This initiative is an outgrowth of the recruitment of a "patient representative" by the FDA to participate in an Advisory Committee to review deep brain stimulation.  Drawing on longstanding patient representative activity in AIDS and cancer, The Office of Special Health Issues (http://www.fda.gov/oashi/home.html) at the FDA has offered training and consultation to cultivate this role.  A series of meetings with the Director and staff of the Division of Neuropharmacological Drug Products and with the Director and staff in the Center For Devices and Radiological Health at the FDA have confirmed the receptivity of the key government officials to include patient input in their processes. These meetings as well as consultations with PhRMA (http://www.phrma.org/), the primary trade association for large drug companies, and a number of pharmaceutical companies, and with other patient representatives, provided the basis for an "independent patient advisors" concept. 

FDA

The "independent patient advisors" concept has been proposed to FDA to provide a mechanism for neurology patient consultation to both the sponsors of new drug applications and regulators as an independent voice for the patient's perspective in the pre-approval interactions betweenthe FDA and drug companies. Because the independent patient advisor concept is breaking new ground in the agency and the Division of Neuropharmacological Drug Products at the FDA, a formal process of review and consultation with all neurological groups is considering how it can accommodate this increased patient participation. As a first step, a workshop to discuss patient representation in the process of CNS drug evaluation is being planned by the FDA to incorporate the views of a wide range of neurological groups.

Sponsors

The Parkinson's Pipeline Project patient advisory activity has been initiated with two bio-tech firms working on development of neuro-protective medications.  One of those companies is Guilford Pharmaceuticals, Inc. (http://www.guilfordpharm.com/) , who has re-acquired the rights to develop the neuroimmunophilins(1) that were formerly licensed at Amgen.  Guilford has been very interested in working closely with the PD community in its development efforts.  A three-person Patient Consultant Panel was formed for Guilford.  The Panel will serve in a liaison role to the PD community and advise Guilford on the conduct of additional studies to bring these promising compounds to market.  Another company Cephalon, Inc. (http://www.cephalon.com/) is planning a large clinical trial on a neuroprotective compound. 

Research Partners

The first steps toward creation of the Partners network are being taken in a joint effort of the Parkinson's Foundation and The Neuroprotective Clinical Trials Program of NINDS/NIH (National Institute of Neurological Disorders and Stroke, http://www.ninds.nih.gov/).  The project will establish a limited network and rigorously evaluate the impact of patient advocates on recruitment, retention and protection of human subjects in clinical trials.

Data Tracking

To support the goals of the project, a workgroup has been formed to create an extensive database and tracking system.  This are being designed to monitor the development of PD drugs and other new treatments in the pipeline using publicly available information.  There are more than a dozen members of the design team, including two from Europe.  The UK member has made initial contacts to extend this project to Europe and their medical regulatory agency, EMEA (The European Agency for the Evaluation of Medical Products ( http://www.emea.eu.int/ ).

Project Resources

With leadership from The Parkinson Foundation the concept and three part program structure has been developed to offer the patient perspective to sponsors and the FDA on design and conduct of clinical trials and to organize a national network of patient advocates to facilitate clinical research in the field.  Some components of the plan are in place, but the program is still developing. 

The work to date is primarily the voluntary efforts of talented PWP who want to make a contribution.  Initial implementation efforts are concentrated on establishing the Patient Advisory services and tracking development of new medications to support the advisory activity.

Funds are being sought to round out the resources needed to make the research tracking database a reality and to expand the other component of the Parkinson's Pipeline Project - "research partners". The establishment of Research Partners in research settings is a task that requires sustained effort of a core paid staff, as well as volunteers,  to keep up to date on the scientific development in the public domain and in private industry.  Seed money is needed for the approximate six-month time-frame to pay for the start up costs of organizing and training the network of Research Partners, and to develop the systems to effectively manage the network.