#Policy: Would Evidence of Patient Preferences Have Changed the Decision on Lecanemab?

The decision of the European Medicines Agency (EMA) to reject the Alzheimer's drug Lecanemab has sparked significant discussion in the medical and political arenas. While the U.S. Food and Drug Administration (FDA) has approved the drug, the EMA decided against it, despite both agencies having access to the same evidence.

Background: Lecanemab and Its Effectiveness

Lecanemab is an antibody that targets the build-up of beta-amyloid proteins in the brain, which are believed to be a major cause of Alzheimer’s. Clinical studies have shown that the drug can slow cognitive decline in Alzheimer’s patients by approximately 27%. However, some patients experienced side effects such as brain swelling and bleeding.

Balancing Benefits and Risks

The EMA had to weigh whether the benefits of Lecanemab outweighed its risks. Although the therapeutic effect was measurable, some experts doubted whether patients would notice a significant difference in their daily lives. Simultaneously, the side effects, especially rare but severe brain hemorrhages, presented a substantial risk.

Consideration of Patient Preferences

A central question in this discussion is how patient preferences were factored into the decision-making process. We know that Lecanemab is effective against amyloid in the brain and that it has side effects ranging from brain swelling to milder reactions. What decision-makers do not know is what preferences patients have.

Interestingly, the FDA approved the drug while the EMA did not, even though both had the same evidence. This highlights that the decisions likely relied heavily on the value judgments of the decision-makers. It is unclear whether these judgments were based on the preferences of the patients for whom these drugs are intended.

Are the authorities trying to protect patients from side effects that these patients might be willing to accept to delay the progression of Alzheimer’s? Or is the EMA correct in assuming that the patients would not tolerate these side effects? This question remains unanswered and is not sufficiently addressed in the decision-making processes.

Human-Centered Research, a field that examines the interaction between patients and medications by focusing on their needs, preferences, and experiences, plays a crucial role here. An uninformed decision, whether for or against the drug, can result in a Type I error (rejecting an effective drug) or a Type II error (approving an ineffective or harmful drug).

Political and Economic Implications

The different evaluations by the FDA and EMA may also have political and economic implications. While the FDA’s approval allows patients in the U.S. to access the drug, it remains unavailable in the EU. This situation could lead to a two-tiered healthcare system, where wealthier patients travel to the U.S. for treatment.

Future Outlook and Conclusion

The EMA’s decision might be a setback for Alzheimer’s patients in Europe, but it does not mark the end of the fight against the disease. It is likely that more data will be collected, and future approval attempts will be made. Hoppefully patient prefernce information (PPI) will be used to make patient-oriented deciosions in the future.

Ultimately, the question remains whether patient preferences were adequately considered in the decision-making process. A more patient-centered approach could potentially lead to different outcomes. Balancing benefits and risks is crucial to ensuring informed and patient-centered healthcare decisions. Let’s hope this was not merely an economic decision influenced by the treatment costs of Lecanemab.