Priority review voucher

Priority Review Voucher (PRV) is a policy tool used by the United States Food and Drug Administration (FDA) to encourage the development of treatments for diseases that are often overlooked by the pharmaceutical industry. This includes rare pediatric diseases, tropical diseases, and conditions that lack significant treatment options. The PRV program allows a company that develops a drug for such diseases to receive a voucher for priority review of another drug, which can significantly reduce the time it takes for the latter drug to reach the market.

Overview

The concept of the Priority Review Voucher was introduced as part of the Food and Drug Administration Amendments Act (FDAAA) of 2007. The goal was to create an incentive for pharmaceutical companies to invest in drugs for diseases that affect fewer people or are prevalent in parts of the world with less economic potential for drug sales. When a company receives a PRV, it can either use it for a future drug application or sell it to another company. This has created a secondary market for PRVs, with prices for vouchers reaching up to hundreds of millions of dollars.

Types of Priority Review Vouchers

There are three main types of PRVs:

1. Tropical Disease Priority Review Voucher: This type of voucher is awarded for the development of treatments for specific tropical diseases. The list of eligible diseases is defined by the FDA and includes conditions such as malaria, tuberculosis, and dengue fever.

2. Rare Pediatric Disease Priority Review Voucher: This voucher is aimed at encouraging the development of treatments for rare pediatric diseases. A disease qualifies as a rare pediatric disease if it affects individuals aged from birth to 18 years and meets the criteria for a rare disease (affecting fewer than 200,000 people in the United States).

3. Medical Countermeasures Priority Review Voucher: Introduced to incentivize the development of treatments for chemical, biological, radiological, and nuclear threats. This category is part of the U.S. government's efforts to prepare for and respond to public health emergencies.

Process and Impact

To qualify for a PRV, a drug must be approved by the FDA, and the application must meet certain criteria, including the demonstration of safety and efficacy. Once awarded, a PRV can be used to expedite the FDA review process for a new drug application (NDA) or biologics license application (BLA) from the standard ten months to six months. This expedited process can be crucial for getting potentially life-saving drugs to the market faster.

The impact of the PRV program has been mixed. While it has successfully incentivized the development of treatments for neglected diseases, critics argue that the program is expensive and may divert the FDA's resources away from other important drugs. Additionally, the high value of PRVs has raised concerns about the program's potential to prioritize profit over public health needs.

Controversies and Reforms

The PRV program has faced criticism for various reasons, including the high prices of vouchers and the potential for companies to game the system by developing drugs that meet the minimum requirements for a PRV without providing significant advancements in treatment. In response to these concerns, there have been calls for reform, including proposals to limit the transferability of PRVs, adjust the criteria for qualifying diseases, and increase transparency in the program.

Conclusion

The Priority Review Voucher program represents an innovative approach to addressing the challenge of developing treatments for neglected diseases. While it has achieved some success, ongoing debates about its effectiveness and fairness suggest that further adjustments and oversight may be necessary to ensure that it fulfills its intended purpose without unintended consequences.