Pipeline Concept The term "pipeline" is a way to describe the time and effort devoted to processes of translation and application scientific discoveries to the delivery of more effective treatments for the benefit patients. The introduction outlines the context and rationale for involvement of patients in the process of evaluation of new treatments.

Goals and Program Elements The goal of the Pipeline Project is to facilitate evaluation and approval of new treatments for Parkinson's  Disease (PD).  The intention is to add the unique patient perspective, insight and credibility to the process of development of safe, effective and timely new treatment.  The goal will be attained through collaboration with sponsors and researchers for Investigational New Drugs (IND), or other treatments and with the FDA and the EMEA.  The project has three integrated components:  *     Consultation to clinical researchers and human subjects in clinical trials *     Consultation to sponsors of clinical research and regulatory agencies *     Tracking data base for New Treatments

Pipeline Project Status Update  September 2002

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Parkinson's Research Partners  Activities to Facilitate Clinical Research A key component of the project is the effort to network PD patient advocates. Research Partners, who will work on the front line in clinical research settings.  The advocates will perform a number of functions aimed at facilitating research.  Drawing on our most valuable asset, the people with Parkinson's, the idea is to create an effective national grassroots network of PWP's.  These people will serve as research ombudsmen in the clinical trial setting to facilitate the process of research and development of new treatments.  In general, the advocates will put the face of the patient, and the urgency that we feel, in contact with our various constituencies.  Drawing on the unique perspective and credibility of PWP, these Research Partners will be available in key clinical research settings to:  1. educate doctors in the community on referrals to research programs,   2. educate/counsel patients and recruit to clinical trials, and   3. educate researchers on patient perspective.   Go to presentation of details of the Pipeline components to facilitate clinical research

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Patient Advisors to Sponsors and Regulatory Agencies Activities to Facilitate Evaluation and Approval of New Treatments For sponsors of IND's, we offer PWP Advisors (members of the Parkinson's Pipeline Project team)  and/or help to establish "Community Advisory Panels" (similar to the AIDS panels) to assist the companies with elements of the design of clinical trials pertinent to our expertise and perspective.  These panels will in turn provide a channel for information from the company to the PD population.  Our intention is to be a strong independent voice with respect to both the sponsor and the FDA for the needs of potential research subjects and patients generally starting as early as possible with FDA and the patient population. We also provide independent advice to the FDA, similar to oncology, where advocates are now being recruited to serve as patient advisors in the pre-approval, clinical trial phase of cancer drug development.  Patient advisors provide guidance to the FDA and to the drug sponsor on topics such as clinical trial design.  More flexible regulatory mechanisms, such as accelerated review and fast tracking procedures, introduced into the FDA process for serious and life threatening illnesses, including PD, can also be influenced by patient advisors and outside patient groups.  The Director of the Neuro-Pharmacological Products Division of the FDA has agreed in concept to recognize the patient advisors as a independent role provided that appropriate confidentiality agreements are in place.  These agreements will allow direct communication between FDA and the PWP Advisor. Independent Patient Advisor Concept.   Patient Advisors establish a formal working relationship with the sponsors with a confidentiality agreement that allows the sponsor to share proprietary information under SEC (Security and Exchange Commission) regulations. In order to maintain independence and credibility with all involved parties, no fees will be charged by the Patient Advisor for consultation, other than reimbursement for reasonable expenses.  If the confidentiality agreement includes language acknowledging the independence of the Patient Advisor as an advocate for the patient perspective, not as an agent for the sponsor, it allows the FDA to consult independently with the Patient Advisor and gives an added degree of credibility to advocacy positions taken. This concept has been accepted in principal by key officials at FDA, but because it is breaking new ground in the agency, the proposal will undergo a thorough review.    Go to presentation of details of the Pipeline components to facilitate regulatory decisions

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Database tracking system on new treatments in pipeline Knowledge and Information Systems Support To support the goals of the project,  an extensive database and tracking system is being created   to monitor the development of PD drugs and other new treatments in the pipeline using publicly available information sources. Data are also collected directly from patients and field research partners. The database includes information on the financial status and business environment of the sponsoring organizations,  information on the science and history of development of the medication,  information on the design of clinical trials and criteria for participation, and collected on the experiences of trial participants.  Staffed in part by volunteer PWP tracking ‘partners,’ the goal is to make this data base the most complete, up to date source of information on clinical trials for PD. This goal is attainable because, as patients, our trackers are numerous (we extend cooperative membership to all PWP who participate in on-line information sharing), highly motivated, and narrowly focused (on 2 dozen new treatments). Because we solicit data directly from patients and our field staff to clarify the patient perspective on the study, we are well informed to represent the patient's views to sponsors of clinical trials and regulators (FDA, EMEA).  As the most complete source of information on clinical trials, the data base and the communications from it (e.g., website presentations) will be a draw to identify motivated patients who are seeking information on trials as potential research subjects. Education and communication:  The tracking data are utilized to inform partners as well as the public about clinical trials and pipeline treatments. The core program staff will provide initial training and a continuous flow of information to the field. Information will be provided through our secure website (www.pdpipeline.org) on such topics as the status of research and science, the development of new treatments, and patients’ reactions to specific clinical trials.  As much as possible, basic topics such as the FDA approval process will be packaged into self paced learning  modules that can be accessed on-line.  Go to presentation of details of the Pipeline components for tracking new treatments

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