Research

JAMA Pediatrics | Original Investigation

A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease

The Medicaid Perspective
Patrick DeMartino, MD; Meredith B. Haag, MD, MPH; Alyssa R. Hersh, MD, MPH;
Aaron B. Caughey, MD, MPH, PhD; Joshua A. Roth, PhD, MHA

Editorial page 565

IMPORTANCE Hundreds of gene therapies are undergoing clinical testing and are likely to be Supplemental content
priced more than $1 million per course of treatment. The association that high prices will have
with insurance coverage of gene therapy remains unclear. Gene therapy for sickle cell disease
has shown early success and would be one of the first gene therapies available for a relatively
large population.

OBJECTIVES To estimate the budget impact and affordability of a gene therapy for severe
sickle cell disease from the perspective of US Medicaid programs with the highest prevalence
of sickle cell disease while exploring the impact of an annuity payment model.

DESIGN, SETTING, AND PARTICIPANTS A budget impact analysis was performed from January 1
to May 31, 2020, for a sickle cell disease gene therapy from the perspective of 10 state
Medicaid plans with a 5-year time horizon, using state-level disease prevalence data from
2012. Disease prevalence, Medicaid enrollment, and expenditures were derived from the
available literature. The eligible population was based on modified clinical trial inclusion
criteria including individuals aged 13 to 45 years with severe disease.

EXPOSURES The gene therapy was assumed to be administered to 7% of the eligible

population annually and was curative (no subsequent disease-related expenditures). The
gene therapy price was $1.85 million in the base case, and baseline disease-related
expenditures were $42 200 per year.

MAIN OUTCOMES AND MEASURES The main outcomes were total budget impact and budget
impact per member per month in years 1 through 5. One-way sensitivity analysis was used to
evaluate uncertainty of market diffusion, size of eligible population, price of therapy, and cost
of routine care.

RESULTS An estimated 5464 Medicaid enrollees would be eligible for the gene therapy
nationally, with 2315 individuals in the 10 Medicaid programs of interest (16 per 100 000
enrollees). The model projected a mean 1-year budget impact of $29.96 million per state
Medicaid program in the sample ($1.91 per member per month). A 5-year annuity payment
reduced the short-term budget impact.

CONCLUSIONS AND RELEVANCE This study suggests that a gene therapy for severe sickle cell
disease is likely to produce a considerable budget impact for many Medicaid plans while
potentially offering substantial benefit to patients. Payers may need to take steps to ensure
affordability and access. Gene therapy for sickle cell disease is likely to provide an early Author Affiliations: Division of
demonstration of the unique financial challenges associated with this emerging drug class. Pediatric Hematology and Oncology,
Department of Pediatrics, Oregon
Health & Science University,
Doernbecher Children’s Hospital,
Portland (DeMartino, Haag); Oregon
Health & Science University,
Department of Obstetrics and
Gynecology, Portland (Hersh,
Caughey); University of Washington
School of Pharmacy, Fred Hutchinson
Institute for Cancer Outcomes
Research, Seattle (Roth).
Corresponding Author: Patrick
DeMartino, MD, Division of Pediatric
Hematology and Oncology,
Department of Pediatrics, Oregon
Health & Science University, 707 SW
JAMA Pediatr. 2021;175(6):617-623. doi:10.1001/jamapediatrics.2020.7140 Gaines St, Portland, OR 97239
Published online March 22, 2021. Corrected on June 7, 2021. (demartip@ohsu.edu).

