A tailored approach to estimate the outlook of the gene therapy market 

Using epidemiological data, our patient-based forecasting model projects the gene therapy market outlook until 2036. Despite uncertainties, it emphasizes the importance of health system preparedness.

“Between 2022 and 2030, 36 new treatments are expected to reach the market with up to 9 in a single year, resulting in a 7-fold increase compared to “today”. Moreover, by 2030, an impressive number of 117,000 patients could be treated annually in France by a gene therapy.[1]”  Such are some of the main estimations of our model forecasting the economic impact of gene therapies, adapted to France, and presented at ISPOR 2022.

A patient-based forecasting model estimating the outlook of the gene therapy market until 2036

Gene therapies are revolutionizing healthcare by offering potentially curative therapeutic options for patients suffering from severe genetic conditions. The past decade has witnessed a significant increase in the approval of cellular and gene therapy products worldwide. Currently, the development pipeline has hundreds of gene therapies. Despite these results and the enormous market opportunity that this new sector presents, scientific and cost related challenges remain for those breakthrough therapies.

While multiple market reports suggest an imminent gene therapy “wall” threatening the sustainability of healthcare budgets, the reality is more nuanced. Those market-based analyses often suggest that the market is poised to grow by a third annually, but actual growth could turn out to be more complex with potential plateaus. Indeed, despite the dynamism of the clinical trial landscape, only a handful of those innovative medicines hit the market. This raises awareness of the fact epidemiological, clinical, economical, and technological parameters will hamper the uptake of new gene therapies. As a result, there is a need for a most robust estimate of the true outlook of gene therapy with the necessary granularity to offer a deeper understanding. We proposed a different approach forecasting the gene therapy market through the patients. In comparison with the market-based route, the patient-based approach follows a bottom-up methodology with epidemiological data as a starting point. Thanks to this process the results of the model are not biased by the lack of historical data.

Market based approach

Patient based approach

The forecasting model identifies and details all gene therapies marketed or in development worldwide over the period 2022-2036. To assess their impact on a given pharmaceutical national budget, some assumptions have been made on the timing of the first commercial patient, the probability of success, price estimates, and penetration rates (reducing one last time the number of patients).  Here we present the results of the model’s application to France, however, this economic model can be applied to any country to forecast the number of patients and the budget impact estimate.

  1. The first step was to extract data from over 800 clinical trials in development. The epidemiological data2 but also the details of each technology used
  2. We kept only one asset per disease to avoid duplicates 
  3. We refined the epidemiological data by calculating specific subpopulations for each asset (e.g., specific genetic mutation excluding 50% of the patients)
  4. Thanks to this granular data base we build the model and several scenarios avoiding most of the uncertainties 

A granular forecast of the gene therapy market can educate on the sustainability concern

Whilst there remain few commercialised gene therapies, they are already showing encouraging results in saving or enhancing patient lives. Despite the wave of excitement and expectation, there are concerns around whether these high prices are sustainable for the healthcare system.

Healthcare system sustainability is a global concern and remains a priority in mature economies. Public resources were already constrained in many geographies, which has been exacerbated by unexpected events such as the COVID-19 pandemic. This has exercised unprecedented pressure on healthcare systems. Many public systems face significant funding challenges because of stalled economies, diminishing tax revenues, and increasing budget deficits. Additionally, as the population in Western economies ages, medical care consumption has increased exponentially, driven by the shift towards personalized healthcare.

A substantial proportion of existing and upcoming therapies are characterized by a one-time administration. This results in a fundamental change in treatment paradigm, through a decrease in scarce healthcare resources and simplification of the patient’s care process. On the other hand, it partially explains the soaring prices of these drugs

The results of our model brings sustainability messages related to stakeholders in charge of public health resources.

Despite some uncertainties the model’s outcomes raise awareness on the necessity to be prepared to face the gene therapy development

The results of this model are subject to uncertainties, as prevalence data are often imprecise for rare diseases. Moreover, the estimation of the duration of clinical trials is also uncertain, in the same way as the price estimates. To address these issues the model proposes different scenarios with low and high estimates of these values. The next few years may be marked by scientific innovation, which, if managed and studied in advance, could significantly benefit patients without threatening national budgets. It will be the opportunity to update the model and refine the inputs to send the clearest possible message.

This forecast model has been co-created with Pfizer France.

For more information, we kindly invite you to consult our poster published at Ispor 2023 or contact us at

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