Statement of Need:
The PCD Foundation (PCDF) recognizes that establishing credible, evidenced-based knowledge of PCD is the most important factor in fulfilling our mission to provide education, support and better therapeutic options to affected individuals. To accomplish this, we acknowledge the need to accelerate research efforts. To date, these efforts have been hampered by:
- Small, geographically dispersed and non-cohesive patient group
- Lack of interest in/knowledge of PCD on the part of clinical researchers and industry sponsors
- Difficulty obtaining diagnosis
- Woefully inadequate funding
Our approach to overcoming these difficulties has been somewhat scattershot, attempting to organize the patient group into a cohesive whole through voluntary participation efforts, reaching out to clinical researchers and industry representatives in a reactive rather than proactive manner, attempting to funnel patients into centers that can reliably diagnose rather than pushing for improved diagnostic standards in general, and functioning with a chronic lack of financial resources inadequate to cover even our scattershot efforts.
Proposal for a New Direction: A Path to Clinical Trials
Based on the above and on advice and recommendations from our clinical advisors, research partners at the NIH and non-profit colleagues who have successfully achieved similar missions (Bob Beall at the CF Foundation, Sue Byrnes at the LAM Foundation), the Executive Committee of the PCDF met in Buffalo, New York on December 6, 2009 and agreed to organize our current program areas--research, education and advocacy—under the rubric of a “Path to Clinical Trials” starting in 2010. This initiative would serve as the foundation for building stronger relationships with clinicians, patients and industry; help us provide better information and support services for our community and allow us to maintain a laser-like focus on a crucially important program area. Having clear cut goals and an identified path for achieving them will also aid in development efforts, making us a more appealing potential recipient of donated and/or grant funds.
This program would consist of:
- Identifying centers to serve as clinical/research sites
- Developing a clinical registry tool to entice identified centers to participate
This model will encourage:
- The development of evidence-based standards for diagnosis and clinical care based on data collected in the registry
- Identification of areas where research is needed to fill gaps in evidence
- Cooperative referral between participating sites and the PCDF
- Industry interest in a well-categorized and easily accessed patient pool for corporate-sponsored trials
- Patient participation in the activities of the PCDF when they see direct applicability of these activities to their lives
First Steps on the “Path”
A. Determine requirements for a PCD registry site:
- Size of potential PCD population (basic demographics—an effective clinical site needs to follow at least 20 patients per Bob Beall at the CF Foundation and Sharon Dell at Sick Kids in Toronto). Start with our current GDMCC sites and one or two additional options and expand from there.
- Access to diagnostic technology and expertise
- Does the facility have an electron microscope used for clinical pathology?
- Are there trained technicians and pathologists familiar with primary vs secondary defects?
- What criteria do they use when analyzing EMs?
- Do they have the ability to check nasal NO?
- Do they order PCD genetic tests?
- Are their clinical practices in keeping with recommendations published by GDMCC clinicians and the ERS guidelines? e.g.
- Sputum and pulmonary function checked every 3-6 months
- Aggressive use of antibiotics for infections
- Importance of airway clearance emphasized?
- Are they willing to work collaboratively with the PCDF and with the existing network of sites?
B. Establish PCD Clinical Registry Parameters
- Determine what data we want to collect, what would be most appealing to researchers and industry representatives and what collection systems would have the most credibility for use by our target audiences. The CF registry is a great model.
- Compare our data collection goals with existing data collection efforts to avoid ‘reinventing the wheel’ (GDMCC, European efforts, etc—ideally data points would correspond so no data is ‘wasted’).
- Establish data entry, retrieval and analysis protocols and provide stipend for participation (CFF pays $300 per year per patient to help defray cost of registry administration. This cost can be budgeted in our funding proposals).