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 Research Original Investigation
M
any gene therapies under development may soon
provide novel curative options for a host of malig-
nant and nonmalignant conditions. Hundreds of such
therapies are undergoing clinical testing and an estimated 40
to 50 will enter the market by 2030.1 Currently, the commer-
cially available therapies are indicated for a small number of
patients while having exceptionally high prices—exceeding $1
million per treatment. The unparalleled price and potentially
curative single administration presents various challenges to
the existing reimbursement system in the US.2 As demon-
strated by direct-acting antivirals (DAAs) for hepatitis C, a cost-
effective therapy may still create affordability challenges for
payers balancing short-term budgets.3 The assessment of value
(cost effectiveness) and affordability (budget impact) do not
need to occur in tandem and, in some circumstances, early con-
sideration of affordability may be essential.
Although clinical benefit and accrued savings may make
a given therapy financially tenable in the long run, what if a
patient changes health plans after a chronic disease is cured
with a high-cost durable therapy? Is the original insurer en-
titled to the long-term accrued savings? Although several al-
ternative payment models have been proposed, much uncer-
tainty remains and there is unlikely to be a one-size-fits-all
approach.4,5
Sickle cell disease (SCD) affects approximately 100 000 in-
dividuals in the US.6 Despite improvements with comprehen-
sive care and hydroxyurea treatment, quality of life remains
inferior when compared with many other chronic diseases,7,8
and median survival is less than 50 years for individuals with
hemoglobin SS or hemoglobin Sß0 genotypes.9 Only alloge-
neic hematopoietic stem cell transplantation (HSCT) offers a
cure, although its use is limited owing to toxic effects and dif-
ficulty finding well-matched donors. The recent approvals of
L-glutamine, voxelotor, and crizanlizumab for SCD also de-
serve acknowledgment, although their clinical utility ap-
pears modest.10-12 The profound morbidity from SCD is asso-
ciated with significant spending, with lifetime SCD-related
health care expenditures exceeding $550 000 per person in
2020 US dollars.13
Gene therapy for severe SCD appears promising, with one
lentiviral-based therapy demonstrating early efficacy in the on-
going phase 1/2 trial HGB-206.14,15 The early data suggest that
this therapy may markedly improve the lives of individuals with
SCD. A gene therapy using the same lentiviral vector received
conditional approval from the European Medicines Agency in
2019 for the treatment of transfusion-dependent β-thalas-
semia. It appears that a gene therapy for SCD may soon enter
the US market, presenting an option for a relatively large popu-
lation in comparison with existing gene therapies for other dis-
eases. However, this therapy is likely to cost more than $1 mil-
lion, presenting a variety of fiscal challenges to US health care
payers covering large numbers of patients with SCD. At least
55 000 patients with SCD are enrolled in Medicaid nationally.16
We sought to perform a budget impact analysis to esti-
mate the short-term affordability of a gene therapy for severe
SCD from the perspective of US Medicaid programs—a payer
group likely to experience the greatest budget impact. Our ob-
jectives were to (1) estimate the potential short-term budget
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A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease
Key Points
Question What will be the short-term budget impact of a gene
therapy for sickle cell disease among Medicaid programs with the
highest prevalence of the disease?
Findings This budget impact analysis estimated a mean 1-year
budget impact of $29.96 million per state Medicaid program, or
$1.91 per member per month increase in spending, in the 10 states
of interest.
Meaning This study suggests that gene therapy for sickle cell
disease will likely present affordability challenges to several
Medicaid plans.
impact of a gene therapy for severe SCD from the perspective
of the 10 state Medicaid plans with the highest prevalence of
SCD; (2) describe how uncertainty in the size of the eligible
population, market diffusion, and pricing are associated with
budget impact; and (3) examine how an alternative payment
model is associated with short-term cost.
Methods
Overview
A budget impact analysis was performed from January 1 to May
31, 2020, for an SCD gene therapy from the perspective of 10
state Medicaid plans with a 5-year time horizon. The annual
budget impact was calculated as the estimated one-time cost
of the gene therapy less the annual savings from patients hav-
ing previously received the intervention. Key model assump-
tions included the percentage of patients with SCD with a se-
vere phenotype, cost of the gene therapy, and annual market
diffusion rate for the therapy (Table 1).13,17,18 State-level dis-
ease burden based on published Centers for Medicare & Med-
ic aid Services data are detailed in the eTable in the
Supplement.16 The state-level SCD prevalence data are from
2012 (most recent available). The total budget for each Med-
ic aid program was available v ia The Kaiser Family
Foundation.19 No institutional review board approval was re-
quired because the analysis used publicly available data with
no protected health information.
Perspective and Population
The 10 state Medicaid programs with the highest prevalence
of SCD included in the study were (descending order of preva-
lence): Mississippi; Alabama; South Carolina; Georgia; Wash-
ington, DC; Louisiana; Virginia; North Carolina; Florida; and
Maryland. Six of the 10 states have not adopted Medicaid ex-
pansion under the Patient Protection and Affordable Care Act.
An analysis was also performed for all US Medicaid programs
in aggregate. International Society for Pharmacoeconomics and
Outcomes Research (ISPOR) practice guidelines informed our
design.20
The population estimated to be eligible for the gene therapy
was based on criteria for HGB-206 trial eligibility.21 This esti-
mation was intended to approximate potential product label-
ing and criteria for coverage. Adapted eligibility criteria were
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 A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease Original Investigation Research

