FDA MOU # 225-19-010
MEMORANDUM OF UNDERSTANDING BETWEEN
OFFICE OF THE CHIEF TECHNOLOGY OFFICER, U.S. DEPARTMENT OF HEALTH & HUMAN SERVICES

AND

CENTERS FOR MEDICARE & MEDICAID SERVICES AND
FOOD AND DRUG ADMINISTRATION AND
NATIONAL INSTITUTES OF HEALTH AND
CENTERS FOR DISEASE CONTROL AND PREVENTION CONCERNING
KIDNEYX: A PUBLIC-PRIVATE PARTNERSHIP TO ACCELERATE INNOVATION IN THE PREVENTION, DIAGNOSIS, AND TREATMENT OF KIDNEY DISEASES

A. Parties

This MEMORANDUM OF UNDERSTANDING (“MOU” or “Agreement”) is entered into by and among the Office of the Chief Technology Officer (Office of the CTO) within the Office of Secretary (OS) of the Department of Health & Human Services (HHS), the Centers for Medicare & Medicaid Services (CMS), the Food and Drug Administration (FDA), the National Institutes of Health (NIH), and the Centers for Disease Control and Prevention (CDC). The Office of the CTO, CMS, FDA, NIH, and CDC are referred to herein individually as a “party” and collectively the “parties.”

B. Introduction

Approximately 40 million adults in the U.S. have chronic kidney diseases (CKD), and approximately 660,000 of them have progressed to end stage renal disease (ESRD), defined as requiring chronic dialysis or receipt of a kidney transplant. CKD and ESRD are debilitating for patients, and drastically impact patient and family quality of life. While kidney transplants provide a better quality of life than treatment with dialysis, they are difficult to obtain due to incompatibility or long waiting lists. Treatment of CKD and ESRD is extremely expensive for the Federal government, with Medicare alone currently spending approximately $34 billion each year on dialysis treatments.

For the last several decades, advances in CKD and ESRD treatment have been modest, with few major innovations reaching patients. Several market, scientific, and government barriers may be limiting kidney health innovation: 1) private investors consider funding early-stage research and development risky due to a lack of clear commercialization pathways and past failures; 2) government research funding is inadequate, and few well-capitalized foundations specifically focus on kidney disease and; 3) the current ecosystem and payment environment favors “status quo” approaches.

C. Vision

In view of the historic barriers to kidney health innovation, a public-private partnership is envisioned to further spur disruptive innovation, resulting in better treatments to patients with CKD and ESRD. This effort, called the Kidney Innovation Accelerator (KidneyX), will hold a series of prize competitions targeting unmet needs of patients by supporting innovators with promising solutions and technologies. In accordance with the COMPETES Act, 15 U.S.C. § 3719, KidneyX aims to drive additional private dollars and populate the early stage pipeline of new products. In addition to the cash prizes and because new innovators often lack commercialization expertise, KidneyX intends to provide participants direct insights from investors, business and manufacturing experts, scientists, engineers, patients, regulators, payers, and others as needed and as allowable under applicable federal law.

HHS has established a public-private partnership with the American Society of Nephrology (ASN) as described in a MOU, laying out a framework to co-sponsor prize competitions within KidneyX. ASN and HHS have a shared vision to accelerate the pace of innovation in kidney care and the expertise of ASN and its communities in kidney care.

D. Authority

The Office of the CTO has the authority to enter into this agreement pursuant to Section 301 of the Public Health Service Act.¹ FDA has authority to enter into this agreement pursuant to Section 301 of the Public Health Service Act.² NIH has authority to enter into this agreement pursuant to Sections 301 and 402 of the Public Health Service Act.³ CMS has authority to provide benefits to ESRD patients pursuant to Section 1881 of the Social Security Act.⁴ CDC has authority to enter into this agreement pursuant to Section 301 of the Public Health Service Act.⁵

E. KidneyX Structure and Operational Plan

Through KidneyX, private entities will collaborate with the Office of the CTO and other parties to the agreement to coordinate the design and administration of a series of public-private prize competitions, using the COMPETES Act prize authority (15 U.S.C. § 3719), which authorizes Federal Agencies to carry out a program to competitively award prizes to stimulate innovation that has the potential to advance the mission of the respective agency. The COMPETES Act enables HHS to partner with private entities in the sponsorship and administration of prize competitions. Other types of funding opportunities or activities may be explored in the future depending on appropriateness, authorities, and funding availability.

