D1 Statement of Work.docx

STATEMENT OF WORK (SOW)

GENERAL INFORMATION

Title of Project:

Research Support Services for the Impact of Rare Diseases on Patients and Healthcare Systems (IDeaS) initiative Pilot Program Statement of Need and Purpose:

The National Center for Advancing Translational Sciences (NCATS) Division of Rare Diseases Research Innovation (DRDRI) is seeking support services from EVERSANA to enable the generation of independent analyses, computational tools, and methodologies to facilitate earlier and more accurate identification of rare disease patients. These services will build on previous DRDRI IDeaS efforts done in collaboration with EVERSANA that detailed the prevalence, clinical course, cost, and utilization impact of patients with rare diseases. This next iteration of the IDeaS pilot initiative intends to use the results of the initial pilot study to inform methods for clinical decision support tools to alert clinicians to possible cases of rare diseases. NCATS DRDRI seeks to identify utilization patterns prior to accurate diagnosis and estimate costs of medical care during the diagnostic odyssey, with the larger goal of using this information to inform the development of approaches to diagnose and identify rare disease patients sooner, while improving overall patient care management.

Background Information and Objective:

NCATS’ mission is to improve health through smarter science that results in better treatments faster for all diseases, rare or common. Within that remit, the NCATS DRDRI’s mission is to advance rare disease research to benefit patients. Currently, there are about 7,000-10,000 known rare diseases that affect people in the United States, yet only a few hundred (less than 5%) have a treatment and even fewer a cure. A rare disease is defined in US law (Orphan Drug Act (ODA) of 1983,1 Rare Diseases Act (RDA) of 2002)2 as a disease or condition that affects fewer than 200,000 people in the United States. Most rare diseases affect far fewer patients than this, with most rare diseases affecting only a few hundreds to thousands of patients.

Collectively, however, rare diseases are not rare at all, and in total are estimated to affect as many as 25-30 million patients in the United States, making rare diseases a large public health consideration.

Given the large number of different rare diseases, each of which affects only a small number of patients, assessing the true impact of rare diseases on healthcare systems in the United States is challenging. Many patients with rare diseases have difficulty obtaining a timely and accurate diagnosis (referred to as the diagnostic odyssey) and there is a lack of data regarding the likely and relatively large number of patients, who have not yet received a rare disease diagnosis.

Additionally, International Classification of Disease (ICD) codes largely do not exist for many rare diseases (>50%), leading to myriad downstream effects including difficulty in identifying rare disease patients within healthcare systems databases, imprecise coding of patients under larger “umbrella” (e.g., developmental delay) or “not elsewhere classified (NEC)” terms, poor tracking within databases, and fiscal invisibility given their fragmentation under 1,000s of different terms. This is particularly problematic for (1) characterizing the clinical course of rare disease patients, (2) quantifying (and alleviating) the cost of medical care for people with rare diseases (by disease and in aggregate), (3) and identifying undiagnosed or misdiagnosed rare disease patients.

NCATS DRDRI, in collaboration with EVERSANA, has performed several preliminary feasibility assessments of medical utilization in an initial set of fourteen representative rare diseases and assessed the impacts (utilization and cost) of rare diseases on healthcare systems by leveraging large health databases and analytics support. This next iteration of the IDeaS (Impact of Rare Diseases on Patients and Healthcare Systems) initiative intends to use the results of the initial pilot study to inform methods for clinical decision support tools to alert clinicians to possible cases of rare diseases. DRDRI envisions this will likely involve utilizing EVERSANA’S access to two large U.S. medical claims databases access already obtained at time of contract start date) for the below steps to circumvent the lack of diagnostic codes (ICD-9/10) across most rare diseases:

· Assessment of further case studies – e.g., the creation of specific rare disease profiling (expanded from the initial set of IDeaS 14 diseases) through case definitions

· Generating list of common features among the rare disease case studies/profiling – e.g., timing of visits (number, specialty), age, cost/utilization, basket ICD terminology

· Applying and/or generating computational methodology/algorithm to predict for those features

· Testing generalizability on different population subsets

· Disseminating methodology/models

These steps to gather, quantify, and delineate the data for rare disease patients will not only accumulate much-needed rare disease knowledge and help to identify research gaps but will also advance both common and rare disease research, care, and segmentation associated with precision medicine. NCATS DRDRI seeks to identify utilization patterns prior to accurate diagnosis and estimate costs of medical care during the diagnostic odyssey, with the larger goal of using this information to inform the development of approaches to diagnose and identify rare disease patients sooner, while improving overall patient care management.

