[Ip-health] Pharmalot: As families in Europe clamor for its pricey gene therapy, Novartis creates a lottery

Thiru Balasubramaniam thiru at keionline.org
Fri Dec 20 07:09:54 PST 2019


As families in Europe clamor for its pricey gene therapy, Novartis creates
a lottery


DECEMBER 19, 2019

Seeking to provide its pricey gene therapy to patients in countries where
the drug is not yet approved, Novartis (NVS) has taken the unusual step of
creating a lottery, a move that drew criticism from patient groups over
concerns the approach would unfairly forsake some babies.

At issue is access to Zolgensma, which costs $2.1 million per patient and
is used to combat spinal muscular atrophy, a rare inherited disease that
afflicts children up to 2 years old and often leads to death. The treatment
is available in the U.S., but not yet in Europe or Japan, where approval is
expected next year. Meanwhile, parents have been clamoring for the medicine.

In recent months, stories have popped up in countries such as Belgium and
Hungary about families using crowdfunding to pay for the unapproved
treatment.  Novartis may have worsened their frustration by countering that
supplying Zolgensma through compassionate use programs was not an option
under European Union laws because another treatment from Biogen (BIIB)
could be obtained.

The ensuing ruckus has resulted in the lottery. The program begins next
month with 50 doses allocated for the first half of the year and up to 100
total doses planned for 2020. The company hopes to make this a “long-term
commitment,” with additional doses becoming available on a rolling
six-month basis, contingent upon patient need and manufacturing capacity.
Two plants are expected to open in 2020.

The AveXis unit at Novartis, which developed the gene therapy, “designed a
program anchored in principles of fairness, clinical need and global
accessibility to best determine the equitable global distribution of a
finite number of doses that doesn’t favor one child or country over
another,” a Novartis spokesman wrote us.

Related: Novartis was aware of manipulation of data supporting gene therapy
approval, FDA says

“Recognizing that the program will not be a solution for all families in
all countries, we understand the desire of parents to seek out the
treatment for their child and are working hard to increase supply and to
design sustainable solutions to further expand global access” to the
medicine. The company expects to discontinue the lottery in countries where
regulatory approval subsequently occurs.

The effort was met with mixed reaction from at least one patient advocacy
group. TreatSMA, which is based in the U.K., appeared to support the idea
of widening access, but was decidedly cool to creating a lottery.

“As much as we applaud AveXis’s initiative to offer Zolgensma globally at
no cost, given the lack of access to any SMA treatment in many places, we
are yet to be convinced that a health lottery is an appropriate way of
meeting the unmet medical needs in this severe disease,” the group said in
a statement on its website.

One bioethicist remarked that the decision to pursue a lottery for such a
life-saving treatment was a sorry commentary on the health care system and
the ongoing struggles to gain access to medicines.

“On the assumption that this is an effective therapy that kids with the
appropriate condition should have access to, the fact that the manufacturer
is lotterying it strikes me as capturing every injustice about health care
globally,” Dr. Steven Joffe, who heads the division of medical ethics at
the University of Pennsylvania, wrote us. “It just fesses up to the reality
of access to care in the most honest way I’ve ever seen.”

About the Author

Ed Silverman

Pharmalot Columnist, Senior Writer

Ed covers the pharmaceutical industry.

ed.silverman at statnews.com

Thiru Balasubramaniam
Geneva Representative
Knowledge Ecology International
41 22 791 6727
thiru at keionline.org

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