standards that impact reimbursement
rates is here,” she says.
The debate about
Just as the launch of Gilead’s Sovaldi
kicked off a conversation about what was
the ultimate benefit of the drug compared
with its pricing, the new immunotherapies that have been coming to the market
have been inspiring a similar debate. Kymriah currently is the first CAR-T therapy but others are expected to enter the
market. Assessment bodies in Europe are
focusing on PD-1 inhibitors such as Merck’s Keytruda, Roche’s Tecentriq, and
Bristol-Myers Squibb’s Opdivo.
IQWiG, the independent authority in
Germany that evaluates new drugs and
plays a key role in what price health services pay for them, has looked into new
immunotherapy drugs for the treatment
of bladder cancer as part of its so-called
early benefit assessment.
The body has criticized a lack of data
directly comparing drugs in a promising new class of cancer immunotherapy,
saying physicians could be overwhelmed
or misled by the available information.
It concluded there were some signs that
patients who could not be helped by previous courses of standard chemotherapy
could benefit considerably from Keytruda and Tecentriq, but said physicians
needed head-to-head drug trials to pick
the best treatment option.
“The procedure of the early benefit assessment does unfortunately not allow
a comparison of new drugs against each
other,” IQWiG Director Jürgen Windeler
said in a statement.
“Such a measure appears to be almost
indispensable in the case of urothelial
(bladder) carcinoma: We now have three
drugs for the same therapeutic indica-
tion, but are unable to reasonably relate
the assessment results to each other.”
IQWiG says regulations should be
changed so that companies are required
to conduct such studies as IQWiG does
not have the mandate to do so.
Tim Turnham, VP of MK&A and former executive director of the Melanoma
Research Foundation, told Med Ad News
that trying to calculate the cost and benefit of these new cancer immunotherapies
is never straightforward.
“First, patients on immuno-oncology
therapy often have treatment holidays,”
he says. “During this time costs drop considerably. When economists calculate the
cost of I/O therapy this is often not taken
“Second, data is very clear that the
benefits of I/O often extend far beyond
the span during which they are adminis-
tered. Costs, then, need to be measured
as $/month of PFS or $/month of OS.
In this situation the calculation becomes
more favorable. We do not yet know ex-
actly how favorable because third, some
people have complete eradication of dis-
ease and may, in fact, be cured.”
Whether these drugs actually achieve
a cure is unknown because they have
no been around long enough, Turnham
says. “We do know that the OS curve
has a ‘tail’ and many patients who start-
ed with Yervoy on clinical trials are still
alive, now over a decade later,” he com-
ments. “Calculating the $/month of OS is
not really possible until we see how long
these people live.”
In metastatic melanoma, the first
breakthough was with the CTLA- 4 inhib-
itors such as Yervoy, and the question of
price came up. “When I was interviewed
at that time, saying what about the price
of this, my comment was, ‘It’s a little bit
disingenuous to talk about price when
you have nothing that would save the
lives of patients with metastatic mela-
noma and now you have something that
does. So let this play out a little bit more,’”
Turnham states. “And so we have seen
this play out a little bit more, and we have
seen it play out in interesting ways in that
the PD-1 inhibitors work better than the
CTLA- 4 inhibitors, in most cases, but it
also turns out that combinations work
better than monotherapies. So now we
have an expensive drug and are adding
another expensive drug on top of it.”
In establishing the true price versus
value of these drugs, there are several fac-
tors that need to be considered, Turnham
told Med Ad News.
“How long to these drugs need to be
given? We’re just really beginning to figure that out,” Turnham says. “But we do
know that people needed to stop therapy
because of toxicity. Sometimes they have
durable responses regardless of stopping
therapy. And sometimes they have a
late-blooming response that shows three
to six months later, in some cases, just after one infusion. Now we know the likelihood of responses goes up with more
infusions, but how do you determine value when you don’t even know how much
drug somebody needs to get?
