Study: Fewer than 1% of geriatric patients with cognitive complaints met Aduhelm research trial criteria. What can we expect about its real-world safety?

Bio­gen’s Aduhelm label far exceeds clin­i­cal tri­al pop­u­la­tion, study says. That could bring real-world sur­pris­es (Fierce Pharma):

While the chat­ter sur­round­ing Biogen’s con­tro­ver­sial Alzheimer’s med Aduhelm has large­ly been cen­tered on a piv­otal Medicare reim­burse­ment deci­sion as of late, ana­lysts are point­ing to one new study that sug­gests there may be “room for sur­pris­es” when it comes to the treatment’s safety.

In a note to clients on Wednes­day, RBC Cap­i­tal Mar­kets ana­lysts high­light­ed a recent study that applied Biogen’s pub­licly dis­closed clin­i­cal tri­al inclu­sion cri­te­ria for Aduhelm to 911 patients in a geri­atric out­pa­tient unit at a hos­pi­tal in Milan, Italy.

Researchers deter­mined that only six patients, or less than 1% of those eval­u­at­ed, would have qual­i­fied for the treatment’s clin­i­cal trials—known as Emerge and Engage—ahead of the FDA’s his­toric approval in June, accord­ing to a let­ter pub­lished in the Jour­nal of the Amer­i­can Geri­atrics Soci­ety in late July.

To the RBC ana­lysts, this sug­gests that there’s room for pre­vi­ous­ly unde­tect­ed side effects for the infu­sion once it’s admin­is­tered to thou­sands of patients beyond those who were rep­re­sent­ed in the clin­i­cal tri­als, accord­ing to the note … This could affect Aduhelm’s “nar­ra­tive and ulti­mate launch curve, not to men­tion poten­tial reim­burse­ment breadth,” the ana­lysts said.

The Analysis:

“Real world” eli­gi­bil­i­ty for adu­canum­ab (Jour­nal of the Amer­i­can Geri­atrics Society):

To iden­ti­fy the pro­por­tion of patients who would be poten­tial­ly eli­gi­ble to receive adu­canum­ab in the “real world”, we sys­tem­at­i­cal­ly applied the cri­te­ria adopt­ed in the pre-approval research pro­to­cols to a rep­re­sen­ta­tive clin­i­cal pop­u­la­tion refer­ring to a geri­atric out­pa­tient unit of a ter­tiary uni­ver­si­ty hos­pi­tal in Milan (Italy). A total of 911 patients con­sec­u­tive­ly attend­ing the unit for cog­ni­tive com­plaints were con­sid­ered … only six patients (i.e., 0.66%) of the study pop­u­la­tion would poten­tial­ly be can­di­dates to adu­canum­ab … It is thus clear how the result­ing gap between the research and “real world” fields is mas­sive, exem­pli­fy­ing once again the “evi­dence-based med­i­cine issue” chal­leng­ing deci­sions in geri­atric care.

News in Context:

• Vet­er­ans Affairs won’t cov­er Biogen’s new “Alzheimer’s drug” giv­en con­cerns over safe­ty and lack of evidence
• Alzheimer’s & Demen­tia researchers chal­lenge FDA’s approval of Aduhelm giv­en lack of evi­dence for beta-amy­loid as a marker
• Can the con­tro­ver­sial FDA approval of Aduhelm back­fire and delay the dis­cov­ery of actu­al Alzheimer’s treat­ments? (Yes, it can)