FDA issues draft guidance for TKV
|Monday, August 31, 2015
We continue to make good progress toward FDA support for TKV as a bio-marker for trial enrichment. I recently let you know that the FDA issued a formal letter of support to C-Path's PKDOC (PKD Outcomes Consortium) for TKV as a potential bio-marker in trials of new therapies for ADPKD. This morning, C-Path announced that the FDA has issued draft guidance for this use.
The draft guidance provides qualification recommendations for the use of TKV in ADPKD clinical trials. Once guidelines are finalized and part of the official government register, biotechnology and pharmaceutical companies will be able to design trials with confidence that the FDA will accept TKV as an enrichment of outcomes. These guidelines will also help them find appropriate candidates, potentially improving the accuracy and efficiency of those trails.
In the words of David Baron, Ph.D., Chief Scientific Officer of the PKD Foundation, "This regulatory support is key to encouraging those developing potential treatments for ADPKD patients to carry on their work in what will hopefully be a more streamlined and productive path to potential approval of new therapies."
According to Ronald Perrone, M.D., Medical Director, Kidney Transplantation, Professor of Medicine at Tufts University School of Medicine, Co-Director of PKDOC and PKD Foundation supporter, "The impact of this biomarker qualification will be to enhance the recruitment and performance of clinical trials in ADPKD, and hopefully bring new therapies to patients in a shorter period of time."
The FDA has requested comments to this draft by Oct. 15. The Foundation's strategy is to work with PKD experts to write an official comment as a sign-on letter to submit to the FDA.
The Draft Guidance for Industry document is on the FDA website and the C-Path PKDOC website, where you can also read the full press release.
Congressional Update August 2015
When Congress returns after Labor Day, it will face many difficult issues:
- the Iran deal;
- preventing a government shutdown on Oct. 1;
- increasing the debt ceiling;
- approving a long-term highway and transportation bill;
- elementary and secondary education bill;
- omnibus budget deal;
- completion of FY16 appropriations.
Congress, especially the Senate, will be focusing most of its attention on the bills with the greatest fiscal impact on the nation as a whole. If the PKD community wants to advance its legislative agenda, it is vital that advocates bring our issues to the attention of our elected officials, especially Senators, as soon as possible.
21st Century Cures Act
On July 10, the House of Representatives passed 21st Century Cures Act (H.R.6) by a vote of 344 to 77. Click here to see how your representative voted.
It is crucial that the Senate pass this important research bill in 2015. The August state work period is the perfect time to contact your senators and urge them to support H.R.6. You can go to www.senate.gov to find your senators and then find the closest office to where you live. Learn more about H.R.6.
NIH and FDA Funding
The new federal fiscal year begins on Oct. 1, 2015. The PKD Foundation is interested in the Labor / HHS / Education bill, which funds the National Institutes of Health (NIH) and the National Institute of Diabetes and Digestive and Kidney diseases (NIDDK) and the Agriculture Appropriations bill, which funds the Food and Drug Administration (FDA). At this time, neither the House or the Senate has debated its version of either bill.
For FY16, the administration requested $31.311 billion in funding for NIH. Of that total, it seeks $1.788 billion for NIDDK. The House bill (H.R. 3020) recommends $31.184 billion for NIH and $1.771 billion for NIDDK. The Senate bill (S. 1695) is more generous. It recommends $32.084 billion for NIH and $1.825 billion for NIDDK.
For the FDA, the administration is requesting a total of $4.930 billion. Neither the House nor the Senate bill accepts this level of funding. The House bill (H.R. 3094) includes a total of $4.549 billion and the Senate bill (S. 1800) recommends $4.560 billion.
Congress is not likely to approve the final funding level until late fall. So, advocates still have time to urge elected officials to provide as large an increase as possible in these funds.
FDA Issues Letter of Support for TKV as ADPKD Biomarker
The U.S. Food and Drug Administration (FDA) has issued a Letter of Support to theCritical Path Institute’s (C-Path) Polycystic Kidney Disease Outcomes Consortium (PKDOC) for the use of total kidney volume (TKV) as a prognostic biomarker for clinical trials of new therapies for ADPKD.
The Foundation’s Chief Scientific Officer David Baron explains, “This is important because it shows that the FDA acknowledges variances in the disease progression of ADPKD patients. It means we are increasing the possibility to be able to treat PKD earlier, at a time when treatments can be more effective, rather than after major damage to the kidneys has already occurred.”
TKV is a measurement of the impact of ADPKD on the size of the kidneys and is believed to be an indicator of future kidney function decline. There is no current accepted measure of kidney function that is an accurate marker of disease progression in ADPKD.
PKDOC is a collaboration between the PKD Foundation (the primary funder), C-Path, members of the pharmaceutical industry, PKD researchers and clinicians, and the FDA.