ASN will act as co-sponsors of individual prize competitions, work with other private partners as needed, and manage the KidneyX website (www.kidneyx.org). Individual prize competitions will be jointly administered by HHS and ASN, and will adhere to all applicable legal requirements and policies. The decision on use of Federal funds, pursuant to the COMPETES Act, will be informed by the judging process described in this agreement and be made by the CTO or another HHS official designated as the award approving official. Final decisions on private funds will rest with respective private organizations.

The topics for each prize will vary, focusing on product/solution development and patient access to innovation, covering specific unmet needs across prevention, diagnosis, and treatment of kidney diseases. Examples of topics for prizes include, but are not limited to the following:

• Renal replacement technologies: implantable and wearable renal replacement technologies; bio-artificial kidneys; and vascular access technologies.
• Patient-centered tools: electronic health record tools designed to identify, track, and manage disease; applications to empower patients to manage kidney disease and its challenging transitions (e.g., individual care plans).
• Diagnostics: point-of-care or at-home testing kits; better tools to measure kidney function and identify early kidney injury or disease.
• Therapies for the prevention and treatment of CKD and ESRD: including drugs, devices, and biologics.

F. HHS Roles and Responsibilities

The HHS Office of the CTO will be responsible for coordinating all activities related to KidneyX with other parties and with the external partner ASN. The Office of the CTO will also be responsible for approving the scope and structure of individual prize competitions for which HHS will be a sponsor in consultation with the other parties. In accordance with the COMPETES Act, all prize competitions offering $1 million or more in prizes must also be approved by the HHS Secretary.

The role of HHS is substantial and collaborative. HHS envisions not only individual agencies to play a role, but also for agencies to work together towards the common goal of accelerating innovation in the development of new medical products and solutions for people living with kidney diseases.

The following table summarizes the roles of the HHS agencies in KidneyX, with more detailed descriptions further down. For any details not included or for any disputes that arise between parties, parties agree to discuss and resolve those issues in good faith when they arise.

 

Office of the CTO:

• Lead KidneyX internal working group meetings.
• Identify lead points of contact to represent appropriate HHS Staff Divisions in KidneyX working group meetings.
• Lead development and implementation of the KidneyX model; coordinate entities and external organizational or individual partners.
• Help identify existing federal resources that could supplement the assistance given to participants in KidneyX.
• Help identify federal funds to be used, in combination with private funds, for the administration of KidneyX and funding of participants.
• Manage contracts, including current contract with Research Triangle Institute, to perform ongoing performance evaluation of KidneyX.
• Lead and coordinate all aspects of communication and marketing of KidneyX.
• Represent HHS in conferences and other public meetings, as appropriate and with approval of managing supervisors and appropriate ethics officials.
• Ensure all prize judges complete financial disclosure and conflict of interest forms.

CMS:

• Identify lead points of contact to represent the Coverage and Analysis Group (CAG), the Center for Medicare (CM), and the Center for Medicare and Medicaid Innovation (CMMI) in KidneyX working group meetings.
• Advise participants in the design of clinical trials that would reduce the likelihood of delay in coverage determination(s).
• Consider case-by-case examples for use of Coverage with Evidence Development program within CAG.
• Work directly with Office of the CTO and KidneyX participants to explore testing the use of new technologies through new or existing payment and service delivery models through CMMI’s authority under section 1115A of the Act, or other demonstration authorities such as Section 402 of the Social Security Amendments of 1967.
• In collaboration with FDA, provide participants information on how to be considered for any applicable parallel review and other pre-submission programs to expedite regulatory and coverage decisions.
• Educate participants about currently existing programs with FDA Investigational Device Exemption categories.
• Educate participants through public documents (e.g. the CMS Innovator’s Guide) or individual conversations about available payment pathways appropriate for new kidney disease technologies.
• Represent CMS in conferences and other public meetings, as appropriate and with approval of managing supervisors and appropriate ethics officials.
• Serve as or recommend expert technical reviewers¹¹ of prize submissions and participate in judging to identify winning submission, consistent with CMS and HHS guidelines.

