[Ip-health] ICER says Novartis’ SMA gene-therapy, if priced at $2M, could be more cost-effective than Biogen’s Spinraza, since the NIH funded drug Spinraza is so damned expensive

James Love james.love at keionline.org
Fri Dec 21 06:55:54 PST 2018


This illustrates the limits of health technology assessment.  ICER has
indicated that a price of $2 million per patient for a SMA treatment is
cost effective, because the alternative is an even more expensive drug,
Spinraza.

(Spinraza was invented on NIH grants, and one of the patent holders, Cold
Spring Harbor, failed to disclose the NIH funding.)

If you can, read this excellent report from endpoints on their web page.

https://endpts.com/novartis-sma-gene-therapy-if-priced-at-2m-could-be-more-cost-effective-than-biogens-spinraza-icer/


Novartis’ SMA gene-therapy, if priced at $2M, could be more cost-effective
than Biogen’s
Natalie Grover,  December 21, 2018


Spinraza — ICER

Biogen’s Spinraza was approved by the FDA amidst much fanfare in 2016 as
the first and only disease-modifying treatment for SMA, a rare and often
fatal genetic muscular disorder. But the price tag of $750,000 for the
first year of therapy (and a lower price thereafter) prompted heavy
criticism, although many payers eventually agreed to reimburse the
treatment. How‐ ever, a report by the Institute for Clinical and Economic
Review (ICER) on Thursday has suggested Novartis’ experimental SMA gene
therapy, Zolgensma, could be more cost-effective in the long run in
comparison to Spinraza.

Zolgensma is currently under FDA review and the agency is expected to
announce its decision on the drug in May 2019. In its review, ICER has as‐
sumed Zolgensma will carry a list price of $2 million, although the Swiss
drugmaker has suggested a price of $4 million gene therapy (
https://endpts.com/category/gene-therapy/), pricing (
https://endpts.com/category/pricing/)

Biogen’s Spinraza was approved by the FDA amidst much fanfare in 2016 as
the first and only disease-modifying treatment for SMA, a rare and often
fatal genetic muscular disorder. But the price tag of $750,000 for the
first year of therapy (and a lower price thereafter) prompted heavy
criticism, although many payers eventually agreed to reimburse the
treatment. How‐ ever, a report by the Institute for Clinical and Economic
Review (ICER) on Thursday has suggested Novartis’ experimental SMA gene
therapy, Zolgensma, could be more cost-effective in the long run in
comparison to Spinraza.

Zolgensma is currently under FDA review and the agency is expected to
announce its decision on the drug in May 2019. In its review, ICER has as‐
sumed Zolgensma will carry a list price of $2 million, although the Swiss
drugmaker has suggested a price of $4 million (
https://endpts.com/stickershock-novartis-says-its-top-drug-prospect-is-worth-4m-5m-for-a-once-and-done-use/)
for the curative therapy, which it acquired via its $8.7 billion takeover
of AveXis, may be justified.

Despite the lack of long-term outcome data for either therapy, the
non-profit estimated incremental cost-effectiveness of Spinraza is $728,000
per QALY in presymptomatic SMA patients, while Zolgensma has an incremental
cost-effectiveness of $247,000 per QALY in patients with symptomatic Type I
SMA (on the basis of a placeholder price of $2 million).

QALYs, or quality-adjusted life-years, are a measure of the state of health
of a person or group in which the benefits — in terms of length of life —
are adjusted to reflect the quality of life. Essentially, one QALY is equal
to one year of life in perfect health.

“Novartis has estimated that Zolgensma would yield 13.3 QALYs in SMA Type I
patients, and could be priced to $4-5 million based on the relation‐ ship
of 10-year cost of approved drugs for ultra-rare diseases and their
corresponding incremental QALYs gained. ICER estimates Zolgensma pro‐ vides
11.33 QALYs, and uses a placeholder price of $2 million. While ICER
undercut both of Novartis’ estimates, the high cost/QALY of Spinraza in
Type I SMA patients offers Novartis bargaining power with payers if the
company can argue Spinraza use could be reduced or eliminated to offset
total costs,” Leerink analysts wrote in a note on Friday.

