Broadstreet takes of a broad variety of work in the health economics and outcomes space. We thought we would share a few of our recent publications which reflect that diversity. A substantial part of what we do is focused on understanding how best to measure patient outcomes. Ensuring measures chosen have the sensitivity and specificity to accurately account for treatment effect promotes better outcomes for patients. In that line, Broadstreet team members Basia Rogula and Lauren Powell have collaborated with colleagues at Biohaven Pharmaceuticals and Pentara Corp.to publish their recent work on the use of composite scales to measure disease progression. Their article “Development of a General Composite Scale (GENCOMS) for Progressive Neurodegenerative Diseases and Implications for the Assessment of Disease‑Modifying Therapies” was published in Neurology and Therapy.
With neurogenerative diseases, it is difficult to reliably assess disease progression and thus the effectiveness of disease modifying therapies. Traditional outcome assessments for neurodegenerative disease tend to be cross-sectional and focused on one specific aspect of the disease while composite scales, which combine items from several scales and can focus on the most progressive aspects of the disease, are well-suited to detect what can be small changes in early stages of progressive diseases. Our study sought to describe a generalized framework for developing composite scales with heightened sensitivity using partial least squares regression. This technique is recommended in situations where the number of explanatory variables is high, and the variables are correlated.
For the creation of composite scales, the objective selection and weighting items from previously validated measures based solely on their sensitivity to disease progression allows for more responsive measurement of clinical decline. These composite measures can also be made disease-stage specific, attuned to capturing disease progression at a certain stage of disease by including items that are generally worsening at that stage. The GENCOMS framework will be helpful for complex diseases because combining all aspects of disease into a single composite makes both the effectiveness of good treatments and the ineffectiveness of bad treatments clear. It may also be particularly valuable in rare diseases with various potential outcomes that are less well understood, therefore mitigating the risk of missing key aspects of the disease. An example of its use in a specific disease can be seen in the team’s earlier publication “Development and Validation of SCACOMS, a Composite Scale for Assessing Disease Progression and Treatment Effects in Spinocerebellar Ataxia” in The Cerebellum.
Another large part of our work is in evidence synthesis and can encompass everything from clinical trial data to real world evidence from chart reviews or insurance claim data, in rare disease to large-scale public health issues. Our recently published study exploring the costs related to obesity “Costs of obesity, obesity-related complications, and weight loss in the United States: A systematic literature review” is a good example. Published in the Journal of Managed Care and Specialty Pharmacy team members Alison Bjornson, Megan Gardner and Shelagh Szabo in collaboration with colleagues at Boehringer Ingelheim conducted a systematic literature review to explore estimates of direct medical costs among people living with obesity, focusing on differing obesity severity and the presence of obesity-related complications. We also looked at the impact of weight loss on direct medical cost savings. With one hundred million American adults living with obesity and most experiencing obesity-related complications, obesity is a significant public health concern. Our review showed that relative to individuals with a normal weight, costs were up to 3.3-fold higher among those in the highest obesity severity group and were up to 5-fold greater when both type 2 diabetes and hypertension were present. Meanwhile, substantial savings were associated with 5% weight loss over 1 year. Despite the importance of this issue, our review highlighted the need for greater evidence on the impact of recently approved obesity medications as their impacts on costs remain largely uninvestigated. Additionally, longitudinal studies that consider a wider range and overlap in obesity-related complications are needed.
A third area of our work focuses on understanding people’s preferences as they relate to healthcare. Team members Alexis Mickle, Karissa Johnston and Jessica Dunne, in collaboration with colleagues at Genentech, The University of Chicago Medicine, Alameda Health System, Acaster Lloyd Consulting Ltd, and Northwestern Medicine recently published two complementary studies exploring the preferences of the general public and patients related to preventative treatment for food allergies. Using discrete choice experiments, a technique that facilitates the identification of people’s preferences through a series of indirect survey questions, both studies sought to understand teenage, adult, and caregiver preferences for attributes for treatments intended to prevent exposure-induced severe reactions. Additionally, we examined how preferences vary by clinical and demographic factors, tolerance to risk, and how much food allergies impact daily life (patients only). In general, respondents showed preferences for treatments that are more efficacious, are less likely to trigger an allergic reaction, and are convenient (i.e. are taken orally [vs. injection], at-home [vs. in a clinic], and less frequently [every two or four weeks vs. daily). Both papers, “Patient preferences for food allergy treatments in the United States: a discrete choice experiment“, and “US general population food allergy treatment preferences: a discrete choice experiment” were published in Current Medical Research and Opinion.
