Improving Access to Rare Disease Treatments: Subsidy, Pricing, and Payment Schemes I: With Manufacturer Pricing, Subsidize Only the Manufacturer; Outcome-Based Pay Raises the Price iff B < k − 1/8Research Paper
Motivation
Treatments for rare diseases can require substantial research and development spending while reaching a small patient population. A public subsidy can cover part of the manufacturer's development cost, part of the patient's price, or both. Which recipient should receive the subsidy depends on how the manufacturer responds. A further design choice is whether the manufacturer is paid when a drug is sold or only when treatment succeeds. Olsder, Martagan, and Tang formulate these choices as a sequential game between the government, a manufacturer, and patients in their SSRN working paper, §§3–4. This mission concerns their results for the case in which the manufacturer sets the price.
The paper reports that a manufacturer subsidy alone is optimal under either payment scheme and that the relative prices under the two schemes change with the government budget. The price comparison is useful because a payment contract intended to protect patients can change the price set by the manufacturer. The mission uses the working paper dated 2019-11-13. Its page and equation numbers are those of that version, which precedes the published article.
Setting
The government first chooses two subsidy shares: covers the fraction of research cost and covers the fraction of the unit price paid on a patient's behalf. The manufacturer then chooses a nonnegative unit price and a positive target efficacy . Research cost is , with , so the manufacturer's effective cost is . The government has a budget . The market size is one, and patient valuations are uniform on ; these conventions are given in §3.1–§3.2.
In Setting (1A) the manufacturer receives the price for every sale. Its profit is . At price and efficacy , patient welfare is , while government spending is . These are the paper's equations (2), (3), and (5). In Setting (1B) payment occurs only if treatment succeeds. The corresponding expressions are , , and , from equations (7), (8), and (10) of the same source.
For each scheme, a manufacturer best response is a pair maximizing that scheme's profit over all , . A government policy is optimal when it has a best response at which spending does not exceed , and its patient welfare is at least the welfare of every feasible policy evaluated at every manufacturer best response. This expresses the government's leader role and the manufacturer's follower role in the game's sequence of events, rather than assigning the closed forms of §§4.1–4.2 to either player by definition.
Formalization targets
Government policy under each payment scheme
Proposition 1 of the working paper, p. 12, gives the unique optimal policy under sales-based payment. The paragraph after (16) on p. 13 says the same policy is optimal under outcome-based payment:
The budget binds at this policy. In both settings the optimal patient welfare and the manufacturer's profit equal . These policy claims, the two best-response formulas, and the two welfare and spending reductions form the six milestones.
Price comparison
Corollary 1 on p. 13 compares the manufacturer's optimal price in the two payment schemes. If and denote prices at optimal policies and best responses, the goal is the three-way statement
The printed second clause uses and says “higher”; equality of the two prices at the threshold is a boundary slip. The formal target records equality separately and preserves the strict high-budget comparison.
Significance
The policy result identifies which subsidy reaches patients most effectively within this model: when the manufacturer sets the price, the government uses its entire budget on the manufacturer's development cost, regardless of whether payment is per sale or contingent on treatment success. The price result adds a distinct comparison. Equal optimal subsidies and equal patient welfare do not imply equal prices; the sign of the price difference depends on relative to . This distinction appears explicitly in the paper's discussion following Corollary 1, pp. 13–14.
The working paper proves these results mathematically. The mission's remaining work is to give machine-checked proofs for the game definitions, both best-response results, both welfare and spending reductions, the unique optimum, and the final price comparison. It does not assert that those proofs have already been formalized. The definitions of attained best response and government optimality can also support later analyses of different subsidy or payment rules.
Difficulty
The government cannot compare the two schemes by holding the manufacturer's choices fixed. The manufacturer optimizes both price and efficacy after observing the subsidy policy, and the two profit functions have different price terms. Even after determining each manufacturer's response, identical formulas for government welfare and spending do not themselves establish an attained and unique optimum on the continuous half-open policy region . The price comparison then concerns the actual best-response prices at that optimum, including the threshold where the two prices coincide. These dependencies explain why the reduced expressions alone cannot serve as the definition of the game.
Formalization scope
Lean represents all parameters, policies, prices, efficacies, profit, welfare, and spending in . The policy predicate requires . Every theorem assumes ; the budget results also assume . These positivity conditions make the paper's denominators meaningful and state its implicit economic regime. Prices chosen by the manufacturer satisfy . Its efficacy choice satisfies and has no imposed upper bound of one: equations (3) and (8) maximize over , and in the high-budget part of Corollary 1 their optimizer can exceed one. This is a convention of the paper's algebraic model, even though efficacy is introduced as a probability.
The demand expressions used in the paper's equations are left unclipped. At the stated best responses, they lie in the relevant nonnegative range. Best responses are represented as sets of all maximizers, and the goal compares every response at every optimal policy. Government optimality requires a feasible best response and comparison with every feasible competing policy and response, so neither existence nor uniqueness is obtained from an empty set. In particular, the reduced welfare and budget formulas (13) and (16) are milestone conclusions, not definitions of government choice. Reusable contributions include real optimization arguments for joint price and efficacy maximization and for attained welfare maxima over subsidy policies.
Selected references
- W. Olsder, T. Martagan, and C. S. Tang, Improving Access to Rare Disease Treatments: Optimal Subsidies, Pricing, and Payment, SSRN working paper 3481150, version of 2019-11-13, §§3–4, pp. 8–14. SSRN.
- W. Olsder, T. Martagan, and C. S. Tang, Improving Access to Rare Disease Treatments: Subsidy, Pricing, and Payment Schemes, Management Science, 2023. DOI: 10.1287/mnsc.2022.4634.