Ensuring Access And Approval For Innovative Therapies In Scleroderma

By Michael F. Murphy, Md, Ph.D., Chief Medical And Scientific Officer, Melissa Vadnais, VMD, PH.D., Fellow, Clinical Research Methodology, And Andrew Kuhlman, Medical Writer

ENSURING ACCESS AS WELL AS APPROVAL

Clinical research and development programs for innovative therapy must accommodate the diverse data needs of multiple stakeholders. Ensuring regulatory approval and market authorization will have primacy; however, creating a clinical trial database that facilitates adoption and patient access is part of the mandate.

Design and operational approaches that enhance product value require planning and preemptive problem-solving. It begins with a systematic review of potential barriers to formulary placement and reimbursement to inform program design and concludes with practical suggestions for trial design, study placement, and patient outcomes (as opposed to study measures) that may facilitate favorable review. The strategic imperative is to create an integrated program capable of demonstrating the “value” of a new product, as well as clinical utility and therapeutic novelty.

This white paper explores the requirements to create an integrated program capable of demonstrating the “value” of a new product, as well as clinical utility and therapeutic novelty.