CHMP Meeting Success: Providing Clinical Perspective to Overcome Major Objections
Gaining marketing authorization from the European Medicines Agency (EMA) is increasingly difficult when a highly influential clinical assessor has a strong negative opinion of the product’s data. For this global biopharmaceutical company, the rapporteur was challenging the clinical meaningfulness of their drug’s effect size in patients who were treatment-resistant disease and had no other therapeutic options.
The Committee for Medicinal Products for Human Use (CHMP) called a Scientific Advisory Group (SAG) meeting to have clinicians weigh in on the efficacy and safety of the drug. The problem was that the company had exhausted new ways to analyze their data to show which patients would benefit – and how meaningful that benefit would be. They contacted 3D to help them face the SAG and prepare for the daunting Oral Explanation to follow. The fate of their drug hung in the balance.
3D brainstormed with the company team and realized the task was not to regurgitate data. We had to help the SAG members understand the significant benefit for patients suffering from this rare disease. We knew the most effective way to achieve this was to hear it from the small group of experts who actually treated these patients. The team engaged two well-known and respected European clinicians to provide their unique clinical perspectives. The physicians drew upon their firsthand experience caring for these patients and explained the magnitude of the benefit and communicated their confidence in the ability of doctors to manage incremental safety risks. Their comments were reinforced by clinical data that conclusively showed that available therapies could not resolve their refractory disease.
The 3D team focused the presentation on a precise group of patients where the benefits outweighed the risks and left no ambiguity around patient selection. Strategically, 3D recommended that the company press the suggested indication during the Oral Explanation – knowing it was different from the rapporteur’s proposal. The strategy was to elicit questions from the member states to resolve any uncertainty about the target population.
The team was able to confidently overcome the rapporteur’s major objections through the combination of a credible, scientific story and the invaluable testimony of experienced clinicians. The SAG discussion and vote were favorable and took the safety objection off the table. This paved the way for an Oral Explanation focused on understanding the clinical relevance of the efficacy data. The CHMP adopted a positive opinion, and the resulting indication was a huge success for the company.
Even when the rapporteur’s major objections seem insurmountable, it is possible to win over the CHMP by credibly communicating the positive benefit-risk. When the disease is rare and esoteric, the committee needs to hear from the experts to understand the patient's experience and fully realize the drug’s benefit.