FDA:

• Identify lead points of contact to represent CDRH, CBER, and CDER in KidneyX working group meetings.
• Advise participants on applicable data requirements to support marketing authorization and, as appropriate, post-market regulatory decision making through the relevant and appropriate FDA processes.¹²
• Advise participants on the design of clinical trials that would support marketing authorization through the applicable FDA processes on the same terms as those available to other applicants and members of the public.
• In collaboration with CMS, provide participants information on how to be considered for any applicable parallel review and other pre-submission programs that expedite regulatory and coverage decisions on the same terms as those available to other applicants and members of the public.
• Educate participants about applicable FDA programs that can be used to expedite development and review of devices, drugs and biologics for serious conditions.
• Provide clarification on regulations, guidance, and policies applicable to participants, including information regarding appropriate points of contacts for regulatory submissions.
• Work to design a regulatory road map for innovative renal replacement technologies, including existing efforts such as the Kidney Health Initiative.
• Represent FDA in conferences and other public meetings, as appropriate and with approval of managing supervisors and appropriate ethics officials.
• Advise the KidneyX judging panels and reviewers on relevant regulatory policies and guidelines that will aid in the review of submissions consistent with FDA and HHS policies.

NIH:

• Identify lead points of contact to represent NIDDK, NCATS, and NIBIB in KidneyX working group meetings.
• Consider publishing a Notice in the NIH Guide encouraging Small Business Innovation Research (SBIR) or Small Business Technology Transfer (STTR) applications aligned with the goals of KidneyX.
• Provide information on the current state of biomedical science and engineering to CMS and FDA to aid them in the development of regulatory, coverage, and payment guidance for novel renal replacement technologies and potentially on future prize topics as appropriate.
• Provide technical assistance to Office of the CTO on the current state of science and engineering for renal replacement technologies and related topics during the planning and development phase of KidneyX prize competitions and potentially on future prize topics as appropriate.
• Provide technical assistance on the scientific merits of proposed solutions to HHS KidneyX prize finalists on an equal basis during the solution development phases, in a manner consistent with HHS and NIH policies.
• Represent NIH in conferences and other public meetings, as appropriate and with approval of managing supervisors and appropriate ethics officials.
• Consider serving as or recommend expert technical reviewers of prize submissions and participate in judging to identify winning submission, consistent with NIH and HHS guidelines.
• NIH will participate in the KidneyX Redesign Dialysis prize competition, launched in Oct 2018, and will decide on participating in other KidneyX projects after the Redesign Dialysis project has finished and been evaluated.

CDC:

• Identify lead point of contact in KidneyX working group meetings.
• Advise Office of the CTO and others in the KidneyX team on the development of economic models to assess the health cost and burden impacts of current and future technologies within the scope of KidneyX prize competitions. Scope and level of effort will be discussed at a later time in good faith.
• Provide technical assistance to Office of the CTO and other HHS collaborators on the current state of public health for kidney diseases and in the planning and development phase of KidneyX prize competitions, including any related to nutrition and lifestyle factors and interventions.
• Represent CDC in conferences and other public meetings, as appropriate and with approval of managing supervisors and appropriate ethics officials.
• Serve as or recommend expert technical reviewers¹¹ of prize submissions and participate in judging to identify winning submission, consistent with CDC and HHS guidelines.
• Support dissemination of KidneyX activities through several communication channels.

G. Timeline

This MOU becomes effective for each party on the date of their signature and shall remain in full force and effect for five years. This agreement may be modified or terminated by mutual written consent by all of the parties or any party may terminate its participation upon a 60-day advance written notice to the other parties. The end date for prize competitions issued under KidneyX may extend beyond the term of this agreement.

KidneyX was announced beginning in April 2018 and launched the first prize competition, called Redesign Dialysis, in October 2018. The basic timeline of forthcoming activities is provided below, all of which are subject to change.

• April 2019 – First KidneyX Summit planned to be held in Washington, D.C.
• January/February 2020 – Conclusion of Phase II of Redesign Dialysis prize.
• Additional prizes addressing other kidney care topics and future prizes focused on next- generation renal replacement therapies are scheduled to be launched in parallel as funding and resources permit.