Period of Performance:

12-month base period of performance

SCOPE OF WORKGeneral Requirements:

Independently and not as an agent of the Government, the Contractor shall furnish all the necessary services, qualified personnel, material, equipment, and facilities, not otherwise provided by the Government as needed to perform the Statement of Work below at time of contract start date:

Specific Requirements:

The Contractor shall perform the following tasks:

TASK AREA 1: ANALYSES

The below analyses should be completed through use of two large United States healthcare databases, IBM MarketScan Research Databases and the Symphony Health Integrated Dataverse (access already obtained by the contractor at time of contract start date). A large United States healthcare database is defined as a health insurance or hospital system database that includes a large (no less than 100 million people) and diverse population of United States patients inclusive of pediatric and adult populations and inpatient and outpatient data, and includes both commercial and public health insurance data sources.

These healthcare databases are of sufficient size and scope to adequately support detailed analyses on large percentages of the US population, such that rare and very rare disease populations may be identified, and where applicable, may permit subgroup analyses of the population based on demographic, insurance coverage (including, but not limited to patients covered by commercial, Medicaid and other payors) and other factors, as well as analyses of individual patient data for more detailed disease course assessments.

When needed and where applicable, EVERSANA shall obtain Institutional Review Board (IRB) or other oversight to access patient data and conduct these data analyses, such as individual patient assessments, if necessary, consistent with the Privacy Act and any applicable Privacy Act System of Records (SORN) notice.3

Part 1: Analyses to Capture Prevalence, Clinical Course, and Cost/Utilization Impact

DRDRI and EVERSANA have collaborated on methodology to allow for rare disease case definition creation. Part 1 of the contract will build upon this methodology to assess further case studies through independent analyses on prevalence, clinical course, and cost/utilization impact of rare diseases, e.g., the creation of specific rare disease profiling (expanded from the initial set of IDeaS 14 diseases) through case definitions, to generate common features among the rare disease case studies/profiling. The Contractor shall:

(1) Assess further case studies – e.g., the creation of specific rare disease profiling (expanded from the initial set of IDeaS 14 diseases) through case definitions

(2) Generate list of common features among the rare disease case studies/profiling – e.g., timing of visits (number, specialty), age, cost/utilization, basket ICD terminology

Part 2: Generating Proof of Concept Computational Tools and Generalizable Methodologies

Generate proof of concept computational tools and generalizable methodologies for publication that could be tested or used at various healthcare provision sites to improve the diagnostic accuracy and timeliness of diagnosis or rare disease patient identification (e.g., algorithms for identifying undiagnosed or misdiagnosed rare disease patients), and to quantify the impact of rare disease patients on healthcare systems. The Contractor shall develop algorithms and data- search refinements to precisely explore individual rare diseases, disease clusters and rare disease clinical research questions. The Contractor shall:

1) Apply and/or generate computational methodology/algorithm to predict for those features

2) Test generalizability on different population subsets

3) Disseminate methodology/models broadly to the research community

TASK AREA 2: DISSEMINATING RESULTS

The Contractor shall, in collaboration with NCATS staff, develop, document, and aid in the generation of publications that present the analyses on prevalence, clinical course, and cost/utilization impact of rare diseases; as well as describing and disseminating computational tools and generalizable methodologies that have been generated. The Contractor shall generate scientific publication-quality charts, tables and graphs from the data generated by the project team; assist with presentation development for public meetings for the project; and, where applicable, generate draft written summaries of these meetings.

GOVERNMENT RESPONSIBILITIES

The Government shall provide access to relevant NIH/NCATS secure systems, online collaboration, google drive and other platforms as required to perform the work. In addition to the scientific and operational feedback to overcome problems, identify areas of additional analysis, and context of where analyses fit within the overall intent of the project.

DELIVERY OR DELIVERABLES

Contract Kickoff Meeting The Contractor shall schedule and conduct a contract kickoff meeting with NCATS DRDRI staff within one (1) week of the contract award date. A contract kickoff meeting should include a detailed presentation of the contractor’s host data platform, previous experience with RDs, and proposed methodology to complete the below tasks. The Contractor shall develop and present an Analytics Plan for iterative results of the analyses under Task Area 1. The Analytics Plan should include a milestone timeline, detailing the types of analyses being completed by certain dates across the contract year.