Read the FDA Letter of Support (scroll to bottom of page)
Input Needed From Patients and Advocates Regarding Gap Between Diseases and Treatments
The House Energy and Commerce Committee has launched the 21st Century Cures initiative to draw attention to and close the glaring gap between the number of diseases and the number of treatments available. The committee is seeking input from patients and advocates regarding what cures and treatments are available for individual diseases, how they work with researchers and other patients, their experience with clinical trials and what role government has played.
As you are well aware, there is currently no treatment to slow or stop the growth of the kidney cysts that plague generations of families suffering from polycystic kidney disease (PKD). PKD patients only remedies are dialysis and transplantation once their kidneys fail. While life-saving, having a treatment that preserves healthy kidney function is the best option.
We encourage you to send your input on the lack of real treatments for PKD and share your experiences to help Congress move the ball forward. All comments must be sent to firstname.lastname@example.org by June 13, 2014. Please cc the PKD Foundation at email@example.com so we can be aware of the input from the PKD community.
Committee Chairman Fred Upton (R-MI) commented, "Ultimately, 21st Century Cures is about patients. Our efforts seek to provide hope to families all across the country. Their invaluable perspective and input in this process is critical and we look forward to partnering together in the months and years ahead as we seek a path to cures."
Learn more about 21st Century Cures here, like the initiative on Facebook, follow along on Twitter and join the conversation using #Path2Cures.
The Department of Defense Peer Reviewed Medical Research Program Awards Three PKD Research Grants
There is more exciting news in the world of polycystic kidney disease (PKD) research, as it was recently announced there will be funding for three PKD research grants totaling $2.2 million. The Department of Defense Peer Reviewed Medical Research Program (PRMRP) recently recommended awarding these three grants.
The Department of Defense, receives yearly public funding from Congress to fund innovative scientific research worldwide. For fiscal year 2013, Congress appropriated $50 million for proposals in 24 topic areas, one of which was PKD.
With stiff competition, this is particularly impressive, and volunteer advocacy efforts made all the difference. Without advocates asking for PKD to be included, the awards below would not have been funded. Read on for more about the awards.
Dr. David Kaplan is the named awardee for a Discovery Award in PKD Research
The goal of the Discovery Award is to support the exploration of an innovative, new concept or method. Studies supported by this award are expected to lay the groundwork for future science.
David Kaplan, Ph.D., of Tufts University, will receive more than $150,000 for his proposal "3D Microperfusion Model of ADPKD." The proposal aims to develop a new system for growing cells with the ADPKD mutation in the laboratory that involves creating a microscale environment that resembles a kidney tubule. A key element of this system is having the proper structural support that allows for cells to form cysts off of a kidney tubule as they would in the body in just 18 months. For more information on Dr. Kaplan, visit his profile.
Dr. Duojia Pan and Dr. Surya Nauli, each named to receive Investigator-Initiated Awards in PKD Research
The goal of the Investigator-initiated Award is to support research that will make an original and important contribution to the field of research or patient care in the topic area of interest.
Duojia Pan, Ph.D., of The Johns Hopkins University, will receive approximately $1 million for his proposed research "Hippo Signaling in Polycystic Kidney Disease." The proposal aims to investigate the functional relationship between PKD1 and the Hippo pathway and also to investigate potential therapeutic targets for PKD. For more information on Dr. Pan, visit his profile.
Surya Nauli, Ph.D., of the University of Toledo, will also receive approximately $1 million for his proposal "The Roles of Primary Cilia in Cardiovascular System." The proposal aims to examine hypertension and aneurysm formation in PKD. Especially within PKD, untreated hypertension can worsen kidney function. Aneurysm rupture in PKD patients is a devastating complication that often could result in stroke and death. For more information on Dr. Nauli, visit his profile.
In addition to advocating for PKD to be included in the PRMRP, several key PKD Foundation volunteers helped make this funding possible. Each year that PKD research funding is available, the PKD Foundation is invited to submit names of individuals to serve as consumer reviewers and participate on the Review Committee. Patients are involved in the review process to help provide a broad understanding of the disease and articulate the impact the funding could have on the patient population. Consumer reviewers representing the PKD Foundation were Bill Brazell, active with the PKD Foundation and former Board of Trustees member, and Klee Kleber, current Board of Trustees member. Dr. Arlene Chapman, a member of the Foundation's Scientific Advisory Committee (SAC) for 10 years and a member of the Board of Trustees, served on a review panel.
Thank you to Dr. Chapman, Bill, Klee and all the volunteer advocates. This brings in approximately $2.2 million for PKD research above the nearly $2.4 million the PKD Foundation is spending on the 15 research grants. Our advocacy efforts are valuable and are making a difference.
For more about the PRMRP, research updates and inspiring consumer stories, visit http://cdmrp.army.mil.