Table 1. Key Model Inputs

Range considered
Parameter Value in sensitivity analysis Source
Patients with SCD with severe 25 10-40 Assumed
phenotype, %
a
Baseline annual expenditure 42 200 17 100-67 250 Kauf et al,13 Arnold et al17
for patient with SCD, $
Cost of single gene therapy 1 850 000 1 440 000-2 170 000 Beasley and Mathias18
treatment per person, $ Abbreviation: SCD, sickle cell disease.
Annual market diffusion rate 7 2-15 Assumed a
Mean of estimates in associated
for gene therapy, %
reference.

Table 2. Budget Impact Analysis, Medicaid Perspective for US and Selected State Programs
Washington, North South
Estimates US Alabama DC Florida Georgia Louisiana Maryland Mississippi Carolina Carolina Virginia
Total No. eligible for 5464 182 44 521 353 245 170 184 260 203 153
gene therapy
No. to receive therapy, 383 13 3 36 25 17 12 13 18 14 11
year 1
No. treated 0.52 1.41 1.18 0.96 1.36 1.11 0.90 1.99 1.02 1.37 1.04
per 100 000 members,
year 1
Budget impact, year 1 707.63 23.58 5.71 67.43 45.67 31.66 22.02 23.77 33.65 26.32 19.76
(million USD), $
Budget impact, year 1 0.12 0.42 0.20 0.29 0.41 0.29 0.19 0.45 0.25 0.42 0.21
(% total Medicaid
spending)
Cost per member per 0.80 2.18 1.81 1.48 2.09 1.81 1.38 3.07 1.57 2.11 1.60
month, year 1, $
Cost per member per 0.53 1.45 1.21 0.98 1.39 1.20 0.92 2.04 1.05 1.41 1.06
month, year 5, $
Budget impact, year 5 471.25 15.70 3.80 44.91 30.42 21.09 14.67 15.83 22.41 17.53 13.16
(million USD), $

Abbreviation: USD, US dollars.

individuals with SCD aged 13 to 45 years with a phenotype of
severe SCD (at least 4 severe pain episodes in 24 months). Our
age range for eligibility differs from the clinical trial (12-50
years), but provides a more accurate and conservative esti-
mate of the population size by conforming to Centers for Medi-
care & Medicaid Services age strata. As per the HGB-206 trial,
patients with hemoglobin SCD were excluded using the as-
sumption that 25% of individuals with SCD have the SC
genotype.
therapy and incurred no SCD-related costs in subsequent years
(therapy is fully curative and durable). All individuals remain-
ing in the standard of care state incurred SCD-related expen-
ditures. The model considers only direct medical expendi-
tures associated with SCD and the cost of gene therapy.
The model provided the annual net budget impact and per-
member per-month (PMPM) cost. The PMPM cost was calcu-
lated as the annual budget impact divided by the total num-
ber of plan enrollees, divided by 12 months.

Intervention Mix and Time Horizon Input Data

The intervention was a theoretical, one-time gene therapy
based on betibeglogene autotemcel (HGB-206 trial). All indi-
viduals entered the model in the standard of care state—
representing routine preventive and acute SCD care, in aggre-
gate. Allogenic HSCT was not considered a competing therapy
under the assumption that an efficacious autologous gene
therapy would be preferred (and the 7% diffusion rate leaves
adequate market share for transplantation among those with
severe disease). Other noncurative therapies were not in-
cluded as competing treatments. A 5-year time horizon was
used to allow for modeling of a 5-year annuity payment.
Analytic Framework Description
A static cohort entered the model receiving standard of care
(eFigure 1 in the Supplement). Each year, a subset of this co-
hort (defined via the market diffusion rate) received the gene
The proportion of patients with SCD and a severe phenotype
was 25% in the base case and varied from 10% to 40% in the
sensitivity analysis. These parameters were determined via
opinion as no data were available to inform the estimate.
The annual market diffusion rate of the gene therapy was
7% in the base case across all 5 years. There were no data to
inform this estimate given the novelty of this drug class; there-
fore, we aimed for a conservative estimate. A range of the an-
nual market diffusion rate from 2% to 15% was considered in
the sensitivity analysis.
The baseline SCD-related health expenditure was $42 200
per year in the base case. There is significant variation among
published data for annual SCD-related expenditures. The base
case was calculated as the mean of 2 prior studies represent-
ing low and high ends of the expenditure range: the low esti-
mate ($17 100) came from a study of Florida Medicaid pa-

