Will the Pediatric Rare Disease Pipeline Create a Financial Tsunami? Implications of a Monte Carlo Projection

Speaker(s)

Moderator: Jamie Sullivan, MPH, EveryLife Foundation For Rare Diseases, Washington, DC, USA
Panelists: Jessica Daw, PharmD, Sentara Health Plans, Virginia Beach, VA, USA; Colin Young, PhD, Center for Biomedical System Design, Tufts Medical Center, Bedford, MA, USA; Ryan Fischer, BA, Foundation for Angelman Syndrome Therapeutics, Marble Falls, TX, USA

ISSUE:

The pediatric-onset rare disease therapy boasts hundreds of candidates in the drug development pipeline. A detailed Monte Carlo simulation based on historic success rates suggests the pipeline will yield about 45 product-indications leading to an incremental $20B in list price drug spending in 2036 prior to any medical cost offsets. A cost surge in the mid-2020s due to sickle cell disease, Duchenne muscular dystrophy and hemophilia gene therapies will also likely occur. The panel will explore the implications of this success for the financial sustainability of US healthcare system participants.

OVERVIEW:

Jamie, Sullivan, panel moderator will provide an overview of the topic and key issues. (5 mins)

Dr. Colin Young will provide the results of the Monte Carlo simulation as well as summarize the model’s methods, data, and assumptions. (7 mins)

Jessica Daw will highlight that high-cost therapy financial concerns are warranted on a per patient, cost growth, and absolute amount. The challenges for small employers/insurers will also be highlighted. A balanced approach with transparent pricing and value-based arrangements is necessary. (4 mins)

Ryan Fischer will emphasize the patient impact likely from these therapies and that budget battles affect lives; affordability is essential for families. Collaborative solutions, from value-based pricing and payment models to patient-centric benefit designs, are vital to ease the financial strain on patients. (4 mins)

Audience polling before and between presenters will consume 10 minutes. The responses on priorities and mitigation approaches will influence the panelist remarks.

The remaining time will be divided between audience Q&A and pre-planned panelist questions if few audience questions occur.

The presentations aim to elucidate the following:

  • Justifiability of financial concerns
  • Opportunities for collaborative financial sustainability:
  • Balancing access, uncertainty mitigation, payer affordability and developer incentives.
Beyond payers, patients, and manufacturers, the session has applicability for policy makers, drug developers, HTA and similar professionals.

Code

108

Topic

Economic Evaluation