Bi-Weekly Meetings Beginning two (2) weeks after the contract kick-off meeting, the Contractor and appropriate NCATS DRDRI staff will meet on a bi-weekly basis, unless otherwise agreed upon, to share and iterate on results of the support activities performed during the previous two weeks. These bi- weekly meetings will alternate as sprint and detailed report meetings.

(1) Sprint Report Meetings: The contractor shall arrange, plan and conduct an hourly meeting on the second week of every month with NCATS IDeaS project staff to report on the work performed in the 2-week period prior to this meeting (i.e., since prior detailed report meeting). The contractor shall follow Agile recommendations for sprints, and the meeting shall include, but is not limited to, additional analyses performed, detailed descriptions of methodologies used and findings from these analyses, problems encountered in the analyses, and discussion with NCATS staff to overcome problems, identify areas of additional analysis, and context of where analyses fit within the overall intent of the project. Meeting materials for sprints are to include an agenda and Powerpoint or other slide representation of the findings and other materials to facilitate the discussion that will be prepared by the contractor and sent to the NCATS IDeaS team at least 1 day prior to the meeting and final version to follow within 2 days after the meeting.

(2) Detailed Report Meetings: This meeting shall be similar, but more detailed than the sprint meeting, and may include citations to any literature or other references and sources used. The contractor shall arrange, plan and conduct a one to two (1-2) hour meeting on the fourth week of every month with NCATS IDeaS project staff to provide a detailed report on the work performed in the 2-week period prior to this meeting (i.e., since prior sprint meeting). The meeting shall include, but is not limited to, a written description of additional analyses performed, detailed descriptions of methodologies used and findings from these analyses, problems encountered in the analyses, and discussion with NCATS staff to overcome problems, identify areas of additional analysis, and context of where analyses fit within the overall intent of the project. A Powerpoint or other slide format may be used to write and describe these results that will be shared with NCATS staff as a draft at least 1-day prior to the scheduled meeting, and final version to follow within 2 days after the meeting.

The Contractor shall present data analysis findings to NCATS DRDRI orally and through written presentations and summaries of the data in charts, tables and figures, where applicable. All iterative results shall be housed within an electronic system, in the Contractor’s environment, accessible to NCATS staff at all times to do further analysis and download (if necessary) to monitor progress.

Method Delivery Schedule

to COR Within 21 days of contract award date

to COR Monthly within 2 days of each Sprint Meeting

to COR Monthly within 2 days of each Detailed Meeting

to COR Annually by the end date for each contract year

to COR Within 30 days after identification of need by NCATS

REPORTING REQUIREMENTS

All reports required herein shall be submitted in electronic format. All electronic reports submitted shall be compliant with Section 508 of the Rehabilitation Act of 1973. Additional information about testing documents for Section 508 compliance, including guidance and specific checklists, by application, can be found at: http://www.hhs.gov/web/508/index.html under "Making Files Accessible."

1. Analytics Plan: Within 21 days after the contract award date, the Contractor shall develop and present an Analytics plan and plan for iterative results of the analyses, to generate: (1) better estimates of rare disease prevalence; (2) understand the clinical course of rare diseases, such as testing, treatments received, and interim diagnoses, among others, to improve understanding of the diagnostic odyssey and healthcare utilization for rare diseases; (3) quantify the cost and impact of rare diseases on the healthcare system and patients; and (4) perform additional analyses (such as subgroup analyses) that may be identified during the course of this work.

2. Monthly Sprint Meeting Report: Within 2 days after each meeting, the contractor shall provide the final version of the agenda and Powerpoint or other slide representation of the meeting’s findings and other materials.

3. Monthly Detailed Meeting Report: Within 2 days after each meeting, the contractor shall provide the final version of the agenda and Powerpoint or other slide representation of the meeting’s findings and other materials. Any relevant materials in suitable formats shall also be included, e.g., graphics/data tables, written descriptions of analyses conducted (e.g., Word), code/algorithms developed, etc.

4. Annual Report: Annual report on work conducted and work products. This detailed written annual report is to include all analyses conducted and findings from the detailed analyses conducted during the prior year, computational methods and algorithms generated, written summaries and publication quality figures generated from the analyses. Where applicable, comparison of the findings to existing medical literature, references and other publication sources relevant to the analyses are to be provided.

5. Medical Publication: In collaboration with NCATS Staff, the contractor shall draft and finalize up to four medical publications-quality documents related to the data analysis findings, inclusive of figures and tables (as needed) for submission to medical journals.

Version 11-30-2016-1 FAR Part 11—Describing Agency Needs