numbers, publication of NICE HST guidance appears to be reduced and positive guidance more challenging to achieve in the post-threshold era.
Download PDF Changing from a vial-based formulation to a PFS can be driven by desire to gain a competitive edge, to improve safety and accuracy, or to reduce wastage or a combination of these factors. The launch of a novel PFS formulation of an existing vial-based drug can typically follow an abridged procedure with price parity the likely outcome. However, price premium can be achieved if benefits demonstrated are in line with the unmet need.
Download PDF Treatment failure in asthma due to suboptimal adherence to inhaled corticosteroids is common. In practice, self-reporting offers a simple, inexpensive method of assessing adherence, but typically over-estimates it. Measurement and management of adherence are at the core of optimising expensive biological treatments for asthma, particularly in the context of COVID-19 pressures on health systems.
Download PDF There are a number of smart inhaler concepts in development, but a key question is what their impact is, and whether they are cost effective and in what settings. Even at their most basic functionality, a smart inhaler is cost effective and cost saving in a paediatric secondary care setting based on early economic modelling.
Download PDF The aim was to evaluate the existing frameworks in place for managed access agreements in emerging markets and identify factors that influence access to innovative therapies. Managed access agreements are a useful tool in introducing innovative therapies in emerging markets, and it is important to plan ahead at an early stage, as well as understand the differences between country frameworks.
Download PDF Access arrangements such as the PAS have become increasingly common in the UK. NICE appraisals before 30/06/20 were reviewed, showing an increasing trend towards confidential agreements since 2007. This is likely to reflect the challenges of achieving UK access in a wider context of global pricing and referencing. With the increase in high-cost therapies, this is a trend that is set to continue.
Indirect Treatment Comparisons have been published on the comparative effectiveness of DOACs for stroke prevention in non-valvular atrial fibrillation. Network Meta-Analyses have focused on comparisons in overall trial populations, despite known differences in patient characteristics across treatments. This analysis explores DOAC treatment effects in an NVAF population at high risk of stroke.
During the pharmaceutical lifecycle, economic modelling is usually reserved for HTA and rarely plays a part in determining TPPs or go/no go decisions. Yet the two are intrinsically interlinked; early modelling is a useful tool to guide strategic decisions in development or in-licensing. Pharmaceutical companies can benefit from involving health economics at an early stage of development.
Download PDF NICE introduced HST in 2015 to consider drugs for very rare conditions with higher WTP thresholds and lower evidence requirements than STA. In general, drugs assessed through HST met most, but not all of the 7 HST criteria, with many orphan drugs in STA meeting the same amount. Consequently, some orphan drugs are assessed via more challenging routes for reasons that are unpredictable and unclear.
Download PDF