Parkinson's Clinical Research:Changing the Way Business is Done
By Robin Elliott
In 2004, the Parkinson’s Disease Foundation (PDF) launched Advancing Parkinson’s Therapies (APT), an initiative designed to help accelerate the development of new therapies for people who live with Parkinson’s disease (PD).
As we reported exactly five years ago in this newsletter, a primary obstacle to therapy development — and one on which we thought we could have the greatest impact — was the low participation in clinical trials among people with PD.
So, when we recently interviewed various colleagues who are involved in the therapy development process to hear their perspectives (as reported later in this article), we were not surprised to hear each one of them identify clinical trial recruitment as an ongoing barrier to therapy development. But we were interested to hear them identify additional roadblocks of an institutional nature, and speak of collaboration and inclusion of people with PD as potential solutions.
This is because over time our own thinking has also evolved. We have come to realize that while low recruitment is an important barrier to address to find new therapies, it is also symptomatic of more complex issues that require our attention.
So, what is our solution? At PDF, we believe that we can no longer afford to see people with Parkinson’s disease as simply vehicles through which to test new medicines.
Rather, we should see people with PD as primary partners in the processes of therapy development, trial design and implementation and peer education. Our panel’s responses tell us that we are coming closer to this ideal, and this news comes at an auspicious time. PDF’s third Clinical Research Learning Institute in October graduated 40 people with Parkinson’s newly-educated in the theory and practice of clinical studies. This brings to more than 100 the number of members of what we will now be calling the Parkinson’s Advocates in Research (PAIR) program of PDF.
As these trained advocates partner with government, scientists and industry, we hope to increase knowledge, trust and transparency among the PD community. Look for more details in the Spring 2011 newsletter. And join with us in changing the way business is done — a new way that ensures a place for people with Parkinson’s, from all walks of life, at the center of the clinical research process.
Mr. Elliott is PDF’s Executive Director.
Danna Jennings, M.D.
Clinical Research Director at the Institute for Neurodegenerative Disorders (Connecticut)
Investigators play a key role in identifying the questions that need to be addressed, designing the research protocols, monitoring the safety of participants and reporting on the results of a trial.
Petra Kaufmann, M.D., M.Sc.
Director, Office of Clinical Research, National Institute of Neurological Disorders and Stroke (NINDS) (Maryland)
NINDS funds clinical research to test experimental therapies for PD, and gathers stakeholders to talk about issues that are important to the community and that can help increase trial efficiency.
Person with PD
Paul Zimmet, D.D.S.
PDF Research Advocate (Class of ’09)
Retired dentist (Virginia)
People with PD play an important role as participants in research studies and can serve as advocates to improve the development of therapies for PD.
Kimberly A. Moran, Ph.D.
Associate Medical Director — CNS
UCB, Inc. (Georgia)
Our goal is to develop safe and effective medications that directly address unmet needs in PD, for example, the non-motor symptoms such as anxiety, depression and gastrointestinal symptoms.
PDF: What obstacles do you see in the development of Parkinson’s therapies?
DJ: The pace at which we are able to recruit people living with Parkinson’s to trials. Problems with recruitment lead to delays in completing trials. These delays, in turn, not only increase costs, but also slow down the pace at which new medications can be developed. We realize that participating in research is not for everyone. We are always grateful to individuals with PD who are proactive about getting involved. As professionals, we also need to be more proactive about informing our patients and the PD community about existing opportunities to participate in research.
PK: Participation in trials. Slow recruitment of people with Parkinson’s slows down research. If we could speed recruitment, we could speed up the development of new therapies. Another obstacle is the lack of a good biomarker. A biomarker is, for example, a blood test or brain image finding that could help us decide early on in a trial if a new treatment has promise. Right now, if we want to assess the effects of a new treatment, then people with Parkinson’s have to stay in trials for a long time. A biomarker could shorten that time.
PZ: First, lack of awareness among people with Parkinson’s about the variety of clinical trials and second, the pace of the drug approval process. Some people see trials asking for participants who are not on any medications yet, and think they do not qualify for any trials. But there are many other ways to participate, such as studies on the therapeutic value of exercise. In terms of drug approval, the time it takes to get it — seven to 10 years — is too long. We need to push medications through the pipeline more quickly.