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 Research Original Investigation
Figure 1. Standard vs Annuity Payment Model: Mean Per-Member
Per-Month Budget Impact of Gene Therapy for 10-State Sample
2.50
Standard repayment Annuity payment
Per-member per-month cost, $
2.00
1.50
1.00
0.50
0 PMPM in year 1.
1 2 3 4
Year
tients with SCD13 and the high estimate ($67 250) came from
a study of costs in the year preceding HSCT.17 These esti-
mates provided the upper and lower bounds for the sensitiv-
ity analysis. All costs are updated to 2019 US dollars using the
Consumer Price Index.
The cost of the gene therapy was $1.85 million in 2019
US dollars for the single administration in the base case—
derived from initial pricing ($1.78 million) of betibeglogene
autotemcel in the European Union for β-thalassemia plus
$70 000 for 1 admission for myeloablative conditioning fol-
lowed by gene therapy infusion.18,22 The cost of incident
admission for autologous stem cell transplantation
($120 000) was derived from commercial payer data and
adjusted to $70 000 in the assumption that Medicaid pays
57% of the commercial rate for inpatient care.23,24 The range
considered for sensitivity analysis ($1.44 million-$2.17 mil-
lion) represents a 23% discount from the base c ase
(Medicaid best-price guarantee for drug) with the upper
bound as the “intrinsic value” of the treatment as proposed
by the manufacturer.18 The list price often overestimates
true cost but is widely used for similar purposes. No costs
associated with prehospitalization or posthospitalization
care or adverse effects were included. The model was
intended to provide a conservative estimate of the cost of
the therapy.
In addition to full upfront payment, a 5-year 20% annuity
payment was evaluated (20% of price paid annually). The
manufacturer of betibeglogene autotemcel established an out-
come-based annuity in Germany and suggests this as an op-
tion in other markets.25 Our model used a 5-year annuity with
no outcome-based component given assumed 100% effective-
ness and durability.
Sensitivity Analyses and Uncertainty
The uncertainty of model inputs was evaluated using 1-way
sensitivity analysis for the following parameters: proportion
with a severe phenotype, market diffusion rate, price of the
gene therapy, and annual costs of standard of care. We report
mean PMPM cost as the outcome of interest across the 10-
state sample.
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A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease
Results
An estimated 5464 Medicaid enrollees with SCD would be eli-
gible for the therapy nationally, with 2315 individuals in the
10 state Medicaid programs of interest (16 per 100 000 enroll-
ees). The model projected a mean 1-year budget impact of
$29.96 million per state Medicaid program (Table 2) with a
mean of $1.91 PMPM (0.31% of program budget). Florida dem-
onstrated the highest absolute 1-year cost among the states
evaluated, at $67.43 million ($1.48 PMPM), whereas Washing-
ton, DC, had the lowest, at $5.71 million ($1.81 PMPM). Rela-
tive to each program’s total budget, Mississippi’s spending was
the highest and represented 0.45% of the budget with $3.07
5
Over time, savings accrued and fewer individuals re-
mained eligible for the therapy. An annual decrease in budget
impact occurred across the 5-year time horizon. For Missis-
sippi, the annual cost for years 1 through 5 was $23.8, $21.6,
$19.5, $17.6, and $15.8 million (PMPM decreasing from $3.07
in year 1 to $2.04 in year 5). The mean budget impact across
the 10 state perspectives decreased from $1.91 PMPM in year 1
to $1.27 in year 5.
Annuity Payment Model
Compared with the standard payment scenario, the 5-year an-
nuity payment decreased the short-term budget impact dur-
ing the first 4 years by deferring some spending beyond the
5-year horizon (Figure 1). By year 5, the annual budget impact
in the annuity model exceeded that of the standard repay-
ment. The same number of patients received the therapy in
both scenarios. eFigures 2 to 12 in the Supplement demon-
strate the PMPM cost for the standard vs annuity payment for
each of the 10 states and for all US Medicaid programs in jag-
gregate.
Sensitivity Analysis
Figure 2 demonstrates the result of the sensitivity analysis at
years 1 and 5 for the mean PMPM cost across the sample. Base-
line SCD-related expenditures had no association with year 1
PMPM spending (no savings from averted expenditures in the
first year), so this parameter is only in Figure 2B. The budget
impact was most sensitive to changes in the rate of market dif-
fusion. Conversely, list price of the therapy and baseline SCD-
related expenditures had a more modest impact. For the gene
therapy price range considered ($1.44 million to $2.17 mil-
lion), the year 1 mean PMPM cost was $1.49 to $2.24.
Discussion
Our results indicate that a commercially available gene therapy
for severe SCD will likely generate a considerable short-term
budget impact and potentially present affordability chal-
lenges for many Medicaid programs. For the 10 states with the
highest prevalence of SCD, our results suggest a 1-year bud-
get impact of nearly $2 PMPM. These findings demonstrate
how, even under ideal circumstances (fully curative therapy
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 A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease
Figure 2. Univariate Sensitivity Analysis: Mean Per-Member Per-Month Budget Impact for 10-State Sample
A Year 1
Rate market diffusion (2%-15%)
Percentage with severe phenotype (10%-40%)
Price of gene therapy ($1.44 million to $2.17 million)
0 1.00
Per-member per-month cost, $
B Year 5
Rate market diffusion (2%-15%)
Percentage with severe phenotype (10%-40%)
Price of gene therapy ($1.44 million to $2.17 million)
Baseline annual SCD expenditures ($17 100-$67 250)
0 0.50
Per-member per-month cost, $
with minimal associated costs of administration), this emerg-
ing class of durable one-time therapies may create unique fi-
nancial challenges. We sought to focus on states with the high-
est prevalence of SCD, but from a national perspective, the
budget impact is not trivial, at $0.80 PMPM for all US Medic-
aid programs in aggregate.
Although there is no threshold for budgetary signifi-
cance for Medicaid plans, any new technology adding $1 to
$3 PMPM is noteworthy, especially when applyed to few
enrollees. No existing therapies are suitable for direct com-
parison, although gene therapy for spinal muscular atrophy
provides context. One recent budget impact analysis esti-
mated a PMPM cost of less than $0.10 for a US payer includ-
ing the gene therapy onasemnogene abeparvovec-xioi on
the formulary. 26 Alternatively, we considered Medicaid
spending on the DAA sofosbuvir in 2014. We estimated an
average PMPM cost of $1.54 in the 10 Medicaid programs
included in our analysis (in 2019 dollars).27 Our results indi-
cate that gene therapy for SCD could present an affordability
challenge comparable with that presented by the DAAs for
some Medicaid plans.
It is unclear if this sizable budget impact would force Med-
icaid plans to implement strategies to limit access. As a soci-
ety, we would need to consider the lack of alternative thera-
pies and inequity in research funding for SCD compared with
other diseases.28 Furthermore, the population with SCD may
be especially marginalized and lacking the degree of orga-
nized advocacy seen in other disease areas such as cystic fi-
brosis. Several Medicaid programs have faced litigation in re-
sponse to access restrictions for the cost-effective DAAs for
hepatitis C.3 Extrapolating from the DAA experience would be
tenuous, as the financing of prescription benefits differs from
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Original Investigation Research
2.00 3.00 4.00
1.00 1.50 2.00
Blue line indicates mean cost per
member per month. SCD indicates
sickle cell disease.
medical benefits. Because SCD will likely present the first of
many affordability challenges in this emerging drug class, more
research is needed evaluating potential reimbursement
strategies.
These findings represent a theoretical 5-year period in
which access to gene therapy for SCD is unencumbered by mar-
ket entry delays owing to manufacturing or coverage negotia-
tions, both of which have occurred in Europe with this therapy
for β-thalassemia.29,30 The uptake in the first year after ap-
proval may be more limited than our 7% rate owing to new
regulatory and manufacturing challenges. In addition, SCD af-
fects racial/ethnic groups experiencing barriers to accessing
care, systemic racism, and a history of exploitation within the
US health care system. Even if the initial years after approval
demonstrate limited diffusion, it is possible that annual de-
mand may exceed the 166 individuals treated in year 1 of our
model.
Upfront and 5-year annuity payment models were ex-
plored given uncertainty regarding the payment model to be
used in the US. As seen in Figure 1, the annuity payment de-
creases the short-term budget impact, partly by moving some
expense beyond the 5-year horizon. Such an approach may be
desirable for payers looking to minimize short-term uncer-
tainty and risk, but it provides no inherent savings or benefit
when assuming a societal perspective. In Germany, a 5-year
installment plan has been offered, with payment being con-
tingent on patients with β-thalassemia remaining transfu-
sion independent.31
For US payers, outcome-based contracting may be appeal-
ing for mitigating risk, although experience implementing such
contracting is limited. Moreover, health system incentives and
infrastructure may impede large-scale implementation of out-
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 Research Original Investigation A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease

