Accelerated treatments to patients
Through the Drug Repurposing program, one drug candidate completed testing and two more drug candidates went through initial screening. The completed candidate was shown to slightly slow the disease progression in one animal model.
In April, Otsuka Pharmaceutical Co. Ltd. announced that its new drug application (NDA) for tolvaptan had been accepted for priority review by the U.S. Food and Drug Administration (FDA). It was the first time that a drug specifically targeted to alter ADPKD progression in humans was shown to be of benefit, which was an exciting milestone for the PKD community.
In June, we released a Request for Application (RFA) for research grant proposals. Grants awarded will be announced early this year. We anticipate that 12, two-year grants will be awarded for $80,000 per year (per grant).
Also in June, the updated PKD Outcomes Consortium (PKDOC) briefing book (preliminary submission package) was submitted to the FDA and the European Medicines Agency (EMA) for review. In November, we received approval from the FDA to proceed with the full submission once additional required data analysis is completed. We anticipate this final submission in the spring of 2014. PKDOC is critical because FDA approval of total kidney volume (TKV) as an indicator of disease progression in PKD provides a clear regulatory pathway for future clinical trial development.
In late August, Otsuka received a Complete Response Letter (CRL) from the FDA. The FDA issues CRLs to convey that their initial review of an NDA is complete and they cannot approve it in its present form. According to Otsuka, the FDA requested they provide additional information. The FDA and Otsuka are discussing next steps.
We funded and/or attended the following conferences: National Kidney Foundation Spring 2013 Clinical Meetings; Federation of American Societies for Experimental Biology (FASEB) Summer Research Conference; ARPKD Clinical Consensus Meeting; International Pediatric Nephrology Association 16th World Congress in Shanghai, China; and American Society of Nephrology (ASN) Kidney Week. These PKD-relevant scientific and clinical meetings bring together PKD scientists from around the world to discuss current findings and encourage research collaboration.
Educated you about PKD
We held five free education webinars, which were presented by experts in the PKD community. Topics included nutrition for PKD patients, understanding your lab results, tolvaptan and other clinical trials, empowering PKD patients and the Affordable Care Act. Watch the webinars >
We posted 24 education videos to our YouTube channel, which have received more than 11,400 views.
We mailed 1,110 information packets to those just diagnosed or looking to learn more about PKD.
In March, we hosted our online national conference, "What's New in PKD: A Day of PKD Learning." Researchers and clinicians from the Kidney Institute at the University of Kansas Medical Center shared the latest information in PKD research, clinical trials, our Accelerating Treatments to Patients initiative and more. Twenty-six local PKD Chapters participated in the event.
In April, we sponsored the first national conference for parents and caregivers of children living with PKD and other cystic diseases. Nearly 20 families attended the two-day conference, PKD/CHF & Other Cystic Diseases: From Diagnosis to Daily Living, covering various topics including: daily living with cystic kidney disease, nutrition, dialysis, transplantation and treatment options.
In November, we opened our PKD Discussion Forums for PKD patients, parents, friends and family, dialysis patients and those preparing for a transplant. This feature on our website lets you engage with others, ask questions and share stories and experiences. Some discussions have already started, including: living with PKD, PKD parents, caregivers, dialysis and transplants. Join the conversation >
In November, we announced that the National Convention is back by popular demand. The Convention will be held June 20-22, 2014, in Kansas City, Mo., where the PKD Foundation is headquartered. See the five tracks that will be offered and sign up to receive all convention alerts.
United to fight PKD
With the help of nearly 11,000 participants, we raised more than $1.85 million for the 2013 Walk for PKD. View the Walk photos >
One hundred thirty-five passionate runners joined our Run for PKD program. Together, they ran more than 49,000 miles and raised $167,708.
- We added three new stops to the Marathon Series including the Bank of America Chicago Marathon, Marine Corps Marathon and Portland Marathon.
- We launched a new Half Marathon Series for spring 2014, featuring the NYC Half Marathon and San Francisco Rock 'n' Roll Half Marathon.
Our 2013 Matching Gift Challenge doubled donors gifts between May 1 and June 30, and we were overwhelmed by the response! Nearly $230,000 was given by donors like you — in addition to the $100,000 of matching funds from generous friends of the PKD Foundation.
In June, we received nearly $1.7 million from Laverne Duvall, which is the largest estate gift the Foundation has ever received.
Fifty-five fundraising events raised more than $405,000 for PKD. Some of these events included the Celtic Marketing Golf Tournament, Kansas City Cocktails for a Cure and Miami Kidney Casino for a Cure.
In December, we rebranded the "Your Local Event" program as Do It Yourself (DIY) for PKD: Fundraise Your Way. This program allows supporters of the PKD Foundation to take the fight against PKD into their own hands by organizing a local fundraiser.
PKD Foundation volunteers brought the mission of the organization to life by giving their time and passion. Together, 297 volunteers donated 11,613 volunteer hours. Interested in volunteering? Learn more >
We launched a new PKD Chapter in northeast Pennsylvania. We also renamed our virtual ARPKD Chapter to PKD Parents Chapter to be inclusive for parents of children with ARPKD, as well as ADPKD.
Elevated the patients' voice
In June, 55 advocates, representing 18 states, conducted 65 meetings on Capitol Hill during our United on the Hill event. At this legislative and policy conference, Foundation leaders and PKD advocates met with members of Congress to raise PKD awareness and discuss legislative priorities.
Our United on the Hill advocates served an important role and we are seeing results, including:
- Advocates were able to encourage 13 members of Congress to co-sponsor immunosuppressive legislation.
- Two members of Congress have joined the Rare Disease Caucus.
- Numerous staffers have requested additional information about the number of kidney transplant recipients with Medicare coverage that have lost a kidney when coverage of immunosuppressive medications ends after 36 months.
Advocacy doesn't only happen at United on the Hill. We also sent three Advocacy Alerts to our advocacy group encouraging action on legislative priorities.
Updates were made to the Advocacy Action Center, containing sample emails/letters to customize, talking points, social media tools and links to find local legislators.
Raised PKD awareness
We reached more than 15,000 Facebook fans (a 38% increase over 2012). Read about how we reached more people this year >
We celebrated March's National Kidney Month by participating in our second annual 31 Days of PKD Challenges, daily challenges that helped raise awareness of PKD. This campaign reached more than one million people and engaged more than 46,000 people on Facebook.
On September 4, we celebrated the first-ever National PKD Awareness Day (thanks to Senate Resolution 201). We turned the day into a social media campaign by calling it #PKDAwarenessDay and devoted the day to raising awareness of this life-threatening, genetic disease and the critical need for treatments and a cure. With your help, we were able to spread the word and reach nearly 200,000 people on Facebook.
In November, former Board of Trustees member Bill Brazell's article, "In Between the FDA and Pharma, People Wait for Treatments", was featured in the Atlantic Magazine.
We were a proud partner of the second annual #GivingTuesday, a social media movement to celebrate and encourage more, better and smarter giving during the holiday season. More than $4,000 was raised through our One Day, Three Ways to Give campaign.
We were named one of the top 50 social media influencers in orphan drugs and rare disease by the World Orphan Drug Congress.
We created Voices of PKD, a collection of stories, testimonials, photos and videos that tell the story of living with the disease through the eyes of the PKD community. More than 95 people have shared their voice. Share yours >
Thank you for being with us on our journey to improve the lives of those affected by polycystic kidney disease. We couldn't have done any of this work without you. We look forward to another great year in 2014!
View our 2012-13 Annual Report to see more of what we've accomplished together and how your money was invested.