The ICER report (
https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Draft_Evidence_Report_122018-1.pdf)
is not final and has been opened to the public for comment, which will be
incorporated into an evidence report in February 2019. This report will be
subject to further de‐ liberation by one of ICER’s independent evidence
appraisal committees in early March, after which a final report will be
released by the end of the month.

“If confirmed in a final report…these findings could offer support for
Novartis in pricing and access negotiations for Zolgensma ahead of 2019 ap‐
proval,” Leerink analysts added.

Like NICE in the UK, ICER analyzes the evidence on the effectiveness and
value of drugs and other medical services in the United States, however
unlike NICE, it is not a government-affiliated body.

In August, NICE refused to recommend the use of Spinraza in the UK (
https://endpts.com/biogens-big-breakthrough-drug-spinraza-runs-into-abarrier-as-nice-turns-thumbs-down-on-reimbursement/)
— despite Biogen discounting its price — on the basis that the long-term
impact of the drug was unclear for patients, some of whom die by the age of
2. Without that data, the price of the drug couldn’t be justified, they
said.

(
https://endpts.com/stickershock-novartis-says-its-top-drug-prospect-is-worth-4m-5m-for-a-once-and-done-use/
)

for the curative therapy, which it acquired via its $8.7 billion takeover
of AveXis, may be justified. Despite the lack of long-term outcome data for
either therapy, the non-profit estimated incremental cost-effectiveness of
Spinraza is $728,000 per QALY in presymptomatic SMA patients, while
Zolgensma has an incremental cost-effectiveness of $247,000 per QALY in
patients with symptomatic Type I SMA (on the basis of a placeholder price
of $2 million).

QALYs, or quality-adjusted life-years, are a measure of the state of health
of a person or group in which the benefits — in terms of length of life —
are adjusted to reflect the quality of life. Essentially, one QALY is equal
to one year of life in perfect health.

“Novartis has estimated that Zolgensma would yield 13.3 QALYs in SMA Type I
patients, and could be priced to $4-5 million based on the relation‐ ship
of 10-year cost of approved drugs for ultra-rare diseases and their
corresponding incremental QALYs gained. ICER estimates Zolgensma pro‐ vides
11.33 QALYs, and uses a placeholder price of $2 million. While ICER
undercut both of Novartis’ estimates, the high cost/QALY of Spinraza in
Type I SMA patients offers Novartis bargaining power with payers if the
company can argue Spinraza use could be reduced or eliminated to offset
total costs,” Leerink analysts wrote in a note on Friday.

The ICER report (
https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Draft_Evidence_Report_122018-1.pdf)
is not final and has been opened to the public for comment, which will be
incorporated into an evidence report in February 2019. This report will be
subject to further de‐ liberation by one of ICER’s independent evidence
appraisal committees in early March, after which a final report will be
released by the end of the month.

“If confirmed in a final report…these findings could offer support for
Novartis in pricing and access negotiations for Zolgensma ahead of 2019 ap‐
proval,” Leerink analysts added.

Like NICE in the UK, ICER analyzes the evidence on the effectiveness and
value of drugs and other medical services in the United States, however
unlike NICE, it is not a government-affiliated body.

In August, NICE refused to recommend the use of Spinraza in the UK (
https://endpts.com/biogens-big-breakthrough-drug-spinraza-runs-into-abarrier-as-nice-turns-thumbs-down-on-reimbursement/)
— despite Biogen discounting its price — on the basis that the long-term
impact of the drug was unclear for patients, some of whom die by the age of
2. Without that data, the price of the drug couldn’t be justified, they
said.


-- 
James Love.  Knowledge Ecology International
http://www.keionline.org <http://www.keionline.org/donate.html>
twitter.com/jamie_love


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