What are advanced therapies?

Advanced therapy medicinal products (ATMPs) are transforming healthcare through providing patients with potentially curative treatment options. ATMPs include gene therapies, cell and tissue-based therapies and combined ATMPs, as defined by the European Medicines Agency (EMA)¹:

“gene therapy medicines: these contain genes that lead to a therapeutic, prophylactic or diagnostic effect”;

“somatic-cell therapy medicines: these contain cells or tissues that have been manipulated to change their biological characteristics or cells or tissues not intended to be used for the same essential functions in the body”;

“tissue-engineered medicines: these contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissue”

A fourth class, combined ATMPs, is defined as one of the aforementioned ATMPs with a medical device as an integral component.

The first ATMP was approved by the EMA in 2015. Although non-ATMP therapies have also provided patients with immense benefits, the sheer contrast in terms of the development, manufacture and impact of ATMPs means that these therapies can be considered unique. With regards to the impact of ATMPs, their high cost coupled with their potentially curative and transformative nature means that it is important to understand their value. This is made even more important given that the number of ATMPs is expected to grow 16.8% by 2030.^2.

What is value assessment and why is it important? 

In England, all health interventions, including ATMPs, are subject to value assessment by the National Institute of Health and Care Excellence (NICE). This informs decision making in healthcare, i.e., whether or not the intervention will be provided to patients by the healthcare system. NICE assesses the value of health interventions using the cost per quality-adjusted life year (QALY)³. The QALY considers the length and quality of life, where the quality of life is measured on a scale from 0 to 1. It is important to note that NICE does consider additional value elements in its decision making but does not quantify them resulting in what could be called a narrow value assessment framework. That is to say, the assessment of value is largely limited to one metric formed of two parts (length and quality of life) with additional elements of value which may be important to patients being irrelevant. These additional value elements may include providing patients with a treatment alternative or reducing the impact on family members.

As a result, experts have raised concerns around the appropriateness and relevance of existing value assessment methods for assessing ATMPs with researchers arguing that there is a misalignment between the value of advanced therapies and the value assessment used by NICE⁴ with key stakeholders in this space calling for increased understanding of the value of these therapies⁵.

The misalignment leads to unfair decision making in healthcare and limits patient access to these innovative and potentially curative therapies. 

How could patient access to advanced therapies be improved?

Given the misalignment between current value assessment and the value advanced therapies are deemed to possess, a key area for research is to identify the value elements of advanced therapies. To identify these value elements a multi-faceted and comprehensive approach should be undertaken.

The starting point, as with all research projects, would be a literature review to identify available evidence on the value elements of advanced therapies from all perspectives For example, patient/carers, clinicians, manufacturers, reimbursement bodies such as NICE as this would ensure that no value elements are missed. The literature review would be followed by qualitative research in the form of individual interviews and focus groups with the same stakeholders to capture elements of value which may not be present in the literature. The qualitative research would also enable validation of elements of value which were identified in the published literature.

Once the value elements of advanced therapies have been identified, discussions around how the findings could be used by NICE to improve their value assessment of advanced therapies should be undertaken. These discussions would include academics, healthcare system representatives, policy makers and, of course, NICE. Discussion points would include the incorporation of additional elements of value, as necessary, and whether this would require changes to the current value assessment processes. 

Ultimately, this would lead to fairer decision making by NICE thus improving patient access to these innovative treatments.

¹European Medicines Agency. Advanced therapy medicinal products:  https://www.ema.europa.eu/en/human-regulatory-overview/advanced-therapy-medicinal-products-overview

²Grand View Research, Advanced therapy medicinal products market size, share & trends analysis report by therapy type (cell therapy, gene therapy, tissue engineered product), by region (North America, Europe, APAC, ROW), and segment forecasts, 2023 – 2030

³NICE health technology evaluations: the manual. 2023: https://www.nice.org.uk/process/pmg36/chapter/introduction-to-health-technology-evaluation

⁴Jonsson B et al., 2019. Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare. European Journal of health Economics; 20: 427-38. 

⁵Advanced therapy treatment centres. 2023. Advanced Therapy Adoption Challenges in the United Kingdom: https://www.theattcnetwork.co.uk/wp-content/uploads/2023/12/WEBSITE-COPY-Advanced-Therapy-Adoption-Challenges-in-the-United-Kingdom-1.pdf