PCD KS FOUNDATION

1. The primary goal of the PCD Foundation is to accelerate access to medical research to develop better therapies and cures to improve the quality of life and health of those affected by primary ciliary dyskinesia.
2. The PCDF believes affected individuals and their families are full partners in the research process and strives, through educational initiatives, advocacy and awareness activities, to empower affected individuals and families to be their own best advocates.

The Path to Clinical Trials

The Path to Clinical Trials (PTCT) is the main focus of our Research Support Program. The PTCT consists of two critical infrastructure components needed to support clinical trial participation in PCD:

1. PCDF Clinical and Research Centers Network PCD‐CRCN or just CRCN (list of sites attached) The PCDF CRNC is modeled after the highly successful Cystic Fibrosis Foundation Care Centers. Each multi‐disciplinary site is required to meet stringent criteria for the diagnosis of PCD and to follow PCDF Consensus guidelines published in Pediatric Pulmonology in 2015 (attached). The sites undergo an accreditation visit and are evaluated by PCDF personnel and by clinical site reviewers. The CRNC currently has 29 sites in various stages of accreditation.

2. North American PCD Registry (NAPCDR)
Our primary focus in the immediate terms is funding the NAPCDR. The NAPCDR is modeled after the Cystic Fibrosis Registry, with the goal of collecting reliable core data from our clinical sites. Additionally, we hope to incorporate a patient‐report component to supplement and inform the core clinical data. The need for reliable clinical data on PCD is critical and finding resources to support the development of the NAPCDR is a top priority.

'PCD on the Move' Annual Scientific Conference

Our annual scientific conference is another component of our Research Support program. This meeting brings together top PCD experts from around the world to discuss cutting edge PCD research. It also serves as a training meeting for new investigators and clinicians.

ATS/PCDF Partner Grant

The PCDF partners with the American Thoracic Society's (ATS) Partner Grant program to provide research grants to meritorious applications received by the ATS.

Program Area 2: Patient Services

The Patient Services program encompasses all aspects of patient community outreach, including educational materials and meetings, online and social media outreach, website maintenance, community fundraising and volunteer management. Additionally, Patient Services receives and routes incoming patient inquiries and provides referrals to clinical and/or social services specialists.

Working with the Clinical Centers program, Patient Services organizes and hosts 'Clinical Center Awareness and Regional Education' (CARE) Days in conjunction with our site visits to introduce local patients to their PCD center and reinforce the value of expert care for PCD.

Working with internationally-recognized experts in PCD who comprise our medical and scientific advisory council (MSAC), we assess needs and prioritize research goals and initiatives. We established, maintain and accredit a growing network of expert centers (PCDF Clinical and Research Centers Network--CRCN) that partner with us to identify gaps in knowledge and care that can be addressed by expert consensus or by research. Our PCDF CRCN sites serve not only as the focal point for continuous two-way feedback with the PCDF, they will also be authorized to input and access high-quality clinical data, bioinformatics and patient reported outcomes through a PCDF-sponsored registry program currently in development. Finally, we facilitate interactive communication with our patient community through on-line support and education sites and by in-person family educational events called PCD CARE (Clinical centers awareness and regional education) Days.

We have established a North American clinical and research network comprised of top academic and pulmonary research sites in the US and Canada and created an evaluation and accreditation system to ensure quality control. Our high-quality network is recognized by US News & World Report, which factors the presence of a PCDF accreditation into their annual scoring of US pediatric pulmonary hospitals. We have sponsored major scientific conferences since 2002 and collaborate with researchers and PCD patient advocacy groups internationally. We partner with the American Thoracic Society (ATS) on PCD-specific grants. We host an annual scientific conference that has become an international and have published PCD Consensus Statement for distribution to non-expert clinicians treating patients with PCD. We maintain an educational website for patients and provide online support for the community.