is she aware of the same?
does she love Norvartis' dope/cash?
life, death, holes in the brain, it is all the same?
U.S. News
Researchers Fret as Social Media Lift Veil on Drug Trials
Online Chatter Could Unravel Carefully Built Construct of 'Blind' Clinical Trials
July 29, 2014 10:30 p.m. ET
Jeri Burtchell at home in East Palatka, Fla.
Bob Croslin for The Wall Street Journal
On her first day in a clinical trial
for an experimental multiple sclerosis drug,
Jeri Burtchell
was convinced she was getting the new drug, not the standard
therapy that some patients were randomly assigned to receive.
When
she bumped into the trial's lead investigator in the elevator that day,
she told him, "I think I know which drug I'm on!" The doctor put up his
hands, waving her off. "Don't tell me!" Ms. Burtchell recalls him
blurting. "I can't know!"
A nurse later
explained to her that knowing who was getting what could compromise the
research. Even so, Ms. Burtchell went home and recounted the incident to
followers of a blog she was writing about the trial. "The mystery medicine is doing its job," she wrote.
For
decades, the clinical trials vital to developing new drugs have
followed a central principle: Researchers and patients must both be
"blinded" as to who is getting the experimental drug and who a placebo
or standard therapy.
If patients know
who has the new drug, or learn too much about how others in the trial
are faring, this knowledge could influence how they report symptoms and
make it hard to tell whether the drug is working. Staying in the dark
throughout a trial is a standard held sacrosanct by researchers. But
lately, not so much by patients.
On
Facebook groups, online forums and blogs, some patients are effectively
jeopardizing the blind. In trials for hepatitis C, multiple sclerosis
and ALS (Amyotrophic lateral sclerosis, also known as Lou Gehrig's
disease), patients have been sharing details of their reactions and
trying to figure out whether they are getting the active drug.
Patients
also swap tips on how to get accepted into trials, even if they don't
meet all the requirements. Some who are in trials collect and analyze
their medical data and others' to get an early indication of whether a
drug will be a success.
Drug makers and
researchers are increasingly concerned that online chatter could unravel
the carefully built construct of the clinical trial, and perhaps put
patients in danger. They worry that patients may drop out if they
suspect they aren't getting the drug being tested, or may report
symptoms inaccurately because of the influence or suggestions of others
in the trial.
Patients who share too
much "could effectively chill a new drug before it ever gets to patients
by misinterpreting early signals," says
Craig H. Lipset,
head of clinical innovation at
Pfizer Inc.,
PFE -0.71%
who wrote an opinion piece in Nature Medicine in March calling on trial sponsors and regulators to study the effects of social media. "It's scary."
Patients,
for their part, often say they have the right to talk about their
experiences. In life-threatening diseases such as ALS, patients "want,
need and deserve to know" how others are doing, says
April Moundzouris,
a Chattanooga, Tenn., woman whose "April's ALS Blog" chronicles her experience with an experimental stem-cell treatment. The early-stage research by
Neuralstem Inc.
CUR -3.81%
isn't blinded.
Neuralstem Chief Executive
I. Richard Garr
, a close reader of April's blog and those of other patients,
says the sharing may pose a challenge later on when it comes time for a
trial designed to prove efficacy to the Food and Drug Administration.
That trial would likely have to be blinded.
A
patient who blogs that he or she isn't feeling well, isn't improving or
has some symptom can sway how other patients report or their
willingness to join a trial, Mr. Garr says.
This is just one of the ways the Internet and social media bear on the drug-discovery process.
Richard Bedlack,
who runs the ALS clinic at Duke University, is involved in a
project that asks patients to tweet tips about alternative and
off-label treatments that he and other doctors assess to see if they
might make promising research candidates.
