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Introduction

On November 15, 2021, the National Academies of Sciences, Engineering, and Medicine held a virtual workshop titled The State of Science of Hematopoietic Stem Cell Treatment and Disability. The workshop was planned by a planning committee,¹ which was chaired by Sara Rosenbaum, the Harold and Jane Hirsh Professor of Health Law and Policy at George Washington University. The workshop was sponsored by the Social Security Administration (SSA) and planned according to a Statement of Task (Box 1-1). In the opening remarks, Rosenbaum explained that the purpose of the workshop was to facilitate a discussion to examine disability following hematopoietic stem cell transplantation (HSCT), and explore key aspects of posttransplant functioning and recovery. The workshop planning committee invited subject-matter experts to present on important aspects of HSCT recovery and functioning in adults and children.

The workshop was divided into four sessions. In the first session, speakers provided an overview of HSCT in the United States, including current outcomes data, and explained patterns in transplantation survival and recovery. In the second session, speakers delved more deeply into the disabling late effects

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¹ The planning committee’s role was limited to planning the workshop, and the Proceedings of a Workshop was prepared by the workshop rapporteurs as a factual summary of what occurred at the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants and are not necessarily endorsed or verified by the National Academies of Sciences, Engineering, and Medicine, and they should not be construed as reflecting any group consensus.

of HSCT in adults and children. The third session featured patients, experts, and caregivers discussing their perspectives on posttransplantation functioning. Finally, the fourth session looked forward at the future outlook for HSCT and related disability.

This Proceedings of a Workshop describes the presentations from invited subject-matter experts and panel discussions held during the workshop, including responses to questions from the SSA and the general public. The speakers, panelists, and workshop participants presented a broad range of information about HSCT; Box 1-2 provides a brief summary of key points made by individual participants. This proceedings follows the same structure as the workshop. Appendix A contains the reference list, Appendix B contains the workshop agenda, and Appendix C contains short biographical sketches

of the workshop planning committee members and speakers. The speakers’ presentations and the webcast have been archived online.²

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² The workshop video can be viewed here: https://www.nationalacademies.org/event/1115-2021/the-state-of-the-science-of-hematopoietic-stem-cell-treatment-and-disability-aworkshop (accessed March 4, 2022).

BACKGROUND

In the opening session, Steve Rollins, the deputy associate commissioner at the Social Security Administration (SSA) Office of Disability Policy, welcomed participants to the workshop, noting that he and his colleagues at SSA were eager to learn about how they may be able to refine the evaluation of individuals who have undergone or may qualify for stem cell-based treatments. The idea for this workshop, he said, emanated in part from an existing tension around stem cell treatments. Rollins explained that these treatments are often presented in the press as having “revolutionary potential,” but the everyday reality of medical care may be somewhat different. He said that SSA needs a deeper understanding of how the potential of stem cell treatments may translate into medical and functional changes for individuals, and how recipients recover and manage late effects over time. The discussions at this workshop will keep SSA informed and prepared as it evaluates cases involving stem cell treatment, and it will guide SSA efforts in this area in the future, said Rollins.

Vince Nibali, policy analyst at the SSA Office of Disability Policy, gave workshop participants a brief overview of SSA’s disability programs and how decisions about eligibility are made. SSA administers two disability-related programs: Social Security Disability Insurance (SSDI) and Supplemental Security Income (SSI). While these two programs have different nonmedical requirements for eligibility (e.g., work history), they share the same medical criteria and use the same sequential evaluation processes to determine initial eligibility. For adults, said Nibali, disability is defined by statute as the “inability to do any substantial gainful activity by reason of any medically determinable physical or mental impairment which can be expected to result in death or last for a continuous period of not less than 12 months” (C.F.R. § 404.1505).³ For children, a finding of disability is based upon having “marked and severe functional limitations” attributable to physical or mental impairments that are expected to cause death or last for a continuous period of at least 12 months (C.F.R. § 416.906). For both children and adults, the outcome of a disability evaluation depends upon the severity of functional limitations arising from the person’s impairment or combination of impairments, either because those

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³ The definition of disability is described in Section 223(d)(1) of the Social Security Act as an “inability to engage in any substantial gainful activity by reason of any medically determinable physical or mental impairment which can be expected to result in death or which has lasted or can be expected to last for a continuous period of not less than 12 months, or in the case of an individual who has attained the age of 55 and is blind (within the meaning of blindness as defined in section 216(i)(1)), inability by reason of such blindness to engage in substantial gainful activity requiring skills or abilities comparable to those of any gainful activity in which the individual has previously engaged with some regularity and over a substantial period of time” (Social Security Act, 42 U.S.C. § 423(d)).

functional limitations preclude work, or because they are marked and severe. SSA’s sequential evaluation processes use a series of specific questions to determine eligibility; these questions are designed to allow adjudicators to decide the clearest cases quickly.

For example, Nibali said, the processes include a “screen-in” step, which captures the most severe and obvious cases of disability by comparing a claimant’s impairments against the criteria in SSA’s listing of impairments. If a claimant does not meet these criteria, the adjudicator continues with the sequential evaluation process to a more in-depth functional analysis. In addition to evaluating initial eligibility, SSA is also tasked with identifying individuals who are no longer eligible for disability benefits. To capture this group, SSA regularly conducts “continuing disability reviews” (CDRs). The frequency of CDRs is based in part on how likely it is that an individual’s underlying impairments will improve over time. In response to a question from a participant, Nibali noted that SSA has medical consultants to provide advice on CDR decisions, but he does not know how many of these consultants have a background in stem cell treatment. The discussions at this workshop, said Nibali, will assist SSA in improving and refining its evaluation processes, as well as contribute to the agency’s mission to provide the most accurate disability decisions as efficiently as possible.