FOR IMMEDIATE RELEASE
Doctors for America
PO Box 53313
1921 Florida Ave NW
Washington, DC 20009-9997
January 11, 2023
WASHINGTON, D.C. – In light of the recent approval of lecanemab (Leqembi) under the FDA’s Accelerated Approval pathway, Doctors For America’s FDA Task Force calls on the FDA to put patients first and commit to holding an Advisory Committee meeting before any decision is made on the full approval of the therapy.
The FDA Task Force is committed to increasing transparency at the FDA and ensuring patient safety is at the heart of all therapeutic approvals. The recent FDA decision to expeditiously approve lecanemab despite reported patients deaths associated with the drug illustrates why the FDA Task Force’s work is desperately needed. Questions also remain about whether the surrogate endpoint used as the basis for accelerated approval, beta amyloid, is indeed “reasonably predictive” of clinical benefit despite over two dozen studies not finding such an association. Moreover, FDA has yet to review the published confirmatory trial as part of the accelerated approval and it remains unclear whether the statistically significant changes seen among patients receiving the drug correspond with meaningful clinical benefit.
In light of the recent expedited approval and the sponsor’s subsequent application for traditional approval, the Doctors for America FDA Task Force is asking the FDA to convene a group of independent advisors for a formal advisory committee meeting to address the many unanswered questions surrounding this therapy. The FDA and its advisory committee should consider the following:
- whether the surrogate endpoint used as the basis of approval is indeed reasonably predictive of clinical benefit;
- if the confirmatory trial results from Clarity-AD showing changes in cognitive scores are indeed clinically meaningful;
- if the drug does show evidence of harm that outweigh its benefits in light of the deaths reported;
- whether the clinical trials have adequate representation of patients who would be prescribed this treatment in the real-world including older adults with comorbidities and communities of color disproportionately impacted by the disease; and
- if FDA took steps in its review of lecanemab to address irregularities in its approval of aducanumab including managing conflicts of interests.
Patient safety is the primary concern of the FDA Task Force. The approval of lecanemab is based on a secondary, unvalidated surrogate endpoint, but critical questions remain as to whether the benefits outweigh the risks reported of brain swelling, brain bleeding, and death among patients who received this drug. As a group of physicians, the FDA Task Force understands the difficult challenge clinicians across the country will face when considering prescribing this treatment. As physicians, we vow to “do no harm” in treating our patients and look to the FDA to provide unbiased, independent guidance to signal through their approval decisions that the authorized treatment’s benefits truly outweigh its risks. Like our patients, we eagerly await a future when there is a clinically meaningful and safe treatment for this debilitating, progressive neurological disease. However, ambition must not cloud our or the FDA’s judgment of the data submitted – doing so may put patients in harm’s way.
Lecanemab’s approval comes just weeks after the House Committee on Oversight and the House Energy and Commerce Committee released an investigative report on “The High Price of Aduhelm’s Approval” and its atypical review process. The Task Force is grateful for the oversight work being done by congressional leaders, and FDA must recognize the public need for greater transparency and scrutiny around its dealings with pharmaceutical companies and interest groups. Despite widespread concern over the approval of aducanumab (Aduhelm) we are frustrated to see that no apparent changes have been made in the review and approval process. Instead, FDA within their approval documents for lecanemab has repeatedly referenced aducanumab as a precedent for this approval, misleadingly stating that it is settled science despite overwhelming evidence otherwise that an unvalidated surrogate endpoint can be used as the basis of expedited review and approval. Patients and doctors across the country want and need a cure for Alzheimer’s disease, not a false hope with a twenty-six thousand dollar price tag.
Last week, we joined other patient, public health, and physician groups in sending a letter to the Centers for Medicare and Medicaid Services (CMS) Administrator Brooks-LaSure urging that CMS uphold its sound National Coverage Determination (NCD) for FDA-approved monoclonal antibodies directed against amyloid for the treatment of Alzheimer’s disease such as aducanumab and lecanemab. Under this policy, Medicare coverage for such therapies approved by the FDA based on a surrogate endpoint would appropriately only be available for patients enrolled in clinical trials, allowing for important data to be collected on clinical efficacy and safety. As lecanemab received accelerated approval by the FDA based on a surrogate endpoint and FDA has yet to complete its review of additional confirmatory clinical trial data, we agree with the NCD in providing coverage only through clinical trials and thus, protecting patients.
For press inquiries and to speak more on this issue with members of the Doctors for America FDA Task Force please contact Claire Onyechi with Continuum Health Group ([email protected]).
About Doctors for America:
Doctors for America mobilizes doctors and medical students to be leaders in putting patients over politics to improve the health of our patients, communities, and nation. We are 27,000 physicians and medical students in all 50 states, representing all areas of specialization. Our impact areas focus on access to affordable care, community health and prevention, and health justice and equity. DFA focuses solely on what is best for our patients, not on the business side of medicine, and does not accept any funding from pharmaceutical or medical device companies; which uniquely positions DFA as the organization that puts patients over politics and patients over profits. Find out more at doctorsforamerica.org and on Twitter @drsforamerica.