Are You a Driver and a Partner? Notes on ASENT and Patient Collaboration

Two Saturdays ago, in a Washington DC hotel, PDF pulled off an interesting little coup in its long-term bid to bring people with Parkinson’s (and other neurological disorders) into the center of conversations about the process of clinical research and drug development.

 The occasion was the plenary session on the last day of the annual scientific conference of the American Society for Experimental Neurotherapeutics (ASENT), and the title of the session was: New Models for Collaboration: Patients as Drivers and Partners in Neurological Research. I have served on the Board of Directors of this very worthwhile organization for three years now, and they asked me to help put together an expert panel on this important topic.

Note that the title of the panel referred to patients and clinical research participants as Drivers, and Partners.  Not as Research Subjects, or Attentive Audience Members – important as these roles are in the right context – but as Drivers, and Partners.  In other words, as full players in the process, shouldering their way up against the other weighty and recognized players in clinical research such as the scientists, the government regulators, and the industry collaborators (e.g., drug companies and biotech firms).

Why is this so important?  The answer is that the needs, opinions and requirements of people who live with neurological disorders have an absolutely crucial role to play in the way clinical research in the United States is organized, conducted and evaluated.  What should we be measuring in a clinical trial (often described as “outcome measures”)?  How should participants in trials be treated – from the information that is shared with them to the reimbursement for necessary travel expenses that is provided to them?  And how can recruitment be accelerated, and retention secured, so as to assure that each trial is initiated and completed in the shortest possible time – for the benefit both of the participants’ well-being and the company’s pocketbook?  On these and related issues, patients/participants have important things to say, and important opinions and needs to be accommodated.

Members of the panel, who were assembled and directed by my colleague Veronica (Ronnie) Todaro, PDF’s Director of National Programs, were diverse, interesting and eminently well qualified for the task.  Two presentations in particular stood out for me: Dr. Petra Kaufman, M.D., M.Sc., Associate Director for Clinical Research at NIH/NINDS, who presented a brilliant and comprehensive overview of how patient organizations can be involved in recruitment and retention for clinical trials of new treatments in brain disorders; and Dr. Russell Katz, M.D., the long-time Director of the Division of Neurology Products at the US Food and Drug Administration, who listed the many ways in which patients can be involved in the process of drug approval.

There were three things that I found most exciting about the panel.

1. First – this was most evident in the presentations of Drs. Kaufman and Katz - it gave an encouraging and convincing picture of the many ways that patients can get involved in the clinical research process.
2. Second, the experience filled me with hope that the health care system is at last ready to consider how patients can be integrated into the clinical research process, to the benefit of all the major partners and to the lasting assurance of the people who live with Parkinson’s and other neurological disorders.  (One reflection of this was the healthy size of the audience of doctors and scientists that we attracted – on a Saturday morning, no less, at the very end of the meeting!).
3. And third, it gave me a great sense of pride to see how my own organization, the Parkinson’s Disease Foundation, was playing such an important role in this process -- not only in behalf of Parkinson’s community, but of all groups that are committed to solve brain disorders.  (A sparkling reflection of this was the masterly performance as moderator of Linda Morgan, a talented MBA pharmacist who is a leader of PDF’s national People with Parkinson's Advisory Council and one of the first advocates active with our Parkinson's Advocates in Research program).

ASENT will soon be making available the slide-decks of our speakers to a wider audience.  We will keep you posted on this blog when they do.

What are your suggestions and opinions? Are there additional ways in which patients can be usefully involved in the clinical research process?  Do you feel as if you have the opportunity to be a driver and a partner?

A Fond Farewell to an Extraordinary Friend, Paula Wittekind

Paula Wittekind

In the loss of Paula Wittekind, who passed away on November 8, the Parkinson’s community bids farewell to a lovely friend, a fine mind, and a courageous and indefatigable presence. To the end, she fought quietly but effectively against her own infirmities and against the indifference of institutions that she felt could always do more to wage serious war against Parkinson’s. But in all of these ventures – whether it was the legendary Parkinson’s Pipeline Group, which she co-founded, the Parkinson’s Action Network, or my own organization, the Parkinson’s Disease Foundation – Paula served as an effective research advocate, always finding a way to deflect attention from herself and her own needs, to others and the work they were doing to advance the interests of her community.

I last spoke to Paula by telephone a few weeks before she died, finding a rare downtime moment between the visits of pilgrims from various parts of her life who were visiting her home in Cocoa, FL – people like Perry Cohen, Peggy Willocks and other heroes of our community. In a just few minutes, she swept away the sadness and trepidation that I brought to the call and replaced them with feelings of love, respect, a laugh or two, and above all inspiration about how I could be working in her wake a little bit harder, a little bit better … and a lot more meaningfully. What a gift she made to me personally during this single conversation, and how much I value it.

Thank you, Paula, from all of us. May your wonderful and far-flung family treasure their memories of you. And may they know how many lives you touched, how deeply … and with what profound effect.

Remembering Arlen Specter

When I learned of the death of former Senator Arlen Specter two weeks ago, I thought immediately of two people in the Parkinson’s community who I knew would strike just the right chord to pull me out of a mood of regret for the loss of a true champion of medical research in America.

I got it right.