“And if you look at the combinations,
you have increased costs but you have
increased responses. The CTLA- 4 monotherapy in melanoma has response rates
of about 12 to 14 percent; the PD-1 in melanoma has a response rate of about 40
percent; you put the two of them together
and the response rates are 60 percent. So
does it make more sense to spend more
money and carve out another 20 percent
of the patient population who are going
to have a response and live longer and
possibly have a durable response? So that
has to be brought into the equation.
The other component to the equation
is quality of life.
“The most important perspective in a
After she passed away, well into 2017 there was still a shelf of one of the kitchen
value framework is the patient’s perspec-
tive but it’s complicated so often we’re
not going to include it,” Turnham says.
“Which is a real cop-out, we’ve got to do
better than that. It’s not just quality of
life, but patients’ personal values have to
weigh into this conversation. Some peo-
ple might be willing to deal with a real-
ly horrible quality of life if it gives them
enough time to watch their child go into
first grade. Another person might say,
‘I’d rather have three really great months
than to have a year where I’m dealing
with all kinds of hellacious things.’ We do
know that these immunotherapy drugs
the immune system is a big powerful
thing, that’s why these drugs work; but it
also can lead to problems. The personal
value, the personal quality of life, the per-
sonal decisions, need to be brought into
this equation somehow.” medadnews
n Oct. 28, 2016, my mother, Joan Biamonte, died of complications from
metastatic carcinoma of the lung. Despite having her original tumor
characterized and going through a regimen of chemotherapy alleged-
ly tailored to her tumor type, her cancer proliferated and spread into
her liver and brain.
cabinets ;lled with the medications she used to take. Crestor. Diuretics. Painkillers.
And her cancer medications. My father has since bundled her unused drugs into a
plastic bag in the basement, awaiting legal disposal.
While looking for something else, I found her A;nitor (see the photo above)
– an unopened box of 28 pills and several packets. Considering that the “listed”
pharmacy price online for A;nitor is $14,524.24 for 28 pills, that is some serious
money sitting there.
My mother had to stop taking A;nitor because she experienced just about all the
debilitating side e;ects and it had no e;ect on the spread of her cancer. But that’s
not the reason for all those extra pills.
Back in July 2016, my mother was explaining to me how much time she had spent
on the phone with her PBM. “They don’t know basic math,” she complained.
Her regimen was one pill a day for two weeks, then a week o;. A “month’s” worth of
medication under that regimen was literally 14 pills. So one box covered two months.
However, her PBM sent her three months of her prescription, as most PBMs do – so
something like three boxes (84 tablets) arrived at a time. This was a logical assumption for a regular, generic, small-molecule drug, but this protocol worked less than
well with a high cost, complex antineoplastic agent.
It took my mother a lot of hours on hold and then ;nally talking with at least two
or three people to get them to stop sending her the A;nitor in job-lot quantities. And
they didn’t want the extra back, for fear of contamination and tampering.
Who paid for that A;nitor? For the most part, Medicare. My mother had her share
of costs to pay, but less than $72,000 comparatively speaking (I think she still had
to pay about $10,000 and some of that was covered by supplementary insurance,
but it was still a hit for someone on a ;xed income). And because of the general
non-transparency of PBMs, there is little chance of knowing just exactly how much
that A;nitor cost Medicare.
This incident is probably just one of many, not just at this PBM but at others as
“It’s got a lot of bene;ts if it’s done correctly but again it’s another one of these
well. There are probably millions of dollars in excess, expensive medications sitting
on shelves all across the country, as someone at a PBM shrugs their shoulders and
thinks, “Well, so long as their insurance is paying for it...”
According to Dan Renick, president, Precision for Value, “there’s been some move-
ment in the market to allow specialty drugs to be delivered by local pharmacies.”
Precision for Value has seen that if a healthcare system bears more risk, “they
become more sensitive to things like that and tend to handle it better. For ex-
ample, in our work with clients we sometimes do something called ‘private care
optimization,’ where it’s just not treating a patient, it’s treating them in the more
e;cient and e;ective side of care. It allows them to access, and better a;ord care,
and eliminates waste.
changes that can be di;cult to put in place because there are so many deeply em-
bedded ways of going about doing the business.”
At current listed pharmacy prices, this is about $18,000 worth of Afinitor.