come-based reimbursements. Although there is uncertainty
regarding alternative financing methods in the US, many agree
that a nonstandard approach is needed.32,33 As evidenced by
the controversy surrounding volume-based managed entry of
the DAAs to the market for hepatitis C, such an approach for a
curative therapy for SCD is unlikely to be accepted.
One-way sensitivity analysis demonstrated that the rate
of market diffusion and the percentage of patients meeting eli-
gibility criteria were both highly associated with the budget
impact (Figure 2). Although the potential price of the therapy
has received much attention, the price has a relatively mod-
est impact when much uncertainty exists regarding the size
of target population and market diffusion.
Our analyses synthesize available data and are not meant
to suggest coverage decisions for Medicaid programs. Rather,
they are meant to inform preexisting methods guiding cover-
age decisions based on states’ unique context. Payers will need
to make decisions in the context of the disease burden among
enrollees, as well as availability of alternative treatments (ie,
comprehensive care and allogeneic HSCT).
The therapy’s price was extrapolated from European pric-
ing for a different indication. We presumably underesti-
mated the true cost of the therapy by omitting ancillary costs
associated with preparation, administration, and follow-up.15
We assumed that the therapy cured all recipients and pre-
vented any SCD-related costs in subsequent years. Even if the
therapy proves to be this effective, organ damage from SCD may
require ongoing care. Future analyses need to consider such
health care use as clinical trial data mature. We intended to
demonstrate how even an ideal therapy may still present short-
term fiscal challenges when limited to those with severe dis-
ease. Last, our study does not consider potential improve-
ments in quality of life or other meaningful outcomes—here
we encourage future inquiry, including cost-effectiveness
analysis.
Our findings cannot be generalized to other payer per-
spectives given the variable prevalence of SCD. In addition,
variation in payer contracting and financing will influence af-
fordability.

