从专利到患者：FDA政策构想助力医疗创新加速与保护

A Blueprint for FDA: Recommendations to Improve Innovation and Access [1] (FDA Blueprint) is a timely white paper authored by University of Southern California scholars that identifies U.S. Food and Drug Administration (FDA) policies that impact innovation and suggests improvements to sustain incentives while ensuring timely, affordable access to medicines.

Innovation Access 

Leadership changes at FDA have prompted a reassessment of past regulatory policies to better align with rapid scientific and technological advancements. [2] The agency recognizes the importance of updating its frameworks to accommodate innovations such as new trial designs, real-word evidence, and AI-powered systems, enabling faster access to transformative medical products while access to transformative medical products while maintaining high safety and efficacy standards. [3] 

Promoting innovation is a central component of the FDA’s mission. [4] The agency acknowledges its unique role in shaping medical product development, establishing evidence requirements, and overseeing products after they reach the market. Among many significant recommendations, FDA Blueprint argues that by reducing regulatory barriers and clarifying expectations for product sponsors, FDA can foster a more supportive environment for innovation that ultimately benefits patients. [5]

Patents, FDA Oversight, and the Path to Innovation and Access

Patents give new medicines and treatments a period of market protection, and FDA’s review pathways for generics and biosimilars are designed around those timelines. Critics sometimes argue that patents hinder innovation and merely keep drug prices high, but that’s not the full story. Patents reward investment in risky research and, after they expire, place the innovation and technology fully into the public domain, paving the way for lower‑cost competition. Once patents or exclusivity rights expire or are successfully challenged, FDA can approve generics and biosimilars, bringing competition that drives prices down. Recent policy changes, such as Medicare’s new drug pricing rules, risk weakening the financial incentives that encourage companies to launch these alternatives quickly. [6] Combined with certain FDA requirements, like added hurdles for biosimilar interchangeability, these shifts could slow patient access to affordable medicines. The FDA Blueprint calls for targeted FDA updates to preserve patent‑linked entry incentives and streamline approvals [7], ensuring the balance between rewarding innovation in its protected years and promoting timely, affordable competition after patent expiration.

Understanding How Patents Shape FDA Review and Generic/Biosimilar Competition

Understanding how patents and FDA review work together is the first step in seeing the bigger picture of pharmaceutical competition. Patents protect the years of research and risk that go into bringing a new medicine to market, but once they expire, they open the door for affordable alternatives to reach patients. Turning that opportunity into real‑world access depends on how efficiently the FDA can move products through its approval pathways. With market dynamics shifting and new policies like Medicare’s drug pricing rules altering the playing field, it’s essential to know where targeted FDA updates could make the biggest difference.

How Patents and Exclusivity Shape FDA Pathways

Patents and statutory exclusivity periods determine when lower‑cost alternatives such as generics for small‑molecule drugs and biosimilars for biologics become eligible for FDA approval. These protections serve to reward innovation by allowing developers time to recover their investment before facing direct competition. The Hatch‑Waxman Act and the Biologics Price Competition and Innovation Act (BPCIA) embody this balance in law, structuring review pathways around the expiry or challenge of patents. Key incentives, like the 180‑day exclusivity for the first generic challenger or priority for the first interchangeable biosimilar, encourage competition at the earliest possible point. [8]

Continue and Modernize FDA Competition Plans

The FDA’s Drug Competition Action Plan (DCAP) and Biosimilars Action Plan (BAP) have been important tools for guiding the efficient review and approval of post‑patent competition. To keep pace with a shifting market, the authors recommend sustaining and refreshing these programs, ensuring they continue to support timely entry once patent and exclusivity barriers fall. [9] Enhanced guidance, streamlined review processes, and proactive identification of emerging competition gaps could help maximize their impact.

Address Impacts of the Inflation Reduction Act (IRA)

The issue has been raised that Medicare pricing provisions under the IRA could undermine the financial returns that drive companies to invest in generic and biosimilar launches, especially if “maximum fair prices” are set soon after patent expiry. Lower expected revenues can reduce the appetite for challenging patents and funding entry. The authors suggest that FDA monitor the IRA’s effects closely, coordinate policy alignment with CMS, and consider supplementary incentives such as targeted expedited reviews in cases where IRA rules risk dampening competition. [10]

Streamline Interchangeability for Biosimilars

Interchangeability status is a powerful factor in market adoption post‑patent, yet current FDA requirements, often including switching studies, may slow this designation. Simplifying the interchangeability process, clarifying evidence expectations, and leveraging [11] accumulated regulatory experience could accelerate substitution at pharmacies, broadening patient access more quickly after protections expire. [12]

Balance Innovation Incentives with Affordable Access

While some view patents as barriers that keep prices high, they remain essential for supporting the development of safe, effective treatments. They incentivize high‑risk, long‑term investment in R&D, with the understanding that, after expiration, the technology becomes public property. FDA Blueprint suggests a legislative update to distinguish regulatory hurdles to expeditious review and approval, including the current “interchangeable” versus “biosimilar” product categories.

Closing Thoughts

These issues invite reflection on how patent law, FDA regulation, and evolving policy work together, and at times in tension, to shape the pace of innovation and affordability. Addressing the friction points thoughtfully could help preserve incentives for the creation of life‑changing therapies, while ensuring those treatments move swiftly from protected innovation to widespread, cost‑effective patient care.

[1] https://schaeffer.usc.edu/research/a-blueprint-for-fda-recommendations-to-improve-innovation-and-access/ (FDA Blueprint).

[2] https://natlawreview.com/article/fda-commissioner-outlines-priorities-jama-article.

[3] Id. and FDA Blueprint, executive summary.

[4] FDA Blueprint, executive summary.

[5] Id.

[6] https://www.healthaffairs.org/content/forefront/medicare-s-drug-price-negotiation-and-innovation-s-off-table-matters-too.

[7] FDA Blueprint, pages 23 and 24.

[8] Id. 

[9] FDA Blueprint, at page 23.

[10]同上。

[11] FDA Blueprint, at page 24.

[12]同上。