Autism | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2015

Autism spectrum disorder (ASD) is a neurodevelopmental disorder characterized by core behavioral symptoms that include impaired social skills, communication deficits, and restricted, repetitive behaviors. The disorder is considered a single diagnostic category with varying degrees of severity, as updated in the fifth edition of the Diagnostic and Statistical Manual of Mental Disorders (DSM-5). Behavioral and developmental therapies are the cornerstones of ASD treatment regardless of severity, and many patients also receive drug therapy to help manage their noncore symptoms or comorbidities, such as attention deficit, hyperactivity, irritability, and aggression. According to interviewed physicians, among the most commonly prescribed drug therapies for irritability associated with ASD are risperidone (Janssen’s Risperdal, generics) and aripiprazole (Bristol-Myers Squibb/Otsuka Pharmaceutical’s Abilify, generics). Interviewed thought leaders agree that finding an effective treatment to address core behavioral symptoms is the greatest unmet need in ASD.

This report provides an overview of the ASD market including a comprehensive analysis of patient populations, current therapies and medical practice, and opportunities for emerging therapies. The findings described in this report are derived from detailed interviews with expert U.S. and European specialists, comprehensive secondary research, and best-in-class epidemiological analysis. This report provides deep insights into this complex and evolving clinical space, and includes a detailed analysis of specific opportunities for current and emerging therapies, including experts’ views on which unmet needs have not yet been addressed by developers of new therapies.

 Questions Answered:

The risk of developing ASD continues to grow. What is the size of the U.S. and European (France, Germany, Italy, Spain, and the United Kingdom) prevalent ASD population? How will population size change through 2024?

The pathogenesis of ASD involves multiple, complicated pathways. What new insights are informing experts’ understanding of ASD? What are the key avenues of ASD preclinical and clinical research and development efforts?

Behavioral and developmental therapies are the cornerstones of ASD management, while pharmacotherapies are used to alleviate noncore symptoms and mitigate specific behaviors. What is the shape of the current treatment landscape and what are common approaches in ASD medical practice? What are physicians’ opinions on the implications of the recent changes to ASD diagnostic criteria in the DSM-5?

Unmet needs in ASD span a range of challenging issues. What are the emerging ASD therapies in late-stage development and to what degree could they address the unmet need in ASD? What are the key primary and secondary clinical end points on which new therapies are evaluated and how do interviewed experts perceive these end points?


Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.

Primary research: Eight country-specific interviews with thought-leading ASD specialists.

Epidemiology: Prevalent cases of ASD.

Emerging Therapies: Phase III: 2; Phase II: 3.

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