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MKA Article - Engaging payers in Parkinson's Drug Development
1. Engaging payers in Parkinson’s disease drug development
Lona Vincent, Senior Associate Director, Research Partnerships
Michael J. Fox Foundation for Parkinson’s Research
In medical research, we often think of regulatory approval as the final hurdle to development of
a new drug. For patients who desperately need those medications, however, there are other
obstacles – drug coverage, reimbursement and access.
With 1 million Americans living with Parkinson’s today and a slew of potential treatments in later
stages of clinical testing, the Michael J. Fox Foundation for Parkinson’s Research (MJFF) is
engaging payers (insurance companies, Medicare and Medicaid) to discuss the impact of
Parkinson’s and the potential effect of new therapies. We hope the echo of these conversations
will be heard when payers are deciding on coverage.
The Foundation has spent more than $450 million in research since its founding in 2000,
focusing not only on a cure but on therapies to treat the symptoms of Parkinson’s. This work is
made possible by a team of scientists on staff who evaluate research proposals and efficiently
determine the best fit with the portfolio and a team of business-trained project managers who
help to deploy donor-raised dollars.
While those funds push projects closer to the goal line, they may not be enough. Payers are the
ultimate decision-makers in determining patient access to new medications and therapies and
conversations with payers are another, valuable form of currency. Right now, Parkinson’s
disease is unique in that there simply are not that many choices for treatment, unlike, say,
oncology where the field is so crowded that some drugs may not make the formulary.
Levodopa, the gold standard treatment for Parkinson’s has been available for more than forty
years. Payers cover most of our current drugs. That, of course, could change with the
introduction of new products. Researchers are now looking at novel treatments like gene
therapy, surgical interventions and vaccine regimens, which have the potential to be quite
expensive.
In order to proactively address some of these novel therapies in the drug development pipeline,
MJFF is taking a proactive approach to communicating to payers the patient perspective and
unmet needs. The education runs both ways, too. We are learning from payers what they value,
and we are communicating that back to biotech and pharmaceutical partners. That
understanding can inform design of future clinical trials; improving the likelihood of a positive
coverage decision should the project reach that stage.
When we were first considering if the Foundation should develop a payer strategy, we were
concerned that payers would be too busy to sit down and work with a patient foundation before
product approval, but we haven’t faced any resistance. In fact, they’ve congratulated us on our
2. forward thinking and for wanting their perspective early in the process. For example, in October,
we gathered more than 20 payers and policymakers from the neurology field at our offices in
midtown Manhattan to discuss what was important in evaluating Parkinson’s treatments for
coverage or reimbursement. The attendees appreciated supplementing their knowledge with our
strong understanding of patients and current research. These payers are evaluating new
therapies for an array of diseases and disorders. Here at MJFF, all we think about is
Parkinson’s, and we bridge the gap between people living with this disease and their loved ones
and researchers and scientists.
At our October meeting, a member of our Patient Council shared his Parkinson’s experience
and the effect of motor fluctuations known as “on/off” episodes. The surprise and interest in the
room was palpable as our guest spoke of altering his daily life to avoid his medication wearing
off and experiencing freezing of movement while he was driving. These therapies may greatly
impact quality of life, but payers need to be convinced that there is a need for additional
treatment.
We discussed three therapies in the pipeline — potentially available within five years — that
could treat those on/off episodes:
• Current gold standard medication, orally administered levodopa administered through a novel
delivery system, an inhaler for fast relief of symptoms
• APL-130277, a formulation of currently available injectable apomorphine, in a Listerine-like,
under-the-tongue strip for relief from “on/off” in an easy-to-use way
• Levodopa/carbidopa (which prevents breakdown of levodopa) administered subcutaneously
through a pump/patch system to maintain medication levels and avoid symptom fluctuation
We have learned from payers that data is an important communication tool. As a result, we
launched a survey of our patient community earlier this year, receiving 3,000 responses on the
impact of “off” time. Ninety percent of respondents reported they had at least one “off” episode
per day, and nearly half said those events are moderate to severe and cause them to avoid or
stop activity.
The survey is another tool to communicate with payers on the value of these products to
patients. It is no longer enough to say that a drug has sufficient data in a clinical trial and the
endpoints were able to move by a few points. The treatment has to be meaningful. We want to
tell payers that patients may return to work where previously they were confined to their homes.
It is all about better function and more independence.
These therapies are accelerating toward approval, and patients should not have to wait any
longer than necessary. Why, then, should we wait to start the conversations that increase their
likelihood of access?