兰德-研发成本透明度要求增加的影响（英）-80页_726kb

R&D Cost Transparency Literature Review Summary

Key Findings

1. R&D Cost Definition & Estimation

   • The R&D cost of a new medicine includes three main components:
     1. Out-of-pocket (OOP) costs: Direct expenses (e.g., supplies, staff), often aggregated at the organizational level.
     2. Cost of capital: Accounts for the time value of investment, influenced by firm-specific risks.
     3. Attrition rate: Reflects the failure rate of candidates, typically calculated from clinical trial data.
   • Estimates vary widely due to differences in methodology (e.g., capital allocation, attrition calculation) and sources (e.g., Tufts-CSDD, stock market data). NGOs like DNDI and TB Alliance publish individual medicine costs, demonstrating feasibility but high data aggregation barriers.

2. Transparency Scope & Stakeholder Views

   • Supporters argue transparency improves accountability (payers), fosters innovation (investors), and enables fairer pricing.
   • Opponents highlight risks, including predatory use by competitors (discouraging competition) and administrative burdens.
   • No consensus on defining transparency; most discussions assume disclosure of OOP costs, timelines, capital, and attrition rates.

3. Proposed Actions

   • Legislation/regulation: Mandating disclosures (e.g., U.S. Inflation Reduction Act), similar to healthcare professional payments.
   • Improved data systems: Enhancing financial reporting and utilizing open-source platforms for collaborative verification.
   • Public-private collaboration: Leveraging partnerships to reduce duplication and share R&D risks.

4. Expected Advantages/Disadvantages

   • Advantages: Clarity for investors, better resource allocation, trust in companies, and fairer pricing.
   • Disadvantages: Potential for reduced competitiveness, administrative costs, and strategic information loss.

5. Policy Implications & Uncertainties

   • International policies (e.g., WHO’s WHA72.8 resolution, EC’s Pharmaceutical Strategy) aim for R&D transparency, but implementation is complex.
   • Evidence lacks clarity on transparency’s impact on prices, access, and innovation. Challenges include commercial confidentiality concerns and inconsistent global regulatory frameworks.

Conclusion

While R&D transparency varies across industries and funding sources, the literature suggests it balances accountability and efficiency benefits against risks to innovation. Greater transparency could improve pricing negotiations and resource allocation but may foster adversarial competition. Further empirical studies and policy implementation are needed to address current uncertainties.