Assessing the impact of single or short-term administration on a therapy’s cost-effectiveness: a hypothetical disease-agnostic model

Figures & data

Figure 1. Model structure. Patients can only transition from progression back to baseline with treatment.

Table 1. Input values.

Parameter	Base case value	Lower bound	Upper bound	Assumption
Utilities
Baseline	1	0.1	1
Progression	0.5	Calculated	Calculated	50% of baseline utility to ensure disease progression lowers utility
Death	0			By definition
Transition probability (annual)
Baseline to progression	15%	1%	30%
Baseline to death	2%	0%	Minimum of {5%, risk of progression to death − 1%}	Risk of mortality is worse in progression than baseline
Progression to death	10%	1%	20%
Progression to baseline^a	25%	10%	50%
Hazard ratios
Baseline to progression	0.65	0.45	0.85
Baseline to death	0.9	0.82	0.98
Progression to death	0.75	0.6	Minimum of {0.9, (HR of baseline to death − 0.05)}	Treatment benefit in reducing mortality is better in progression
Costs
Baseline (annual)	$0	$0	Minimum of {$100,000, progression costs – $10,000}	Direct medical costs must be lower in baseline than progression
Progression (annual)	$100,000	$10,000	$200,000
SST therapy	$1,500,000	$100,000	$5,000,000
Chronic therapy (annual)	$54,000	Calculated	Calculated	Based on amortizing SST costs over patients' lifetime

^aOnly with hypothetical treatment.

Table 2. Impact of discounting and timing of treatment for the base case.

	Chronic therapy vs. SoC			SST vs. SoC
	3% Discounting	No discounting	% Difference with discounting	3% Discounting	No discounting	% Difference with discounting
Incremental Costs	$647,000	$1,289,000	−50%	$1,270,000	$1,289,000	−2%
QALYs Gained	6.8	14.9	−54%	6.8	14.9	−54%
ICER per QALY	$95,000	$86,000	10%	$186,000	$86,000	116%

Notes: Results rounded to nearest $1,000. Equal discounting was applied to costs and health benefits.

Abbreviations. SoC, standard of care; SST, single or short-term therapy.

Table 3. Impact of varying discount rates on ICER per QALY.

			Costs
			0.0%	1.5%	3.0%	5.0%
Chronic therapy vs. SoC	Benefits	0.0%	$86,000	$59,000	$43,000	$32,000
		1.5%		$90,000	$66,000	$49,000
		3.0%			$95,000	$70,000
		5.0%				$104,000
SST vs. SoC	Benefits	0.0%	$86,000	$85,000	$85,000	$87,000
		1.5%		$130,000	$130,000	$133,000
		3.0%			$186,000	$190,000
		5.0%				$282,000

Note: Results rounded to nearest $1,000.

Abbreviations. ICER, incremental cost-effectiveness ratio; QALY, quality-adjusted life year; SoC, standard of care; SST, single or short-term therapy.

Figure 2. ICER per QALY for both hypothetical therapies with varying survival gains. Gold line represents the difference in value. Abbreviations. ICER, incremental cost-effectiveness ratio; QALY, quality-adjusted life year; SoC, standard of care; SST, single or short-term therapy.

Table 4. Impact of discounting on “value-based” pricing.

	Scenario	Annual cost of current treatment	QALYs gained	QALY gain price component	Cost-offset price component	Cumulative “value-based” price
Scenario 1^a	Pearson et al.^15 (no discounting) – recreated	$200,000	50.0	$5.00 million	$2.00 million	$7.00 million
	SST with discounting^c	$200,000	17.6	$1.76 million	$1.57 million	$3.33 million
	Equivalent chronic therapy^c	$200,000	17.6	$1.76 million	$1.57 million	$120,000/year
Scenario 2^b	Pearson et al.^15 (no discounting) – recreated	$200,000	10.0	$1.00 million	$10.00 million	$11.00 million
	SST with discounting^c	$200,000	4.8	$0.48 million	$4.83 million	$5.31 million
	Equivalent chronic therapy^c	$200,000	4.8	$0.48 million	$4.83 million	$220,000/year

Notes: Results rounded to the nearest $10,000. Each QALY gained was valued at $100,000.

Abbreviations. QALY, quality-adjusted life-year; SST, single or short-term therapy.

^aScenario 1 assesses a new cure for a fatal disease in a 5-year-old patient, who would have died in 10 years with current treatment. With the novel treatment, the patient gains 50 QALYs¹⁵. A baseline utility of 0.851 was assumed, aligning with the age- and gender-adjusted utility of the general US population, a value used by the Institute for Clinical and Economic Review in their studies when calculating equivalent life years^41,72,73.

^bScenario 2 assesses a new cure for a nonfatal disease in a 15-year-old, who would have lived for 50 more years with current treatment. With the novel treatment, the patient gains 0.2 QALYs per year in improved quality of life over the 50 years¹⁵.

^cCosts and health benefits discounted at 3% per annum.

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Data availability statement

Qualified researchers may request access to the data that support the findings of this study from Sarepta Therapeutics Inc., by contacting [email protected].