Kidney X A New Model to Spur Innovation

Kidney. X A New Model to Spur Innovation Sandeep Patel, Ph. D Office of the Secretary U. S. Department of Health & Human Services 1

Dialysis Has Changed Little 1962 2018 “If we are going to keep patients alive by artificial means, we then incur the responsibility to see that it is a good life and an enjoyable life. ” – Willem Kolff, 1968, pioneer of hemodialysis and artificial organs #Kidney. X | @Kidney_X | Kidney. X. org

Current State for Kidney Patients Note: 40 M estimate for people living with CKD based on CDC NHANES survey data; USRDS reports an estimated 30 M people living with CKD #Kidney. X | @Kidney_X | Kidney. X. org 3

Cost to System is Unsustainable $34 billion in annual Medicare spending goes to pay for end-stage renal disease (ESRD), automatically covered by Medicare • Roughly equivalent to NIH budget • 7% of Medicare annual expenditure • ~1% of entire Federal budget Source: The United States Renal Data System (USRDS) #Kidney. X | @Kidney_X | Kidney. X. org 4

Recent Investments in Biopharma Silicon Valley Bank, Trends in Healthcare Investments and Exits Mid-Year 2018, Pitchbook #Kidney. X | @Kidney_X | Kidney. X. org

Most Active Device Investors Silicon Valley Bank, Trends in Healthcare Investments and Exits Mid-Year 2018, Pitchbook #Kidney. X | @Kidney_X | Kidney. X. org 6

Boosting Kidney Innovation Financial • Increase end-to-end capital that can take development to patients • Share risk between government, foundations, and industry Perception • Create more attractive incentives for new talent in research, development, and care • Convey a sense of opportunity rather than hopelessness • Expand our notion of what is possible Policy • Create clearer pathways through regulatory and payment processes • Demand innovation #Kidney. X | @Kidney_X | Kidney. X. org 7

Kidney. X Goals (1) Offer funding opportunities through a series of prize competitions each targeted at a set of specific unmet needs (2) Improve coordination across HHS, including NIH, FDA, and CMS, to clarify paths to commercialization and patients (3) De-risk commercialization to unlock investment capital and encourage more shots on goal (4) Create a sense of urgency on behalf of people living with kidney diseases #Kidney. X | @Kidney_X | Kidney. X. org

Principles Patient-Driven: Ensure all product development is patient-driven Urgent: Create a sense of urgency Achievable: Ground in scientifically-driven technology development Catalytic: Reduce regulatory and financial risks to catalyze investment Collaborative: Foster multidisciplinary approaches Additive: Addresses known barriers to innovation Sustainable: Invest in a diverse portfolio to balance risk #Kidney. X | @Kidney_X | Kidney. X. org 9

Kidney. X HHS-ASN Public-Private Partnership HHS Market regulator Research funder Payer ASN Nephrology expertise Broad network $25 million committed #Kidney. X | @Kidney_X | Kidney. X. org 10

First Prize: $2. 6 Million Total #Kidney. X | @Kidney_X | Kidney. X. org 11

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Phase One: Design Solutions • • Seek promising ideas on paper Up to 15 prizes of $75, 000 Submissions closed on February 28, 2019 We received 165 submissions from across the country and world • Prizes awarded April 29, 2019 #Kidney. X | @Kidney_X | Kidney. X. org 13

Phase Two: Develop Solutions • • Seek prototype or component technology Up to 3 prizes of $500, 000 Submissions accepted April 30, 2019 – early 2020 Do not have to participate in Phase One to be eligible for Phase Two #Kidney. X | @Kidney_X | Kidney. X. org 14

Potential Areas of Focus for Kidney. X DIAGNOSTICS point-of-care or at home testing kits, real time kidney monitoring PATIENT-CENTERED TOOLS electronic health record tools designed to identify and track disease, applications to empower patients to manage kidney diseases (e. g. , nutrition apps) MEDICATIONS drugs specifically designed to treat and slow progression of kidney diseases NEXT GEN DIALYSIS wearable or implantable dialyzers, vascular access technologies to improve dialysis patient quality of life; bio-artificial kidneys #Kidney. X | @Kidney_X | Kidney. X. org

HHS Initiative to Transform Kidney Care • Goal 1: Slow Progression of CKD to Kidney Failure • Goal 2: Improve ESRD Treatment • Goal 3: Increase Access to Kidney Transplants #Kidney. X | @Kidney_X | Kidney. X. org

