Righting the Clinical Research "Enterprise"

By Ronnie Todaro, M.P.H., Director of National Programs

When opening the Sunday Review section of the New York Times this past weekend,  I did a double-take as a topic of much discussion here at PDF was featured front and center - trumping commentary on the state of the economy, international relations and the latest political debate.

"Do Clinical Trials Work?," by Clifton Leaf serves as a primer on some of the key issues that prevail within the clinical research enterprise - the tension between scientific inquiry and patient priorities; the odds that study sponsors take when betting on the success of a potential therapy; and the disconnect between those who participate in a study and those who may actually benefit from the treatment being tested. It also serves as yet another wake-up call on why people who are living with a disease, be it cancer, Alzheimer's or Parkinson's, need to be formally included in research decision-making.

I can't help but wonder, would clinical research be in a different state if people who actually had the disease were sitting around the table, eye-to-eye with researchers, study sponsors and federal regulators? Would their presence and perspective change the priorities and processes of clinical research? Would it create the sense of urgency that occurs when the personal is married with the professional?

Of course, we here at PDF would  answer in the affirmative as would our 200 Research Advocates across the country.  The bottom line - we can right this "Enterprise" - but only if we recognize who should be at the helm - the patient.

From PPAC: Parkinson's Isn't Newsworthy ... Until It Personally Touches You

By Peggy Willocks, member, PDF People with Parkinson's Advisory Council

April is Parkinson's Awareness Month. And it won't be newsworthy that I came all the way from Tennessee to New York to participate in this past Saturday's Parkinson’s Unity Walk, where about 10,000 people with Parkinson's, friends and family "walked" through Central Park as a symbolic gesture to raise funding for research.

As a member of the Parkinson's Disease Foundation's (PDF) Advisory Council, it isn't newsworthy that I am also here as a voice of the patient in helping to have better and faster approval of therapies for the debilitating illness. PDF adopts the promise of a cure by creatively incorporating the opinion of those who actually live with the disease into the research program. After all, who wants to find a cure more than the patient? We met with PDF, the oldest established Parkinson's non-profit organization, for the final two days of last week.

But it should be newsworthy that Lizzie Graham was there from across the pond. Graham is Director of Fundraising and Global Communications of the European Parkinson's Disease Association (EPDA) from the UK. Maybe with the two group's collaboration we will make some headway.

What does it take to be newsworthy? Finding a cure would definitely be great news. The gold standard treatment is a pill discovered over 40 years ago. We won't find a cure this year. One reason is the cutting of funding from the NIH budget due to sequestration. Another is the lack of participants in clinical trials for Parkinson's.

It isn't newsworthy that this year marks my 20th year with the disease. But it may be newsworthy that this is my 13th year of having been one of six people in the world who allowed transplantation of retinal cells into my brain from a donor eye in hopes of producing dopamine, the chemical my brain lacks. However, the trial was halted several years later due to not meeting its endpoints.

Parkinson's is more than a tremor or immobility. Many are plagued with non-motor symptoms such as depression, psychosis and dementia, creating an economic burden of over $14 billion. Living with Parkinson's isn't a death sentence; it is a life sentence.

I hope I live to see the cure - now that would be newsworthy.

Peggy Willocks of Johnson City, TN, is a member of the PDF People with Parkinson's Advisory Council. Learn more about her work in the Parkinson's community by reading her biography on PDF's website here.

The Doctor Is "In"... Your Living Room

Yesterday kicked off an exciting and intensive two-day gathering of researchers, health care providers, administrators, patient organizations, people with Parkinson’s and care partners on Long Island, NY. We gathered for the 2013 Merinoff Symposium, “Leveraging Telemedicine to Deliver the Highest Quality of Care to All Parkinson’s Patients,” hosted by the Feinstein Institute for Medical Research at North Shore–LIJ Health System and co-hosted by PDF, other national Parkinson’s organizations and telemedicine organizations.

Merinoff Symposium on Thursday, April 11

We are charged with exploring how telemedicine can enhance care for those living with PD, and creating a call to action for the future.

What is telemedicine? Also referred to as “virtual” office visits, it means being able to see your doctor or other health care professionals remotely, using your computer. You both would be able to see and talk to each other using video.

The common perception is that telemedicine is a futuristic tool used only when people live in remote rural areas, miles away from a movement disorder specialist, neurologist or primary care physician. What was made crystal clear by the end of day yesterday is that telemedicine, or what may be more accurately described as technology-enhanced care, has broader potential to transform care and services for all people PD, regardless of where they live. This transformation will result in providing people living with PD the care and services need, when they need it.

