[Ip-health] WSJ Pharmalot: Congress Should fix the Priority Review Voucher Program: MSF’s Rius Explains
joanna.l.keenan at gmail.com
Thu Dec 4 02:04:36 PST 2014
Congress Should fix the Priority Review Voucher Program: MSF’s Rius
*Twice in recent months, drug makers have purchased priority review
vouchers that can be used to obtain faster FDA reviews for medicines being
developed for certain diseases. One voucher was issued to spur development
of treatments for rare pediatric diseases and the other had been issued to
provide incentives to create medicines for neglected tropical diseases. In
the most recent sale
Knight Therapeutics sold a neglected tropical disease voucher to Gilead
for $125 million. The deals marked the first time these vouchers were sold,
but while the voucher program has won praise, critics say the program does
not do enough to ensure drug makers develop needed medicines. A bill is
before Congress to add Ebola to the list of neglected tropical diseases,
which Judit Rius, the U.S. manager and legal policy adviser for Doctors
Without Borders, says should be strengthened. This is an excerpt of our
*Pharmalot*: First, what do you think is good about the voucher program?
*Rius*: It’s good that the US created an incentive not based on extending
market exclusivities. By that, I mean a monopoly for distributing and
manufacturing a product in exchange for investing in underfunded R&D, such
as neglected tropical diseases. There’s a lot of suffering caused by these
diseases and there’s no appropriate treatment – vaccines or diagnostics –
which additional development could alleviate. And that’s why it’s important
to create incentives to promote innovation.
*Pharmalot*: But you believe it could be improved. What do you think needs
to be done?
*Rius*: Until the Gilead deal, it has arguably not been incentive enough to
promote innovation. We’ve twice written to Congress to complain. The Gilead
deal does not involve billions of dollars, but the fact that a company
receives $125 million shows it has some economic value. What we propose is
that the mechanism be fixed. We asked Congress to do so in four different
ways. Congress is trying to act on it in two ways, but is ignoring another
two ways that we suggest.
*Pharmalot*: And these would be…
*Rius*: We need to increase the potential economic value by making the
voucher for neglected tropical diseases transferable indefinitely, instead
of only being able to transfer a voucher just once. For rare pediatric
diseases, a voucher can be transferred without any limit. Congress should
extend this to neglected tropical diseases as well, because companies can
keep trading a voucher and, theoretically, raising the economic value. The
bill we’ve seen that Congress is looking at doing this.
*Pharmalot*: What else?
*Rius*: We see a problem when a voucher has been awarded to a company that
has not invested in new R&D or a new product. There are several examples in
which companies have received vouchers (from the federal government) that,
we think, have not deserved it. If you don’t do innovation, you shouldn’t
get a voucher. Companies should demonstrate they have made a significant
investment before receiving a voucher.
*Pharmalot*: Explain how a company can be awarded a voucher if they have
not done the work.
*Rius*: The way the mechanism works is that a company isn’t required to
provide proof that there was basic research and development, such as
investing in clinical trials to bring a new product to the market. The
voucher is supposed to be an incentive for innovation. The deal with Gilead
is an example.
The company that got the priority review voucher is Knight Therapeutics,
but the product, Impavido, has been available for many years in other
countries [to treat leishmaniasis, a parasitic disease spread by sand
flies]. So if a product is already in the market, then there is no
innovation taking place.
What this company has done is nothing new – the product was already in use
to treat the disease in many countries. The company simply registered the
product in the U.S. and then got the incentive – in the form of a voucher –
from the government. It’s like gaming the system. It’s unfair [for the
government] to give an economic benefit for not doing anything.
*[EDITOR’S NOTE*: Paladin Labs bought the drug in 2008 from Aeterna
Zentaris. When Endo Pharmaceuticals bought Paladin earlier this year,
Jonathan Goodman, a Paladin co-founder, formed Knight Therapeutics to
retain rights to the drug in the U.S. Goodman says that Paladin spent $10
millions to obtain FDA approval over a four-year period, which involved
“significant investment, time and risk,” although he could not say how much
was spent on R&D. Spokesmen for Endo and Aeterna could not provide any
information on R&D spending ].
*Pharmalot*: So you’re saying Knight got a voucher that is supposed to spur
R&D, but neither Knight nor Paladin did R&D and Knight got a voucher anyway?
*Rius*: Yes, exactly. But there’s another problem. There’s no guarantee
these products will be accessible to patients anywhere in the world – the
U.S. or globally. There is no mandate. Congress gives the reward, but there
is no guarantee these [medicines] will be affordable. Paladin and Endo have
exclusive rights globally and Knight has exclusive rights in the U.S. But
the product has been in the market for many years and there is nothing to
say it will be accessible.
*Pharmalot*: So you’re saying that the existing price is out of reach for
*Rius*: Yes, but Congress is not addressing this. They’re looking at a bill
<https://www.congress.gov/bill/113th-congress/senate-bill/2917> that would
extend the voucher to include Ebola as a neglected tropical disease and
this would increase the value of a voucher to pharmaceutical companies. But
Congress is not looking at anything that will ensure any product is
accessible. And the bill does not require a company to invest in new R&D
for a new product. So Congress is doing nothing to ensure the mechanism
will not be gamed again.
*Pharmalot*: What do you believe Congress should do then?
*Rius*: They could reward companies that only invest in R&D and require
they disclose their pricing strategy. Ideally, this would make sure [a
medicine] is affordable. And the companies should only be eligible for a
voucher if they explain these two points. The program is a good idea, but
it needs to be improved. Incentives are good, but they should work.
Right now, this is a huge missed opportunity. By including Ebola in the
list of neglected diseases, the bill is getting a lot of attention. But we
need to fix these loopholes. The sale to Gilead shows there is value, but
it can be used to generate innovation, which is it not doing. And we could
have more medicines for diseases that are being neglected by companies. So
it could be very important from a public health perspective.
Médecins Sans Frontières - Access Campaign
P: +41 22 849 87 45
M: +41 79 203 13 02
More information about the Ip-health