#Kidney. X | @Kidney_X | Kidney. X. org Sandeep. patel@hhs. gov | @innovationwonk 17

For Further Reading 18

Kidney. X Mission (1) Ensure patients have continual access to the most effective medical products (2) Change status quo around dialysis treatment (3) Rethink HHS’ role in catalyzing medical product innovation #Kidney. X | @Kidney_X | Kidney. X. org 19

Kidney. X Logic Model #Kidney. X | @Kidney_X | Kidney. X. org

Few FDA-approved Drugs Drug Year Approved Indication Mircera (methoxy polyethylene glycol-epoetin beta) 2007 Anemia, Erythropoietin stimulating agent, eculizumab 2007 PNH, approval for a. HUS (2011), m. Ab to Complement C 5 belatacept 2011 Kidney Transplantation, CTLA 4 -lg Fusion Protein, pegenesitide 2012 Anemia, Erythropoietin stimulating agent, patiromer 2015 Hyperkalemia etelcalcetide 2017 Secondary Hyperparathyroidism in Dialysis Migalastat 2018 Fabry Disease (based on reduced GL-3 deposits in renal blood vessels, as seen on biopsy) Source = clinicaltrials. gov; Does not include drugs initially approved for a non-CKD indication, e. g. Tolvaptan: 1 st approved in 2009 for hyponatremia and was recently (April 2018) approved for ADPKD Compare to 29 cancer drugs approved 2015 -2017 #Kidney. X | @Kidney_X | Kidney. X. org

Barriers to Innovation #Kidney. X | @Kidney_X | Kidney. X. org

An Entrepreneur’s Journey Seek private/venture funding Idea Justify public health need citing CDC/CMS data Seek NIH research/SBIR funding Seek joint venture partnerships / exits Seek private insurance payment Product Discuss clinical trial requirements with FDA Seek coverage and reimbursement from CMS U. S. Department of Health & Human Services #Kidney. X | @Kidney_X | Kidney. X. org

A (Better) Entrepreneur’s Journey Idea Seek private/venture funding Seek joint venture partnerships / exits Seek private insurance payment Product • Justify public health need citing CDC/CMS data • Seek easier to access non-dilutive capital • Discuss clinical trial requirements with FDA AND CMS together • Utilize collaborative roadmaps U. S. Department of Health & Human Services Additionally, • Encourage multidisciplinary and functional collaborative community • Holistically consider barriers to commercialization and patient access #Kidney. X | @Kidney_X | Kidney. X. org

Dialysis Patient Survey Results* Conditions Would Most Like to See Addressed Fatigue or Low Energy Ability to Travel Washed out/ drained feeling after dialysis Dialysis-free Time Sleeplessness or Insomnia Cramping Ability to Work Decrease in Blood Pressure Anxiety or Stress Depression Mortality Vascular Access-Related Discomfort Lack of Appetite or Taste Itching Impact on Family Body Aches Nausea or Vomiting Blurry Vision 59% 38% 37% 32% 25% 23% 19% 17% 16% 15% 13% 12% 11% *Conducted by Dialysis Patient Citizens; Base: All Respondents 10% (n=979); Question: Which conditions below would you most like 7% to see be addressed through new product development (drugs, 5% devices, biologics, or otherwise)? Select up to 4 responses. #Kidney. X | @Kidney_X | Kidney. X. org 25

Dialysis Patient Survey Results* Products Would Most Like to See Developed Next-generation renal replacement therapy (such as a wearable or artificial implantable kidney) 72% Medications to prevent/slow progression of CKD 44% Improved transplantation immunosuppressive drugs 36% Medications that treat the side effects or symptoms of kidney failure 28% Improved in-center hemodialysis dialyzor technology 28% Improved vascular access 21% Improved home-hemodialysis dialyzer technology 12% Improved peritoneal dialysis dialyzer technology 12% *Conducted by Dialysis Patient Citizens; Base: All Respondents (n=979); Question: Which new products would you most like to see be developed and become available to you or to other kidney patients? Select up to 3 responses. #Kidney. X | @Kidney_X | Kidney. X. org 26

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R&D Activities Kidney Function Activities #Kidney. X | @Kidney_X | Kidney. X. org