Transcript Presentation

James P. Simsarian, M.D., MBA
Director Multiple Sclerosis Program
Neurology Center of Fairfax Ltd
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Private Practice of Clinical Neurology in an Urban
Community – With MS Subspecialty
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Principal Investigator in Company Sponsored Phase III
and Phase IV Trials
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Conducted Investigator Initiated Clinical Trials
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DSM monitor in Phase III Trial
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Consulting, Advisory Boards and Speaker Bureaus
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Past Chair, Department of Neurology Fairfax Hospital
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Past President Consortium of Multiple Sclerosis Centers
Deciding How to Decide: What Processes Do
Patients Use When Making Medical Decisions?
Maria J. Silveira, Lorna Rhodes and Chris Feudtner: Journal of
Clinical Ethics; Vol 15 #3: P1-13; 2004.
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“We conclude that the informed decisionmaking process may be only one aspect – an
aspect not always present – of a more
complex sequence of medical decisions.”
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2,944 people surveyed on which hypothetical
cholesterol drug they would choose: one that had a
"patient outcome" (reduced heart attacks) or one that
only had a "surrogate outcome" (lower cholesterol).
Participants were divided into three groups.
◦ One group was told only what the drugs did.
◦ A second group was given the same information but also
warned that it wasn't clear that the second drug helped
patients "feel better or live longer."
◦ A third group got the information, the warning and was told
to "ask for a drug shown to reduce heart attacks."
Archives Internal Medicine 2011
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Patients would probably be better off choosing the
first, more "proven" drug, assuming they
understood that the first drug was less risky than
the second.
In the study 71% of each of the two "informed"
groups chose the first drug, the better option. But
only 59% of the first group, which was given no
guidance, chose the better drug.
The problem seemed to be that people mistakenly
assumed all drugs on the market were very safe,
having passed the FDA's standard.
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The study showed that most people thought
the FDA-approval bar was set a little higher
than in reality
◦ 39% thought the FDA approves only extremely
effective prescription drugs
◦ 25% believed that only extremely effective drugs
can be advertised
◦ 25% thought the FDA approves only drugs
without serious side effects
◦ 17% thought that drugs with serious side effects
can't be advertised
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January 2008
◦ 44% of Americans said they viewed pharmaceutical
and biotechnology companies unfavorably
◦ 27% said that they do not trust them to provide
reliable information about drug side effects and
safety
◦ 45% said they do not trust research sponsors to
inform the public quickly when safety concerns
about a drug are discovered
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Current Clinical Condition
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Potential Benefit of the Medication
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Potential Side Effects
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No symptoms
Fear of new or worsening symptoms
Disability – current or potential
Pain
◦ Cure
◦ Symptomatic improvement
◦ Prevent further worsening
◦ Known side effects
◦ Theoretical risks
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What others say
◦ Internet
◦ Friends and family
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Would I be better off doing nothing
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Cost
◦ Insurance benefits
◦ Out of pocket costs
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Doctor Patient Relationship
◦ “You wouldn’t prescribe anything bad for me”
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The results suggest that the maximum
added risk that subjects are willing to
tolerate in exchange for realistic weight
losses are far smaller than the added risks
that can be detected in most extant obesity
randomized clinical trials.
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The study looked at MS patients' acceptance of potentially
fatal risks related to MS therapies.
10,259 MS patients in the NARCOMS Registry's online cohort
were invited to complete a web-based questionnaire on
treatment decision-making to determine MS patients'
tolerance for risky therapies and evaluate correlations with
patient characteristics.
Two standard gamble paradigms to evaluate risk tolerance
(RT): 1) curing MS [CureMS], but a risk of immediate painless
death; and 2) natalizumab [NAT], with benefits defined by
clinical trial results but a risk of PML.
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5,446 (53.1%) invitees completed the survey
The odds of the risk within each scenario
were adjusted iteratively to identify the
person’s maximum RT. Disease and
behavior characteristics were also collected.
Median RT for both scenarios was 1:10,000
◦ In both scenarios, RT correlated
 with disability (p<0.0001; higher tolerance in disabled)
 and sex (p<0.0001; males more tolerant).
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Significant predictors of lower tolerance for
risk in NAT were: female sex, older age, lower
disability, relapses in the past 12 months,
dependents at home, seat belt use, and
greater monitoring behavior.
Patient characteristics identified related to
tolerance to risk, included sex, age, disability,
and disease activity
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Only one-third of respondents reported a
Risk Tolerance that would allow treatment
with NAT.
This proportion would shift if the patientspecific risk of PML were to shift.
This shift is clinically relevant given the
emerging knowledge on risk factors for PML
related to natalizumab and our ability to
manage these risks.
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What is the Benefit for the Individual Patient?
◦ How do we determine individual benefit from the group effect
reported in clinical trials?
◦ What is the benefit we know?
◦ What is the potential benefit in the longer term?
 disability and mortality
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What are the Risks?
◦ Known risks
◦ Potential risks
◦ How likely are they to occur?
 Disability and mortality
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What if I do nothing now
◦ Wait for more information before starting a newly available drug
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When assessing a patient’s
acceptance of a new medication the
informed decision process may be
only one aspect of a more complex
sequence of medical and non-medical
decisions.