Securing Regulatory Agency Approval and Reimbursement

Project #2: Determining the validity of a measurement for treatment efficacy

Before a drug can receive approval from medical regulatory agencies such as the US Food and Drug Administration (FDA), its manufacturer must quantify the severity of the conditions the drug was developed to treat, using validated measurements. In the case of Humira, an Analysis Group team assessed the validity, responsiveness, and meaningfulness of the hidradenitis suppurativa clinical response (HiSCR) as the endpoint in clinical trials for treatment of hidradenitis suppurativa (HS), a rare condition involving painful bumps under the skin. HiSCR (defined as achieving a 50% or greater reduction in the number of symptomatic bumps) was validated to reflect treatment efficacy in HS disease control. Patients who achieved HiSCR had significant improvements in both disease severity and quality of life.

The measurement we validated was the key outcome used in Humira’s application to the FDA, and that application ultimately secured FDA approval for using Humira to treat HS. Analyses like this one have important policy implications, not just for a particular client but for all biopharmaceutical companies that Analysis Group supports throughout a drug’s life cycle.



Project Team Member Profile


Min Meet a Team profile

Min Yang

Associate (now Vice President), Boston

When we were engaged for this project, the HiSCR endpoint was a new clinical outcome. Existing clinical measures were inconvenient and difficult to use, so our client developed HiSCR to evaluate treatment outcomes. Since it was much more straightforward, we used Phase II trial data to demonstrate that it was a valid and meaningful endpoint. The analysis supported using HiSCR as the primary efficacy endpoint for Humira’s Phase III trial. The FDA and other regulatory agencies cited our publication, which had established HiSCR as a valid endpoint.

Several analysts made critical contributions – in fact, one analyst coauthored a publication about the project. As the project manager, I worked on strategy, trained analysts, conducted analyses, and communicated with our client about how best to demonstrate the endpoint’s validity. We also worked closely with Dr. Alexandra B. Kimball, MD, the CEO and president of Harvard Medical Faculty Physicians at Beth Israel Deaconess Medical Center.

Teamwork was important to the project’s success, as were keeping the big picture in mind and clearly articulating to the client why the rigor of the design mattered. This project was satisfying because junior consultants were involved in work that had a clear and meaningful impact on patients’ lives. We could see that the work wasn’t just about crunching numbers – we were providing an opportunity to improve patients’ quality of life.

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