BACK of the BOOK
Heard in the Blogosphere
Gene Therapy With
a Money Back
Guarantee?
The BMJ
@TheBMJ
“Time pressures can force doctors to treat rather
than counsel, and to investigate rather than
reassure”
In Europe, a new gene therapy
for treating severe combined
immunodeficiency, commonly
known as “bubble boy disease,”
is being offered with a moneyback guarantee. If the ex vivo
stem cell therapy Strimvelis
(consisting of autologous CD34+
cells transduced to express ADA)
fails to cure the disease, a patient
will be refunded in full, the company
says. Doctors and a spokesperson from
the drug’s manufacturer spoke with CNN
about the new pricing strategy.
“In my opinion, the main benefit is the control of
appropriateness. With the warranty in place, it’s likely
that only those patients most likely to benefit will be
prescribed this highly individualized treatment. … Additionally, if the performance of the drug slips over time,
the drug’s cost-effectiveness will be renegotiated.”
—Prof. Luca Pani, director general of the Italian Medicines
Agency, which negotiated the price with GlaxoSmithKline
Hal Burstein, MD
@DrHBurstein
I would think about this a bit more but I’m
running late in clinic.
Kathi Apostolidis
@kgapo
The three As in #mhealth: Affordability,
Accessibility, Awareness
“In pricing Strimvelis, GlaxoSmithKline reviewed how
best to make the one-time treatment cost for the
medicine affordable to health care systems. Given the
extremely low patient numbers for Strimvelis and therefore low budget impact, many payers have indicated
that Strimvelis is likely to be funded on a case-by-case
basis and does not warrant a potentially complicated
pricing arrangement.”
—Sarah Spencer, a spokeswoman for GlaxoSmithKline
“It is expensive, but the other options are a bone marrow
transplant, which is equally if not more expensive, or
long-term enzyme replacement.”
—Helen Heslop, MD, DSc, director of the Center for Cell and Gene
Therapy at the Baylor College of Medicine
ASH
@ASH_Hematology
Submit a paper today to
Blood Advances and get
published in ASH’s new
open-access journal,
launching at #ASH16
bloodadvances.org.
Confronting Stem Cell Hype: Against Hyperbole,
Distortion, and Overselling
“Enthusiasm and optimistic speculation are natural parts of research and innovation and can be a constructive force
that helps to attract funding and to build research communities. … Stem cell research has generated much hyperbolic
media attention, which moves away from discussions of ethical concerns and toward positive portrayals of potential
therapeutic applications. This raises the risk of harmful consequences, including misleading the public [and] creating
unrealistic expectations. Because the forces that twist how science is communicated are complex, systemic, and interrelated, correcting for science hype will not be easy and will require the simultaneous use of various policy tools and
vigilant self-control. Hype is not inevitable.”
—Timothy Caulfield, Douglas Sipp; Charles E. Murry, MD, PhD; George Q. Daley, MD, PhD; and Jonathan Kimmelman, PhD,
discussing the International Society for Stem Cell Research’s new guidelines for “translating” stem cell research, in Science
Steve Gschmeissner/Getty Images
Let’s Cure Cancer, Not Just Turn It Into a Chronic
Disease
“Cancer survivors shouldn’t be content with living under the shadow of their disease and the potential for recurrence.
… Moving from survivorship to cure will take a concerted effort. The cancer community can’t afford to mimic the way
lawmakers do business, so it will take focused resolve to turn Vice President Joe Biden’s cancer moonshot from dream
to reality. Researchers must be willing to share data. Cancer patients young and old must be given access to clinical
trials and encouraged to make their data available. Minority communities must not only be included in these trials but
be embraced in them. Pharmaceutical companies must forge partnerships with medical schools and cancer centers.
And research institutions must recruit the next cadre of premedical and medical students and excite Generation Y to
join the cancer research revolution.”
—June M. McKoy, MD, associate professor of medicine and preventive medicine and director of geriatric oncology
at Northwestern University Feinberg School of Medicine, in STAT News
68
ASH Clinical News
November 2016