July 17, 2025
I’m Dr. Diana Zuckerman, president of the National Center for Health Research. Thank you for
the opportunity to share my views today.
The National Center for Health Research is a nonprofit think tank that conducts, analyzes, and
scrutinizes research on a range of health issues, with a particular focus on the safety and
effectiveness of medical products. We do not accept funding from companies that make
products that are the subject of our work and therefore have no conflicts of interest.
My perspective is as a cancer survivor who in trained in epidemiology and public health and
held research positions at Yale and Harvard before coming to DC to work in the US Congress,
HHS, White House, and as the president of a research center. On a personal and professional
level, I understand the importance of the FDA and this Committee and I thank you for your
service.
In order to consider whether the benefits outweigh the risks of Blenrep, it’s important to ask
who was studied in the research you’re considering today.
- Fewer than 5% were U.S. patients (and why so few?) Thank you to Dr. Pazdur
for his comments on that issue today. - 5% were Black in one study, and none in the other. More important than the
percentage, there were only 12 Black patients, and that is much too few to
generalize whether the drug is safe or effective for them. The number of Asian
and Hispanic patients were also too small to conduct subgroup analyses or to
generalize. - Patients over 75 were also under-represented: 15% & 12% in the two studies.
That is fewer than half of the percentage that is typical for this disease.
Under these circumstances, would the treatment be approved for Whites only, or not
approved for Blacks? Nobody would want to do that.
There are other flaws in the studies
- The Comparator arm in DREAMM-8 is not an approved regimen in the U.S.
- We agree with the FDA that there are other, better treatment options for
multiple myeloma. And half the patients had only one previous treatment, and
those were not the most popular treatments available, which again indicates
that these patients are not generalizable to patients in the U.S. - Many patients lowered dosage due to poor tolerability of selected dosages – so
how can FDA approve an indication based on those dosages?
I’ve attended hundreds of FDA Advisory Committee meetings and I’ve never seen such
a serious side effect as ocular toxicity that affects most patients. As the FDA pointed
out, even blurred vision can be debilitating and risky. Unfortunately, we did not hear
from patients who were harmed by that adverse event.
- Ocular Toxicity is unique and serious
- May be asymptomatic at first, which means it can be more harmful because when it is finally diagnosed, it may not be reversible
- In real world, toxicity monitoring will not be as careful as in a clinical trial. And the rural patients who might most benefit from this treatment option would be the ones least likely to have access to careful monitoring by an ophthalmologist.
In conclusion, Blenrep has benefits, but are they enough to outweigh risks?
- The primary endpoint of progression free survival was met – but that is compared
to an unapproved treatment in DREAMM-8 and not compared to optimal
treatments in DREAMM-7. Since the unapproved treatment is not available,
we’re left with basically just one study indicating a benefit, not two. - Given the very clear risks, the small number of U.S. patients, the under-
representation of Blacks and older patients, and the availability of other effective
treatment options, what should patients be told of this drug? Can Black patients
or patients over 75 be adequately informed of the benefits and risks of this
treatment for them if this treatment is approved?