Excerpt from a post on Jeri Burtchell's blog
"(The nurse) tells me the shot should go in the outer half of my thigh, somewhere in the center 1/3. I didn't know I was going to have to be good at fractions to be able to do this, but I listen to her and nod like I'm understanding it all. She wipes with alcohol, and the next thing I know I'm seeing that big needle sink into my thigh like I was the Pillsbury dough boy's sister. It went right in with no effort at all and I couldn't even feel it. She pushed the plunger and I braced myself for a sting or burn as the medicine hit my muscle, but…nothing. It was at that point that I'm thinking "hey!! I bet that was just water!!: which means I can quit worrying about Avonex side effects and worry about Fingolimod ones.''
By contrast, some pharmaceutical
companies have tried to avoid using social media so they won't learn
about something that might make clinical analysis of an experiment more
challenging or might have to be reported to FDA, according to
Joseph Kim,
an executive at Irish drug maker
Shire
SHPG +0.18%
PLC.
Says Mr. Garr: "The FDA is going to have to figure out how to accommodate social media."
A
spokeswoman for the FDA says the agency doesn't have a policy on
subjects in trials using social media to communicate with one another.
PatientsLikeMe,
a Cambridge, Mass., company that runs a patient network and research
platform where patients share health information, including during
clinical trials, is wrestling with how to address the effects the
constant sharing of information can have on trials.
In
a 2012 paper,
Paul Wicks
of PatientsLikeMe wrote that according to the company's data, a
little more than 400 patients on the site have added treatment
information that shows they are on a clinical trial. If patients start
trying to determine if they are receiving the new drug, he asked, "how
should we respond to this as researchers or as a company?" Dr. Wicks
says the company is trying to get patients and researchers to work
together to design trials, believing that collaboration will lead to
better understanding of the issues.
To
date, no clinical trial has been stopped because of online sharing of
patients' experiences. But drug makers are starting to take pre-emptive
steps.
Shire helped create educational
videos about the consequences of sharing too much, working with a
Boston-based nonprofit called the Center for Information and Study on
Clinical Research Participation. In one animated video,
a blue bean's description of side effects from a drug causes a yellow
bean to drop out of a study. Shire, whose board has agreed to a takeover
by
AbbVie Inc.,
ABBV +0.60%
tells patients how to download the videos.
"The
industry needs to do a lot more,'' says Shire's Mr. Kim, director of
patient recruitment and engagement. "It is a hard thing to modify
behavior without it being too controlling."
Vertex Pharmaceuticals Inc.
VRTX -3.71%
was testing a drug for hepatitis C when
Lindsay McNair,
then its senior medical director, got an alert from an
investigator that patients in the trial were posting about it. She went
to MedHelp.org, a site that hosts patient message boards, and started
reading. "They were really incredible threads," she says.
One
patient advised those who wanted to know whether they were getting the
experimental drug to ask a doctor not involved in the trial for a test
of viral load. If it had dropped dramatically, the patient wrote, that
was a sign the person was getting the new drug, because standard therapy
wouldn't have such an effect. Some patients discussed dropping out if
they concluded they weren't getting the new drug. (Some did quit the
trial, though it isn't known if online sharing was a factor.)
Dr.
McNair discussed the posts with others at the Boston-based company.
They were concerned about patients harming their health by not reporting
symptoms out of concern they could be taken off the trial. If enough
dropped out, it might be hard to get an adequate test of the drug, says
Dr. McNair, now chief clinical research officer at WIRB-Copernicus
Group, which provides regulatory and ethical reviews of research.
So
for its next trial, Vertex added an unusual paragraph to the consent
document for subjects, explaining that rumors about side effects or
about the drug's efficacy might affect results and require doing the
study over. Participants should feel free to discuss their participation
with family, friends and medical providers, the paragraph said, but not
publicly such as in Internet message boards.
"It
was meant to be informational and educate about the implications
discussions could have, but not to say they can't talk or there would be
repercussions if they did,'' says Dr. McNair.
Not
all the patients understood this. One who signed the document then
posted on the MedHelp Hepatitis C forum: "There's no specific legal
recourse spelled out, but there is another section that states that my
participation in the study can be ended at any time without my
permission." The patient wrote that the forum provided him with
important information about managing his disease and about the trial. "I
hope people will still talk to me here?!" he added.
