Position papers

Biotech products and HTA

‘One size fits all’ approach to HTA appraisals inadequate for complex biotech medicines.

This paper intends to outline key aspects related to innovative, cutting-edge biotech products in the context of Health Technology Assessment (HTA).


Healthcare biotechnology has a tremendous impact on meeting the needs of patients and their families – it not only encompasses medicines that are manufactured using a biotechnological process, but also gene and cell therapies and tissue engineered products.

Among others, biotech therapies form the core of many Orphan Medicinal Products (OMPs), Personalised Medicine and Advance Therapy Medicinal Products (ATMPs).

EuropaBio believes that a ‘one size fits all’ approach to the content, timing and procedure of HTA appraisals may fail to take account of the complexity of biotech medicines and ignore medicines’ particular benefits to individuals or selected groups. When assessing biotech medicines and integrated technologies, real-life relative effectiveness data can be instrumental in supporting added value and addressing key uncertainties. We believe that HTA evaluation processes should become more predictable, contributing to reducing uncertainty and improving efficiency of R&D investments. HTA of biotech drugs should therefore be an open, continuous process, leading to early dialogue; early access decisions and continuously adapting recommendations and rewards in light of newly developed evidences.

The understanding of HTA principles and methodologies can vary greatly depending on the size of companies. There are a number of potential hurdles faced by biotechnology small and medium-sizedenterprises (SMEs) when dealing with HTA requirements including relatively limited internal HTAskills and expertise compared to larger companies. Other challenges are related to the often inconsistent data requirements from different national or regional HTA agencies. Therefore, there should be sustained efforts to facilitate a greater joint dialogue between SMEs and HTA authorities at both European and national level.

In parallel, there should be an emphasis on encouraging more effective discussions between the EMA and national HTA authorities during early dialogue and scientific advice activities. There is a need for close interactions between clinical trial sponsors and HTA bodies to avoid unnecessary duplication of clinical trials for the benefit of patients and society.


HTA in assessing Orphan Medicinal Products (OMPs)

OMPs are an important component of the healthcare biotechnology. They are intended for the diagnosis, prevention or treatment of life-threatening or seriously debilitating conditions that are rare. OMPs often represent the sole therapeutic option available for patients living with rare diseases. Nonetheless, treatments for rare diseases will unfortunately often receive an unfavourable opinion when assessed through some standard HTA procedures in specific settings or jurisdictions, such as those using cost-effectiveness analysis as main criteria to inform decision making. Current methodologies for the HTA of medicines that treat more common diseases are in general based on criteria such as burden of illness (severity of the disease + unmet medical need), the relative clinical efficacy and safety (when comparing with “standard of care”) and even in countries such as the UK,

NL and Sweden the cost-effectiveness of a new medicinal product is compared to other products in the same therapeutic field, where they exist.

OMPs usually target life-threatening diseases. The clinical benefit/risk ratio may therefore be very positive, while research & development (R&D) costs are high. Higher acquisition costs raise cost-effectiveness ratios for orphan drugs. Consequently, orphan drugs are often unable to meet standard cost-effectiveness thresholds in HTA. In addition, due to the rarity of the condition, the amount and type of data needed to conduct useful HTA is usually unavailable at the time of marketing authorisation for OMPs. Furthermore, conventional economic evaluations used by some HTA agencies often do not capture the societal value of OMPs.

Against this background, we believe that HTA bodies should broaden the range of criteria they consider in assessments of OMPs, including qualitative data from patients reported outcomes (PROs) and a framework for systematic consideration of ethical and social issues in the appraisal process.


HTA in assessing Advanced Therapy Medicinal Products (ATMPs)

ATMPs represent a category of human medicines which are complicated to develop. They include gene therapy, cell therapy, and tissue engineered products.

The complexity associated with developing these products, combined with the rarity of some of the targeted conditions, requires an adapted HTA approach that can evaluate the impact on global health and societal impact of such novel medicines as they approach the market. We support adoption of a consistent and predictable, yet flexible approach towards conducting HTA for ATMPs across Europe. This flexible approach would ideally guarantee early access to ATMP products, while allowing for real-life evidence development. Therefore, we welcome any pan-European HTA/Regulatory initiatives favoring early dialogue and adapted approaches to assess ATMPs, including conditional access initiatives.


HTA in assessing Personalised Medicine

As personalised medicine is making its transition from theory to reality, it will inevitably have a large impact on the type of treatments being developed.

In essence, as the concept of personalised medicine revolves around the notion of pre-selecting patients who are likely to respond most favourably to the treatment offered to them, that treatment would naturally result in better health outcomes and improve efficacy, safety and overall public health. This would be a marked shift from the notion of ‘one size fits all’ towards a more personalised philosophy of ‘the right treatment for the right patient.’

HTA recommendations and/or decisions are traditionally made after marketing authorization. However, personalised medicines/treatment may not yet have shown their full value so soon after market authorization. We support close interactions between clinical trial sponsors and HTA bodies with the hope that such early dialogue will lead to more timely and straightforward assessments, resulting in appropriate access for patients in need.

Studies on the cost-effectiveness of personalised medicines are still on the way but promising results are available. Personalised medicine could provide more value for money because of improved drug effectiveness and reduced toxicity, and it could also decrease the average research and development costs for new medicines. But these efficiency gains may show only in the long run.



It is important that Europe’s methodologies for conducting HTA take into account the innovations and developments that biotechnology is pioneering and championing.

All stakeholders (HTA agencies, regulators, product developers, clinicians, and patients) need to cooperate to create a coordinated, transparent approach to the evaluation of biotech therapies. We support the fostering of European early dialogue programs involving relevant decision makers and stakeholders as well as the development of innovative HTA approaches adapted to OMPs, ATMPs and Personalised Medicine. We support close interactions between clinical trial sponsors and HTA bodies to avoid unnecessary duplication of clinical trials for the benefit of patients and society.


About EuropaBio

EuropaBio is the European Association for Bioindustries. Our membership is composed of 55 corporate members, 15 associate members and Bio regions, and 17 national biotechnology associations, who in turn represent more than 1800 small and medium sized biotech companies in Europe. Members of EuropaBio are involved in research, development, testing, manufacturing and commercialisation of biotechnology products and processes. EuropaBio’s mission is to promote an innovative and dynamic bio-based economy in Europe.

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