Identifying Value Drivers for a Novel Treatment Targeting Core Symptoms of Autism Spectrum Disorder

OBJECTIVES:

Autism Spectrum Disorder (ASD) is a highly heterogenous neurodevelopmental condition that affects communication skills, social interaction, learning skills, and behavior. There are currently no pharmacological treatments indicated for the core symptoms of ASD. Treatment often relies on patients’ access to behavioral therapy and the management of associated symptoms through off-label use of antipsychotics, SSRI's, and other medicines, which often come with significant adverse events. This research explores payer and physician perceptions of the unmet need in ASD and what stakeholders view as the most important value drivers for a potential novel treatment targeting core symptoms of ASD.

METHODS:

This research used a combination of primary and secondary research methods. A total of 35 Payers and 15 physicians were interviewed across US, DEU, FRA, CAN, and JPN to assess the remaining unmet need in ASD, evaluate current treatment options, and identify the value drivers for a novel treatment for the core symptoms of ASD.

RESULTS:

Stakeholders agreed there is a high unmet need driven primarily by the lack of treatment options targeting core symptoms. The most important value drivers for a novel treatment for ASD include: demonstrating clinically meaningful improvements in core symptoms, long-term durable outcomes (demonstrated through a clinical trial of 6-months or longer) in targeted patient groups given the heterogenous population, improving associated symptoms of ASD (such as anxiety and depression), and reducing polypharmacy for symptom management.

CONCLUSIONS:

There is a high disease burden for people living with ASD given the lack of novel treatments targeting core symptoms. With the growing prevalence of ASD across the globe, there is a remaining unmet need for effective treatments. If a manufacturer is looking to launch a novel ASD treatment, the most important factor to consider is long term improvement in core symptoms without compromising on safety or QoL outcomes.