Do Pills Produce Compliance?
The most obvious difference between medications like these and Novartis' Gilenya (fingolimod) is that there's no injection.
Gilenya is the first oral medication to gain FDA approval as a first-line treatment for patients with relapsing forms of MS
in the United States, administered as a 0.5-milligram pill to be taken once a day. "Many people prefer to take a capsule because
they don't like to stick needles into themselves," Dr. Nick LaRocca of the National Multiple Sclerosis Society told the Associated Press in September 2010
Negative patient reactions to injectibles may be why Mundel maintains that one of the biggest differentiating factors of Gilenya
is the lifestyle change marked by the convenience of an oral treatment. Up until the FDA advisory committee met last June
to hear patients' accounts of their experiences with Gilenya, "the skepticism was overwhelming," says Mundel. However, one
patient account may have swayed a panel that was still very uncertain about whether the compound was really a breakthrough.
A young woman spoke to the committee about her lifestyle with injectible treatments and shared how she would often cry for
half an hour before injecting herself. Mundel believes this testimony and others like it swayed the committee in their decision
to make Gilenya a first-line, rather than a second-line, treatment because, "they had to ask themselves, 'What do we say to
patients like this one if we have to tell them it won't be a first-line treatment, and that they have to continue on like
Certainly, at first glance, this shifting of disease treatment and management seems to be quite significant. "If you ask a
22-year-old, 'Do you want a shot every day or a pill every day?', I don't think there's any contest," says chief medical officer
for the Multiple Sclerosis Association of America (MSAA) and neurologist Dr. Jack Burks. But Burks cautions that oral delivery
of medication has its own inherent problems.
Challenges to Market Takeup
While the obvious assumption may be that the convenience of a pill would cause patient adherence to skyrocket, Dr. Burks warns
that commonplace oral medications for MS may cause patients to become blasé, or to take the disease and its treatment regimen
less seriously. "If this drug works well and the patients' symptoms of MS are very minimal with Gilenya, I'm concerned it
will be like patients on blood pressure medication—unless you keep pushing them, they won't take their medication," he says.
"None of these drugs cures a disease—the disease is still there," advises Burks. "So it's going to be up to the physicians,
and the medical community in general, to keep reminding people that the reason they feel so good is because they're on the
medication. And in fact, the reminder of taking a shot every day is much more powerful than the reminder of taking a pill
every day; we have to be very vigilant."
In fact, there seems to be a greater "If it's not broke, don't fix it" mentality springing up among all those involved—patients,
physicians, and analysts alike. "Although the majority of the disease-modifying therapies for MS are currently given via an
injectible delivery mechanism—which is inconvenient, painful, and requires specialized storage conditions—compliance is quite
high, at 80 percent or higher," notes Datamonitor healthcare analyst Trung Huynh. MS patient L. Mello echoes this sentiment
when she says, "The medication I am on is working for me. Why chance switching over when I don't know what will happen?"
Science, History, and Safety
Another point of differentiation for Gilenya may be why and how it affects MS. Mundel translates the science into layman's
terms for Pharm Exec: "Gilenya effectively blocks a system that the body has in place to control certain white blood cells—lymphocytes, which are
trafficking around your body and through the lymph nodes," he explains. These lymphocytes monitor what's going on in the body,
assessing potential invaders and problems. In the case of MS, the lymphocytes get out of control in the body and "go wild,"
participating in the destruction of the myelin sheath that surrounds the nerves of the CNS. Gilenya effectively contains the
rogue lymphocytes, keeping them locked in the lymph nodes and preventing them from reaching the CNS, where they could potentially
attack this protective covering. This method is unique among MS treatments in that many other medications kill off the lymphocytes,
whereas fingolimod merely traps them.