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Cogentia

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Better Off Dead: Mitigating For Situations Where Improving Survival Isn't Cost Effective

Project type: Economic modelling, HTA Submissions

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.

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The Evolution of Value-Based Agreements in US Healthcare: Barriers, Opportunities and Future Prospects

Publication type: Publications
Project type: Market Access Strategy, Market Understanding

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.

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The Emerging Potential of International Reference Pricing (IRP) in the US: An Analysis of Trends, Challenges, Opportunities and Learnings from Europe

Publication type: Publications
Project type: Market Access Strategy, Market Understanding

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.

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Joint Clinical Assessments

Publication type: Publications, Case studies, Articles
Project type: Joint Clinical Assessments

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.

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By Any Genes Necessary: From Pipeline to Pricing and Reimbursement: Trends in the Gene Therapy Landscape and Implications for the Future

Publication type: Publications
Project type: Landscape

"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.

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Analysis of Funding Conditions for Orphan Medicines in Spain: Trends in Pricing and Reimbursemment

Publication type: Publications
Project type: Market Access Strategy

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.

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Will NICE’S New Severity Weighting Criteria Be Capable of Identifying Sever Conditions? An update

Publication type: Publications
Project type: HTA Submissions

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.

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Has the NICE Methods and Processes Update Accelerated Access to Rare Disease Technologies Routed Via The Single Technology Appraisal (STA) Pathway?

Publication type: Publications
Project type: HTA Submissions

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.

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The Innovative Medicines Fund or the In Need of Medicines Fund: Analysis of NICE Technology Appraisals to Explore Barriers to Managed Access Agreements

Publication type: Publications
Project type: HTA Submissions

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.

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Consistent Decision-Making by NICE's TSOP Highlights Product Innovation as Critical for HST Routing Success

Publication type: Publications
Project type: Market Access Strategy, HTA Submissions

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.

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From Pipeline to Pricing & Reimbursement: Economic Impact and Access Challenges of Upcoming Gene Therapies

Publication type: Publications
Project type: Landscape, Opportunity assessment

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.

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Patient Involvement Initiatives in HTAs; How can we do better?

Publication type: Publications
Project type: HTA Submissions

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.

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Advancements in Digital Health: Current Landscape & Evaluation Processes for US, UK, & EU4 Markets 

Publication type: Publications
Project type: Landscape, Market Understanding

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.

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Evaluating High-Cost Innovative Medicines: A Three-Year Analysis of Pricing Trends in France's Early Access Programme (AAP) 

Publication type: Publications
Project type: Landscape, Market Access Strategy, Market Understanding

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.

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To HST, or not to HST - that is the question’

Publication type: Publications, Articles
Project type: HTA Submissions

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:

  1. To explore the impact of routing decisions on the ultimate appraisal outcome
  2. To establish the key contributing factors to treatments being routed via HST or STA
  3. To better understand the topic selection decision-making process, including the interpretation of each criterion and the use of external sources to inform it
  4. 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.

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With double of the US population and universal healthcare access, Europe represents the second largest healthcare market in the world.

Publication type: Articles
Project type: Market Access Strategy, Market Understanding

During April and May 2024, we have done a series of articles describing the European opportunity for US-based pharmaceuticals and biotech companies. The posts allow companies to understand the challenges and commercial opportunity of the European market for different disease types. We also describe the pricing, reimbursement landscape and key future developments impacting the European reimbursement of pharmaceuticals.

Find more details on our research and links to all articles below.

- The special opportunity that Europe represents for RARE DISEASE products

- Challenges to commercialise pharmaceutical therapies in Europe

- Pricing and reimbursement landscape in Europe

- Special considerations for the evaluation of Orphan drugs that support higher reimbursement pricing

- Why is a pragmatic approach needed for commercialisation of prevalent disease therapeutics?

- Future developments impacting the European reimbursements, Joint Clinical Assessment description and potential short-term challenges.

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Cogentia conducted a systematic literature review exploring the costs of Varicella and its complications, which was published in the peer reviewed journal of Human Vaccines & Immunotherapeutics

Publication type: Publications
Project type: Evidence synthesis (including SLR, TLR and meta-analysis)

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.

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Leverage - Utilising Early Economic Modelling to Maximise Produce Value & Evidence Development by Quantifying Lever Values

Publication type: Publications

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.

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Healthcare Equity: Is it Fair?

Publication type: Publications

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.

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Stem Cell Therapeutics: The Need for Early Market Access

Publication type: Publications
Project type: Landscape, Market Access Strategy

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.

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Distribution Cost-Effectiveness Analysis: a case study on its potential prospects in HTA

Publication type: Publications, Articles

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.

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Highly Sensible Tactics: Strategic Insights for Navigating Highly Specialised Technology Topic Selection

Publication type: Publications, Articles

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).

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Transferable Exclusivity Vouchers: A Silver Bullet?

Publication type: Publications, Articles

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.

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The In Need of Medicines Fund: Analysis of NICE Technology Appraisals to Explore Barriers to Access via The IMF

Publication type: Publications, Articles

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.