H. Resource Responsibilities

This MOU represents the broad outline of the parties’ intent to run prize competitions in the future for collaborative efforts in areas of mutual interest to each party. Each party shall bear its own costs and expenses in fulfilling its responsibilities under this MOU, including, without limitation, its own expenses for travel and accommodation. This MOU does not commit a party to any obligation or expenditure of Federal funds. Expenditures by each party will be subject to its budgetary processes and to the availability of funds and resources pursuant to applicable laws, regulations, and policies. Each party agrees that any supplies or services it provides pursuant to this MOU are provided without expectation of payment from any other party, and each party agrees to waive any and all claims for such payment. Furthermore, while the parties intend to cooperate with respect to the activities outlined herein, neither party will be required to commit funds, personnel or resources under this MOU.

I. Meetings and Administration

KidneyX HHS parties will meet on a regular and as needed basis. Meeting support including agendas will be provided by Office of the CTO. Meeting participants may invite agency technical experts and others to meetings as needed to answer questions or provide information related to particular agenda topics. All meeting attendees must maintain confidentiality in accordance with existing laws and regulations concerning discussions of a sensitive nature, including procurement-related matters.

For additional information on the Kidney Innovation Accelerator, please reach out to Sandeep Patel at
(202) 205-0355 or [email protected].

J. Approvals

Edwin Simcox, Jr., J.D.
Chief Technology Officer
U.S. Department of Health and Human Services
Date: 6/12/19

Francis S. Collins, M.D., Ph.D.
Director
National Institutes of Health
Date: 6/12/19

Norman E. Sharpless, M.D.
Acting Commissioner
Food and Drug Administration
Date: 6/12/2019

Seema Verma, M.P.H.
Administrator
Centers for Medicare & Medicaid Services
Date: 6/11/2019

Robert R. Redfield, M.D.
Director
Centers for Disease Control and Prevention
Date: 6/10/19

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1 42 U.S.C. § 241
2 Id.
3 42 U.S.C. § 241, 282
4 42 U.S.C. § 1395rr.
5 42 U.S.C. § 241
6 Includes the Office of Business Management and Transformation (OBMT) under the Assistant Secretary for Administration (ASA), as well as the Assistant Secretary for Planning and Evaluation (ASPE).
7 Includes the Centers for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research (CBER), and the Center for Devices and Radiological Health (CDRH).
8 Includes the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the National Institute of Biomedical Imaging and Bioengineering (NIBIB), and the National Center for Advancing Translational Sciences (NCATS).
9 Regulatory guidance and advice (feedback on marketing pathway, planned testing, clinical trial design, etc.) related to a specific product would be provided through appropriate FDA Centers (CDER, CBER, CDRH). Of note, the type and extent of regulatory guidance and advice will depend on the FDA Center and the product type. Any such guidance and advice will be provided through existing FDA processes on the same terms available to other applicants and members of the public.
10 FDA CDER employees’ participation in the Kidney Accelerator would be consistent with the roles and responsibilities for CDER PPP Liaisons as outlined in MAPP 4100.2. https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ManualofPoliciesProce dures/UCM532571.pdf. Any input on clinical trial design or advice will be provided through existing FDA processes on the same terms as those available to other applicants and members of the public.
11 Each reviewer is envisioned to provide initial scores for a subset of submissions based on their experience and expertise. Aggregated scores from multiple reviewers will then inform the recommendations that the judging panel will ultimately create for the HHS CTO to decide winners. Reviewers will be a combination of Federal and non-Federal individuals and judges will be Federal employees or Special Governmental Employees (SGEs). All reviewers and judges will be asked to sign conflict of interest and non-disclosure agreements.
12 The type and extent of regulatory guidance and advice will depend on the FDA Center and product type. FDA Centers may also have additional programs available to support innovation. For example, CDRH will foster interaction between KidneyX participants and the National Evaluation System for Health Technology and offer lab space as well as other scientific collaborations as appropriate. Any regulatory guidance and advice will be provided through existing FDA processes on the same terms as those available to other applicants and members of the public.