One was of these was Jim Cordy  – a veteran of the 1990’s “Washington Wars” to get Parkinson’s disease recognized – and funded -- as a national priority.  He was a close colleague of Joan Samuelson (read on), the founder of the Parkinson’s Action Network.  And he was best known (in addition to the frogs he raised, and that his wife Deborah tolerated, in their suburban Pittsburgh home) for the hour-glass that he carried everywhere, setting it on the desk or podium before him to symbolize what the passage of time means for someone who lives with Parkinson’s disease.   In Parkinson’s, he would say, every moment counts; every hour drains energy from a productive life; every year is another finger of reproach wagged at those of us who use other, more prosaic, measures of time, like annual budgets or fundraising plans.    

During our telephone conversation, Jim reminded me of the hour glass, and said he remembered how Arlen, then Senator Specter (R, PA) , ranking member of the Subcommittee on Health and Human Services, went to floor of the Senate during the debate on the federal support for research involving embryonic stem cells, referring repeatedly to “Jim Cordy’s hour glass, reminding us that time, for someone with Parkinson’s, is not neutral!”

My other witness was Joan Samuelson, the gifted and charismatic California lawyer who was diagnosed with Parkinson’s disease in her forties; went to Washington during the administration of the first President Bush to fight for federal support of research involving the cells of discarded fetuses; then, in the mid-1990s, founded the Parkinson’s Action Network which now, under the leadership of the Amy Rick, serves as the PD community’s nerve center and power source on public policy issues.

Joan remembers Senator Specter’s unremitting commitment to medical research, the Republican counterpart to the equally indefatigable Tom Harkin (R, IA), his partner on the HHS subcommittee.  Together, they spearheaded the memorable drive to double the NIH budget between 1998 and 2003 – a spectacular achievement  that transformed the capacity of the United States to lead the world’s medical research and pave the way to new cures.  (She also reminded me, with admiration, of Senator Specter’s final legislative battle, in which he almost single-handedly persuaded Congress to add $10 billion to the NIH research budget as part of the stimulus program that President Obama put forward as a means to shake the country out of the 2009 financial crisis.)

As for me, I best remember Arlen Specter – dubbed by his fellow-Pennsylvanian Jim Cordy as the leader of “Arlen’s Army,” a play on the nickname (“Arnie’s Army”) of that other famous Pennsylvanian, the golfer Arnold Palmer – at the memorable Congressional hearing in the Fall of 1999, when the Senator and his friend Tom Harkin presided over a public hearing of the subcommittee on the subject of Parkinson’s disease research.  The hearing featured Dr. Gerald Fischbach, the head of the National Institute of Neurological Disorders and Stroke; Jim Cordy (armed with his hour-glass, of course!); Joan Samuelson; and the star, recently “out” with his PD diagnosis, a winsome, charming, young Canadian-born actor of the name Michael J. Fox.

PDF helped pull this event together and I was never more proud than when I saw this titanic cast playing out the Parkinson’s case before a national audience.  It wouldn’t have happened without Arlen Specter, and the PD community has never been the same since.

Goodbye, Arlen.  You did us proud.

PAIRing up for Research: The Importance of Saying Thank You

Last week, we were excited to see a great example of pairing up (the slogan for our Parkinson's Advocates in Research, or "PAIR" program) at Vanderbilt University Medical Center (VUMC). In an article entitled, "Events honor early patients of novel Parkinson's study," VUMC reports on their study of deep brain stimulation surgery as a treatment for early stage Parkinson's. DBS is approved by the FDA as a treatment for mid stage Parkinson's, but not for those with earlier stage PD.

We were impressed to see how VUMC thanked their volunteers, all people with Parkinson's, who made the study possible. As the article notes, their 35 volunteers did an extraordinary thing by agreeing to either undergo surgery early on in their Parkinson's or to receive standard medicines. Each person also dedicated five separate weeks of their time over the course of two years.

As with all clinical studies, we only learned new lessons about PD because of those volunteers.

Will You "PAIR Up" for Parkinson's Research? 
This story rang true with PDF because VUMC's event reflects values similar to those that drive our PAIR program.  At PDF, we believe that people with PD must be primary partners in research­ as participants, but also in other capacities,­ in order for us to speed new treatments.

The importance of saying thank you is particularly key, because of findings such as those from a 2007 CISCRP survey which say that:

• 79 percent percent of clinical study volunteers report that they never hear from the sponsor or research staff after a trial has concluded, and; 
• 83 percent of volunteers say they are never told the results of their trial

Through PAIR, we are committed to changing this statistic and to ensuring that someone says thank you.  We are doing this in part through our signature PAIR Up for PD Research forums each April that - similar to VUMC -­ publicly thank people with Parkinson's disease who have volunteered for research studies.

    Courtesy of PDF Research Advocate Carolyn Weaver, made for her fellow Research Advocates.

Most of these forums are led by one of PDF's 180 Research Advocates (all of whom have completed one of our in-person PAIR Learning Institutes) in collaboration with local researchers.

PDF Research Advocate Bob Hankin at an April 2012 PAIR Up for PD Research Forum in New York, NY.

The thank you at the forum is usually a simple public recognition along with a small token from PDF (see first and last photos).  But it's an important step in ensuring people with PD are at the front and center of research.

We applaud VUMC's efforts to communicate with their volunteers and say thank you.  

What do you think about their efforts? Will you PAIR Up?

Visit our website to learn more about getting involved with PAIR, through our upcoming training for people with Parkinson's and care partners in Oak Brook, IL or our new online course.