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The Promising Future of Digital Health: A Road to Reimbursement in EU5 & US Markets?

Publication type: Publications, Articles

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.

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Reflection on Medicines That Have Recently Come off Patent and Have Been Rejected by NICE in the Past 20 Years: A Case Study of Abiraterone for Treating Newly Diagnosed Metastatic Hormone Sensitive Prostate Cancer (mHSPC)

Publication type: Publications, Articles

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.

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How Wide Ranging is the Implementation of HTA and Cost-Effectiveness Assessment (CEA) in Major Healthcare Markets

Publication type: Publications, Articles

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.

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Pricing and Reimbursement Evidence Requirements for a Mature Pharmaceutical Product

Publication type: Case studies, Articles
Project type: Market Understanding

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.

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Celebrating women in science

Publication type: Articles

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).

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Is this the year for Biosimilars?

Publication type: Articles
Project type: Market Understanding

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?

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Will NICE's new severity weighting criteria be capable of identifying severe conditions

Publication type: Publications
Project type: HTA Submissions

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.

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What doesn't kill you: Re-evaluation our approach to high burden, low mortality disease with a focus on Migraine and Multiple Sclerosis

Publication type: Publications

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’

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Utilising Health Economics to enable optimal transition from a Platform Technology to a Development Programme and Product

Publication type: Publications
Project type: Opportunity assessment

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.

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When it comes to incentives for Antimicrobial Development - Is a united front essential?

Publication type: Publications
Project type: Market Understanding

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?

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Four is the new seven: The application of new routing criteria for NICE's HST Programme

Publication type: Publications
Project type: Opportunity assessment, Market Access Strategy, HTA Submissions

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.

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An early cost effectiveness analysis of minimally invasive paddle type spinal cord stimulation for the treatment of Chronic Neuropathic Pain

Publication type: Publications
Project type: Economic modelling

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.

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By any genes necessary: An eventful 12 months in the Gene Therapy Pipeline

Publication type: Publications
Project type: Landscape

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.

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Analysis of the Prevalence of Disease Areas in the Gene Therapy Pipeline: from Ultra-rare to Prevalent

Publication type: Publications, Articles
Project type: Landscape, Market Understanding

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.

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Framework for Gene Therapy Disease Area Targeting

Publication type: Publications, Articles
Project type: Landscape, Opportunity assessment, Market Access Strategy, Market Understanding

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.

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Is the gene therapy revolution sustainable and affordable?

Publication type: Publications, Articles
Project type: Landscape, Market Access Strategy, Economic modelling, Market Understanding

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.

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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.

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How does stakeholder consultation inform the development of NICE Clinical Guidelines? The example of Atrial Fibrillation: Diagnosis and management

Publication type: Publications, Articles
Project type: Market Understanding, HTA Submissions

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.

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Analysis of the therapy areas targeted by gene therapy manufacturers in clinical trials

Publication type: Publications, Articles
Project type: Landscape, Market Access Strategy, Market Understanding

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.

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Highly Specialised Trials: Has eligibility for NICE Highly Specialised Technology appraisals become more strict?

Publication type: Publications, Articles
Project type: Landscape, Market Understanding, HTA Submissions

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.

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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.

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NICE Highly Specialised Technology Assessment- Has Introduction of Willingness to Pay Thresholds Impeded Positive Guidance?

Publication type: Publications, Articles
Project type: Economic modelling, Market Understanding, HTA Submissions

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.

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Pricing and Market Access Considerations in Changing from Vial to Pre-Filled Syringe Packaging: EU5 and beyond

Publication type: Publications, Articles
Project type: Market Access Strategy, Market Understanding, Pricing, Value Communication

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.

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Non-adherence in severe asthma – a problem of measurement

Publication type: Publications, Articles
Project type: Landscape, Market Access Strategy, Market Understanding

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.

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Early economic modelling of a smart inhaler exploring cost-effectiveness in a severe asthma setting

Publication type: Publications, Articles
Project type: Economic modelling, Market Understanding, Pricing

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.

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Early access and targeted supply considerations in emerging markets

Publication type: Publications, Articles
Project type: Landscape, Market Access Strategy, Market Understanding, Value Communication

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.

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Deal or No Deal- Twenty Years of Pricing and Access Agreements in Products Assessed By NICE

Publication type: Publications, Articles
Project type: Market Access Strategy, Market Understanding, HTA Submissions

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.

Comparative effectiveness of direct oral anticoagulants for stroke prevention in non-valvular atrial fibrillation

Publication type: Publications, Articles
Project type: Market Understanding, Evidence synthesis (including SLR, TLR and meta-analysis)

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.

Early Health Economic Modelling As A Tool To Guide Strategic Clinical Development And In-Licensing Decisions

Publication type: Publications, Articles
Project type: Opportunity assessment, Market Access Strategy, Economic modelling, Market Understanding

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.

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NICE Highly Specialised Technology Assessment- How Are Selection Criteria Applied In Practice?

Publication type: Publications, Articles
Project type: Market Understanding, HTA Submissions

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.

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