This point was driven home by the symposium’s panel of people with Parkinson’s and care partners. This panel was organized by PDF, moderated by our Executive Director, Robin Elliott and included PDF Research Advocates Jay and Marilyn Phillips. The panel was spot-on in identifying the range of issues that can be addressed by telemedicine - lack of access to core services and resources, challenges to managing the multiple medical visits across specialty areas, limitations of three-month or six-month physician visit intervals in providing an accurate picture of the challenges of living with the disease and the inability to participate in a clinical study that is too far away.

The panel was also essential in helping symposium attendees gain a deeper understanding of how partnering with patients can accelerate the adoption of telemedicine and reap the benefits at a faster rate than what would occur without this partnership. Panelist Steve DeMello discussed the need for patients to be engaged in “creating the science” and urged the audience to move away from a model where providers, “watch me do things and then tell me things.”

This theme of patient engagement is near and dear to the heart of those of us here at PDF, where we have been advocating and pioneering ways for people with PD and care partners to work in partnership with the research community to bring about treatments at a faster pace. Our Parkinson’s Advocates in Research program has trained 200 Research Advocates around the country through our Learning Institutes. These Advocates, along with many others with PD, are ready to join forces with health care providers, researchers, patient organizations, administrators and policy makers to create a new way of doing business when it come to living with PD in the present and determining the future.

It is time to embrace and act on the invitation of panelist Jay Phillips when he said, “we are here and we are ready to work with you!” It is time that we recognize the value and necessity of this outstretched hand and how essential the offer of this partnership is to the success, not only of telemedicine, but to health care and research as a whole.

Parkinson’s and the President: How Does the $100 Million BRAIN Initiative Announcement Impact our Community?

Dr. Francis Collins, Director of the National Institutes of Health (NIH), introduced him as the "Scientist in Chief."  And he introduced himself as "the kid who had trouble with high-school physics."  But there was no mistaking the passion for his subject of the man who stood in front of us in the East Room of the White House this morning, announcing a major federal initiative in brain science.

It was President Obama at his rhetorical best, mixing easy banter with a deeply serious expression of his commitment to the brain research initiative that he described as the "next great American project."

When he described the pathos of someone watching "a beloved family member slip behind the mask of Parkinson's disease," you could practically feel a shudder of recognition among the normally restrained scientists, government officials and advocacy group leaders like myself who packed the room.  Perhaps "Empathizer in Chief" is more to the point; the guy really seems to mean it.

So what does this mean for Parkinson's?  We won't know for sure at least until the President delivers his budget message to Congress next Wednesday, and possibly not even then.  But the overall strategy of the BRAIN Initiative is clear: to use the resources of the federal government and private sectors to find out more than we know now about how the brain works – specifically, how the various parts connect with one another, and in what patterns.

All of this could have exciting implications for finding the solution to Parkinson's disease.

As Dr. Eric Kandel, the Columbia University scientist who won the Nobel Prize for his work on memory, said to me at a reception following the event, "this is an historic moment!"

We'll see.  There are several big questions to be answered, beginning with money:

• The President is calling for initial spending of $100 million in the year that begins October 1, 2013, but it is far from clear how much of this will be new money and how much will be existing funds repackaged for the new initiative.
• Then there's the matter of who spends it and how between the NIH, the Defense Advanced Research Projects Agency and the National Science Foundation.  And there will be others.
• Lastly, how will patient advocates be involved? PDF certainly supports this initiative’s collaborative nature, utilizing federal research agencies and private scientific organizations.  We urge the President to expand this commitment to include patient advocates as we have done through the Parkinson’s Advocates in Research program.  After all, the people affected each day by Parkinson's disease are critical to our goals of finding better treatments at a faster pace.

But the overall picture is encouraging -- even exciting. This the first time that the President, after years of slogging through the thickets of the nation's fiscal crisis looking for an exit, has made a major statement about medical research, comparing the challenge -- and the potential for its success -- with the moon shot of the 1960s, the development of the computer chip in the 1970s, and the exploration of the human genome of the 1990s.

The common thread through all of these triumphs was what he described as the American "genius for innovation," an outgrowth of a national character of "dreamers and risk-takers."

"How can we afford to do this?" he asked.  Then he answered himself: "How can we afford not to?"

To learn more see PDF's official statement on our website here.

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?