Limitations
This study has some limitations. As with any theoretical analy-
sis, the validity of the model inputs determines the validity of
the findings. For example, our estimation of the proportion of
patients with the severe phenotype meeting eligibility crite-
ria was imprecise, with no data available to inform the param-
eter. The market diffusion rate was also a challenging param-
eter given the paucity of data for this drug class. Patients with
SCD may be reluctant to be early adopters or early commer-
cial success may increase demand. In addition, the eligibility
criteria used may differ from the eventual product label as one
trial plans to enroll children aged 2 to 14 years.25
Conclusions
A gene therapy for severe SCD is likely to produce a consider-
able budget impact for many Medicaid plans while early clini-
cal trial data suggest that this therapeutic class may consid-
erably improve the lives of patients. With payers needing to
balance short-term budgets, we expect some action will need
to be taken to ensure affordability. This may prompt Medic-
aid plans to increase revenue, restrict access, or establish al-
ternative reimbursement methods. Gene therapy for SCD is
likely to provide an early demonstration of the unique finan-
cial challenges associated with this emerging drug class.

ARTICLE INFORMATION REFERENCES 5. Seeley E, Chimonas S, Kesselheim AS. Can

Accepted for Publication: December 2, 2020.
Published Online: March 22, 2021.
doi:10.1001/jamapediatrics.2020.7140
Correction: This article was corrected on June 7,
2021, to fix an error in the text.
Author Contributions: Dr DeMartino had full
access to all the data in the study and takes
responsibility for the integrity of the data and the
accuracy of the data analysis.
Concept and design: All authors.
Acquisition, analysis, or interpretation of data:
DeMartino, Haag, Hersh, Caughey.
Drafting of the manuscript: DeMartino, Haag, Roth.
Critical revision of the manuscript for important
intellectual content: DeMartino, Hersh,
Caughey, Roth.
Statistical analysis: DeMartino, Haag, Hersh, Roth.
Administrative, technical, or material support:
DeMartino.
Supervision: DeMartino, Roth.
Conflict of Interest Disclosures: Dr Roth reported
receiving personal fees from Genentech, Bristol
Myers Squibb, and personal fees from Bayer
outside the submitted work. No other disclosures
were reported.
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