Early phase modelling establishes opportunities and feasibility of ATMP for rare genetic disorder

The Project

A project consortium was launching an EU-funded clinical trial for a first-in-class advanced therapeutic medicinal treatment (ATMP) for a rare genetic condition needed an economic assessment to understand the cost effectiveness of the product. The consortium comprised several hospitals across Europe and an expert team from PharmaLex to perform the health economic assessment.

The product being researched is an off-the-shelf fetal stem cell treatment. The clinical trial was conducted in several locations in Europe. While the first phase of the trial has concluded, trial data is still being gathered and patient follow-up will continue for another 2 years. Subsequent trials are also planned.

The Challenge

The Challenge

The primary challenge for the research partners was to understand the cost effectiveness of the product to determine whether it would be feasible to introduce as a standard treatment, assuming it achieves satisfactory clinical outcomes. The consortium also needed to understand the positioning of the treatment, based on clinical benefit as well as cost effectiveness in different subtypes of the disease.

The Solution

The Solution

PharmaLex developed a study protocol for the health economic assessment, based on trial endpoints, to describe cost effectiveness of the ATMP. Based on the assessment, the team could show what clinical effectiveness would be needed for cost effectiveness, and in which subtypes the treatment would be relevant as standard treatment. The team gathered resource use, cost and quality of life input from clinical experts within the trial as well as from literature to build the early phase health economic model.

The early phase modelling was based on fractures, a prominent feature of the disease. Patients with the disease often experience fractures prenatally; however, the disease has various subtypes and different levels of severity. While the early phase model was confined to fractures, a full health economic assessment can be refined with additional criteria, once data from the clinical trial program is in place.

The Outcome

The Outcome

The consortium held its last meeting in Stockholm in December 2022, during which the PharmaLex team presented the results from the early phase modelling. The team found that the ATMP would not be feasible to introduce as standard treatment for all types of the disease but would be cost effective if used to treat more severe forms of the disease.

The team noted that further substantiation will be needed to demonstrate the wider impact of the treatment, taking into account the costs for stem cell preparation and manufacturing and the cost of administering the stem cells weighed against the cost of treating fractures. One advantage the product has over many other ATMPs is that it is an off-the-shelf product and will therefore be less costly to produce than autologous products.

The PharmaLex team has recommended that during Phase 2 trials researchers collect quality of life data to further determine impact. Separately, the consortium is considering establishing a company

in order to attract investors to support further research and to create a commercial product that can be used outside of the confines of the consortium hospitals.

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