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.

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.

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.

"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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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’

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.

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?

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.

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.

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.