Making a Difference for Patients: The Kidney Community Rallies on May 1, 2014
The PKD Foundation is pleased to give voice to the more than 20 million Americans with kidney disease through its participation in Kidney Community Advocacy Day. Today, the PKD Foundation will join 14 other leading kidney organizations to push for essential change in kidney care. Participants in Kidney Community Advocacy Day will meet with government leaders to discuss two issues that will improve the treatment and quality of life for millions of Americans: increasing federal investment in kidney research, and extending lifetime immunosuppressive drug coverage for kidney transplant recipients.
Funding Innovation, Improving Lives
Research and innovation are critical to advancing new therapies for the more than 20 million Americans with kidney disease. Kidney Community Advocacy Day participants will campaign for an additional $150 million per year for 10 years in additional kidney research funding to spur innovation. This funding is needed to help develop new therapies that enhance patients' lives and ultimately cure this public health burden.
Immunosuppressive Coverage—A Common Sense Choice
Kidney transplant recipients must take immunosuppressive drugs to maintain the health of their transplants. Currently, Medicare only covers the cost of these drugs for 36 months. Those who cannot afford to pay for the immunosuppressive drugs (approximately $4,000 per year) end up back on dialysis (the government spends approximately $90,000 a year for care for patients on dialysis). Extending Medicare coverage for immunosuppressive drugs over a recipient's lifetime is the common sense solution, improving quality of life for people with kidney disease and saving taxpayer dollars.
Leaders participating in Kidney Community Advocacy Day know what's at stake:
- More than 20 million Americans have kidney disease, the 8thleading cause of death in the United States.
- 200,000 of these Americans are children and adolescents, 15,000 of whom are reliant on dialysis or a kidney transplant to remain alive.
- More than 600,000 Americans have kidney failure, known as end-stage renal disease (ESRD).
- More than 185,000 Americans live with a kidney transplant and more than 100,000 more are on the wait list.
- Nearly all patients with kidney failure are Medicare beneficiaries regardless of age, income, or disability.
- ESRD patients account for nearly 7% of Medicare costs but less than 1% of Medicare patients— a total of nearly $35 billion annually.
- Nearly 28% of Medicare expenditures involve patients diagnosed with kidney disease.
Big Win for Transplant Recipients
On March 10, 2014, the Center for Medicare & Medicaid Services (CMS) announced a decision to drop its proposal to remove drugs from protected class status under their Medicare Part D plan. This means transplant recipients will continue to have access to all immunosuppressive drugs under their Medicare Part D plan. This is a definite win for those patients pre- and post-kidney transplant.
On March 4, at the invitation of the National Kidney Foundation, the PKD Foundation joined three other kidney groups (Alport Syndrome, IGA Nephropathy Foundation and AAKP) in Washington D.C. for a day of advocacy to kickoff National Kidney Month. The PKD Foundation was represented by many volunteers from around the country, including Board of Trustee members Klee Kleber and Senator Robert Bennett.
This proposal was one of the topics covered with congressional and senate offices. The advocacy voice was heard!
The advocates also asked for support of increased funding of research into developing treatments and cures for the many causes of kidney disease. Additionally, advocates asked those in the House of Representatives to please consider joining the Congressional Kidney Caucus if they were not already a member and for those in the Senate in helping establish a Senate Kidney Caucus or joining one once it is established.
MODDERN Cures Act Reintroduced
Modernizing Our Drug and Diagnostic Evaluation and Regulatory Network
In September 2013, Rep. Leonard Lance (NJ-7) reintroduced the MODDERN Cures Act in the House of Representatives. One of the PKD Foundation's legislative priorities, this act aims to advance the development of new treatments for chronic and rare diseases, and is critical in the search for treatments for PKD.
Read the bill here.
The bill will:
- Encourage research on treatments, which have been abandoned in the lab, but hold promise for treating diseases like PKD that have unmet medical needs.
- Encourage rapid development of new, safe and effective medicines.
- Provide a pathway to bring promising new compounds to market.
- Remove barriers that limit medical innovation.
- Provide incentives for researchers to develop new diagnostics.
- Ensure timely and appropriate reimbursement for new tests and treatments so that patients have access to the latest medical technology as soon as possible.
- Establish a predictable timeline for the introduction of generic equivalents.
- Advance creative solutions for developing companion diagnostic tests.
- Create a system that rewards efficiency and effectiveness to benefit all people with chronic diseases.
The bill was developed in partnership with the National Health Council (NHC), of which the PKD Foundation is a member. The NHC provides a united voice for people living with chronic diseases and disabilities and their family caregivers.
What you can do:
Contact your local representative and ask them to co-sponsor the MODDERN Cures Act. To see the current list of cosponsors, click here. Visit Advocacy Action Center Tools for templates and other tools to assist you in contacting your representative.