In the first article of this section dedicated to Health Technology Assessments (HTAs), we examined what HTAs are, careful reviews that governments and health authorities conduct before deciding whether a new treatment should be made available and reimbursed in their country. In the second part, we explored why the voices of patients and families are so important in this process and how they can contribute so that regulatory authorities take into account the aspects of living with HD that matter most to them.Â
In this third article, we turn to one of the most decisive and controversial components of HTAs: economic evidence, which primarily compares the benefits of a treatment with its associated costs. Economic evidence is taken into account along with clinical evidence and other evidence such as ethical implications, social, organisational, and legal aspects.Â
The controversy around economic evidence lies in the fact that it requires establishing what is a reasonable amount to pay for a given benefit, which ultimately places a monetary value on a health benefit. This can be very uncomfortable and can feel unfair, since it’s ultimately people’s quality and length of life we are talking about. Nonetheless, decision-makers need to rely on this type of analysis when considering how to allocate the public budget used to fund healthcare services.Â
Under the new EU HTA regulation, all EU member states are expected to consider the same information in terms of clinical effectiveness and safety through the Joint Clinical Assessment (JCA), which aims to shorten the approval-to-access timeline.Â
The JCA is built around what is known as the PICO (Population, Intervention, Comparator, and Outcomes) framework. PICOs are the key elements that define what kind of information developers must provide when requesting an HTA evaluation. In practice, PICOs determine which patients will be assessed, what treatment will be evaluated, which existing options it will be compared to, and which outcomes, such as improvements in function or quality of life, will be measured.
Understanding PICOs is particularly important for the HD community because they directly influence the type of data regulators and evaluators, will consider and review to decide on access and reimbursement. Patient organizations and key opinion leaders must be prepared to provide their perspective on what matters to people and families living with HD, which helps ensure that the PICOs truly reflect.
In terms of accessibility, even if a therapy receives EMA approval, before it reaches patients, each country still needs to perform its own national HTA to decide whether the therapy should and can be reimbursed. These national HTAs aim to ensure that the therapy not only meets strong criteria for efficacy, but is also cost-effective within the country´s healthcare budget, evaluating if they can afford it, and if the demonstrated benefits are worth the cost.
The national HTAs include economic evaluations, which help assess whether a therapy offers good value for the investment it represents within the national healthcare system. These evaluations mainly focus on comparing costs with clinical outcomes. Still, the broader impact a therapy might have on patients, families, and the overall healthcare system is increasingly being recognised as part of the overall value.
The conclusions drawn from the results of these analyses directly impact how quickly and under what conditions families affected by HD would actually have access to the approved therapy. Because each country has a different budget and can incorporate different models to make reimbursement decisions, access can vary greatly from country to country.Â
There are two main types of economic evaluations often performed within the HTAs: cost-effectiveness analysis and cost-benefit analysis. Even though they sound very similar, they have some major differences.Â
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The cost-effectiveness analysis (CEA) is the most common approach, and it mainly compares the costs of a treatment with the health benefit it provides to the person using it. In simple terms, it intends to measure how much health is gained per euro spent.
To showcase the results of this analysis, authorities often use a measure that reflects both the quality and length of life added by a therapy, known as QALY (cost per quality-adjusted life year). If the cost per QALY remains below a certain level, it may be considered cost-effective.Â
This type of analysis brings us back to the controversial aspect we discussed earlier, since the predefined levels or thresholds may not always reflect the real impact a treatment can have on a patient’s life. This is why it is so important to advocate and raise our voice to express the difficulties experienced by the HD community and how a treatment may have an impact beyond the predefined thresholds.Â
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The cost-benefit analysis (CBA) goes a step further than the CEA and captures a broader perspective. This type of analysis translates both the costs and benefits into monetary terms. In practice, CBA takes into consideration the direct and indirect economic benefits of a therapy to determine if it outweighs the costs of implementation. In simple terms, it intends to measure euros gained or saved for euros spent.Â
This allows decision-makers to consider more profound aspects, such as reduced caregiver burden, if the patient’s productivity or independence has improved, and if long-term healthcare costs, such as the need to provide institutional care, have lowered.Â
There are different models used across European countries to perform HTAs. These models establish a framework that shapes whether, when, and how much weight the types of evidence (clinical, economic, and others) are given.Â
All countries consider some form of economic evidence within their HTA process. However, the weight of this evidence differs from country to country. In some systems, economic evidence is the key factor to decide whether a therapy should be reimbursed, while in others, they offer support for price negotiations.
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In this model, both clinical and economic analyses are performed, and other aspects may also be evaluated. Nonetheless, to determine whether a therapy is reimbursed or not, economic evaluations such as the CEA and CBA are central.
In countries where this model is used, such as in Ireland and the Netherlands, decision makers mainly focus on whether a therapy offers good value for money. If the cost per QALY gained is below a certain level, the therapy is usually considered cost-effective and more likely to be reimbursed. In some countries, these levels can vary and may be adjusted for therapies aimed at rare diseases.Â
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In this model, clinical benefit, which examines whether a therapy offers meaningful improvement, is assessed first and considered the key factor.
In countries where this model is used, such as in France and Germany, economic evaluations are used as supporting evidence when it comes to price discussions and budget allocation. In general terms, economic evidence serves as a guide to decide on pricing rather than reimbursement eligibility.Â
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In this model, which is an emerging one, elements from both economics and clinical effectiveness are considered.Â
In countries where this model has been used, therapies are evaluated more holistically, considering equally important factors such as quality of life, disease severity, social aspects, budget impact, and other relevant aspects. This model is particularly beneficial for rare diseases, where traditional models may not fully capture patient and caregiver impact.Â
Even though these models aim to help healthcare systems allocate their resources in the best way possible, numbers and data alone can’t define value.Â
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For people living with HD, a therapy that prolongs independence, allows them to read a book, reduces caregiver burden, or enhances cognition in any way can be truly impactful, even if those benefits are difficult to measure in economic terms.Â
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We look forward to EU countries implementing models that combine rigorous evaluations with patient input and real-world evidence, where decisions are both economically sustainable and patient-centered.Â
To advocate effectively, HD organizations across Europe need to understand how reimbursement decisions are made in their country, what type of model is being used, how economic evaluations influence decisions, as well as when and how patient input can be incorporated. Being aware of these aspects and prepared for discussions helps ensure that what truly matters to the HD community is considered when the value of a new therapy is defined.Â
– Article written by Jarelys López Delgado