Vertex
says oversharing on social media isn't grounds for eviction from a
study. It continues to warn about the problem in consent forms. Vertex
says there was no effort to identify or remove from the trial the person
who posted on the MedHelp forum.
The hepatitis C drug won FDA approval, and now is sold as Incivek, but "the experience was nerve-racking," Dr. McNair says.
Dr.
Bedlack of Duke says the information patients share online can turn out
to be incorrect. Before revealing the drug information at the
conclusion of a trial, he often asks patients to guess whether they got
active drug. "Most times they don't get it right," he says.
Ms. Burtchell, the multiple sclerosis patient, did guess right.
Now
a 53-year-old resident of East Palatka, Fla., she was diagnosed in 1999
with the kind of MS that involves relapses interspersed with remission.
By 2007 she estimates she had suffered nearly 30 relapses and was so
exhausted she had difficulty caring for her young son.
She enrolled in a trial for an experimental therapy from
Novartis AG
NOVN.VX +0.44%
that aimed to be the first pill for the disease, which was
treatable only with injectable drugs. The trial matched one of
injectable therapy, Avonex, against the experimental oral drug, called
fingolimod.
Ms. Burtchell started a
blog to chronicle her experience from start to finish. In the post after
her first treatment, she told why she believed she was getting
fingolimod.
In the past, she had taken
injections for her condition. In the trial, she didn't feel the stinging
or pain she remembered from shots—a sign, she believed, that her
injection in the trial was a placebo.
Also,
after she was given a pill, she noticed a nurse recorded a fall in her
blood pressure, which she knew was a potential side effect of the
experimental drug.
Nearly three months
into the trial, Ms. Burtchell posted that, for the first time since her
MS diagnosis, she was able to hop on one foot.
"Maybe it's the Fingolimod…," she speculated. "Whatever it is, I feel better every day."
When information on the study was revealed, it turned out she was indeed getting the experimental drug.
A
spokesman for Novartis says consent forms for its trials don't prohibit
patients from talking about their trial participation. The spokesman
says the company didn't interact with Ms. Burtchell during the trial.
The
drug was approved by the FDA in 2010 and now is sold as Gilenya.
Novartis continued to study it in nonblinded research, in which Ms.
Burtchell participated. Eventually, she served for a time as a paid
speaker for Novartis, sharing her experiences.
She also continued to blog.
One of her followers was Pfizer's Mr. Lipset, who was interested in how patients use social media.
In
September 2011, he sent her a message on Twitter asking to talk. Mr.
Lipset says he wanted to discuss the downsides of patients comparing
notes. They talked, and each saw the other's side.
Mr.
Lipset says Ms. Burtchell helped him realize that "it's not the
patients who will change, but the researchers who have to change." She
reminded him, he says, that "ultimately patients are human beings. They
are going to talk."
In Ms. Burtchell's
post after their talk, she conceded that the Pfizer executive had made
many good points. Even so, she concluded, "If I'm going to be poked and
prodded, I'm going to be here blabbing about it."
Later,
her views started to shift. In 2012, Mr. Lipset invited her to appear
on stage with him at a pharmaceutical-industry conference in Boston. She
was asked if she ever worried about influencing others in a trial. "I
think that is when the realization of what I had done really hit me,"
she says.
At a February drug-industry
conference in Miami where she was asked to speak about patients and
social media, Ms. Burtchell proposed her own solution: that drug
companies create online forums where patients can get practical
questions answered by investigators and can commiserate with other
patients about social issues in a moderated setting. She set up Partners
in Research, a website that guides patients through the clinical-trials
process.
She continues to take Gilenya
for her MS and has had two relapses while on it, most recently in
February. She is part of a study looking at its long-term effects.
But she has stopped her blog.
"I'm too aware of the impact,'' she says, "too conscious of my audience, to blog like that again."
Write to Amy Dockser Marcus at amy.marcus@wsj.com
see also faustmanlab.org
and pubmed.org faustman dl
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