KM: Inefficiencies in the drug development process. As a scientist, I have been privy to some great discoveries that will help us understand and treat PD. But only one in 10 molecules actually becomes a new drug. Problems arise when translating discoveries made in the lab to clinical trials that may benefit people living with PD. For example, scientists have discovered targets in the brain that play a role in PD, but we haven't narrowed down the right therapy to attack that target. Also, difficulties are observed when designing the correct trial design for a specific PD population (i.e., early or advanced PD).
PDF: What improvements are you or others working on to tackle these obstacles?
DJ: One of the best developments that has occurred in terms of education and recruitment is the development of accessible web-based sources, such as the website http://www.pdtrials.org/, that provide comprehensive information about studies to people with PD. I also think that strengthening the relationship between the foundations that are behind the research, and the people with PD themselves, can be very helpful. Improvements in outreach and education to neurologists in the community are also important mechanisms for spreading the word about research opportunities.
PK: We are working to increase trial efficiency by supporting the infrastructure of the process — for instance, through the grants we make to clinical research centers. (PDF does similar work in its support of the Parkinson Study Group.) We are also launching a biomarker initiative for Parkinson’s disease. This will be an important step towards accelerating trials, so we hope that people with Parkinson’s disease will participate.
PZ: Part of our work as research advocates is to improve awareness and participation in trials through community education. We can do this by going out into the community and saying, “please consider getting involved!” I work with people living with PD, support groups, neurologists, and others in my community to accomplish this, and I have found that the personal touch makes a world of difference. Research advocates are also trained to provide input on studies, so I was able recently to attend a meeting at the National Institutes of Health (NIH) that addressed questions of Parkinson’s trial design.
KM: Collaborative opportunities are the way to improve the process. My company is looking for collaborative relationships — with companies, academic centers, health care providers and patient organizations — to take efficiency to a new level and bring medications to people sooner. For example, we try to guide scientists who have developed new molecules to translate them into medications. We partner with nonprofits to increase awareness. Trials can move no faster than the pace at which they are able to enroll participants, so getting word of appropriate trials to the appropriate people with PD is key to their success.
PDF: How are people with Parkinson’s part of the process?
DJ: We work regularly with a group of people living with Parkinson’s. They help us by brainstorming ways to improve clinical trial recruitment and education efforts. We ask: what are the barriers to getting people to participate in our trials? This group may tell us that certain factors — for example, the lack of transportation to and from the study site or the need for a better understanding of what trial participation requires — play a role.
PK: We make it a practice to engage people with PD in our work because they, after all, are the key stakeholders. For example, we invite people with PD to speak with us about their experiences in trials. I was a faculty member for PDF’s Clinical Research Learning Institute, which is another way for NINDS to find out how people with PD view the research process. We encourage the inclusion of people with PD in trial planning, and on data safety monitoring boards. With PDF’s training, people with PD can now be more equal partners in the process and can educate others.
PZ: PDF research advocates are finding places on formal advisory boards to ensure the input of people with PD in trial design. I was able to join an Institutional Review Board at Georgetown University, where along with researchers and doctors, I review 15–20 clinical trials a month. My job is to provide the patient point of view, and I do believe that my presence helps. We are trying to increase the representation of research advocates across the US — the more people that we have as advocates, the better chance we have to assure that the goals and results of trials are made transparent to people with PD.
KM: There are many ways people with Parkinson’s can play a role in studies. Right now, we have a person with Parkinson’s at most of our meetings, which provides us with the context that reminds our staff why we are here. My grandfather had Parkinson’s. I like to think that my work every day is potentially benefitting people living with PD and that we are working as fast as we can to provide better medications and to educate doctors so they know the best way to treat Parkinson’s.
Play a Part in Research
Join our research advocates in making a difference in the development of Parkinson’s therapies:
- Get the Facts: What should you know? Test your research IQ, find the facts and order our publications, so you know your rights before getting involved.
- Find a Trial: Ready to participate? Search for a trial by city, symptom or keyword by visiting www.PDtrials.org.
- Become an Advocate: Use our tips to become part of the movement ... educating your community and ensuring the voices of the Parkinson's community are heard.
- Learn More