Congress Considers Priority Review Vouchers for Medical Countermeasures

Posted 12 February 2016 By Michael Mezher


Members of the House Energy and Commerce Oversight and Investigations subcommittee were briefed on recommendations at a hearing Friday for improving the nation's biodefence strategy against natural and man-made threats.

The recommendations are particularly timely as legislators and public health officials scramble to mount a response to the Zika virus outbreak across Latin America and the Caribbean.

Many of these recommendations come from the Blue Ribbon Study Panel on Biodefense's October 2015 report A National Blueprint for Biodefense, which makes 33 recommendations to "institutionalize biodefense" by centralizing biodefense leadership within the office of the Vice President, developing a unified biodefense strategy with strong budgetary support and providing stronger incentives to promote research and development (R&D) in areas critical to biodefense.

One of the major issues outlined in the report is the tendency for attention towards biodefense to shift from crisis to crisis, rather than remaining focused between outbreaks and other health emergencies.

This lack of consistency also creates reservations in the private sector for investing in biodefense, as it is not always clear that there will be a buyer for vaccines, diagnostics or other medical countermeasures (MCMs).

"Unlike products with a viable commercial market, the market for most medical countermeasures … is defined and supported solely by the government, making it a major source of research funding and the primary purchaser of vaccines, therapies and diagnostics against these unique threats," said Jim Greenwood, president of the Biological Industry Organization (BIO) and Blue Ribbon Study Panel member.

Incentivizing R&D

Later in the hearing, Rep. Kevin Cramer (R-ND) asked the witnesses what Congress could do to help the private sector "feel comfortable" with investment in MCMs.

One issue, Greenwood warned, is that funding for these countermeasures has decreased in recent years, while the number of threats has risen, as "the regulatory pathway [for MCMs] is not always clear."

"I don't know any other way to do it except with financial incentives," Shalala replied.

"One of the most important incentives in the report is the priority review voucher (PRV) … an extension of the priority review voucher program to include material threats is viewed by many as a way to offset the dramatic decline in procurement funding for MCMs," Greenwood said.

The priority review voucher program was created to incentivize R&D for products that treat rare pediatric diseases or neglected tropical diseases (NTD). Under the program, a company that gets a product approved for a rare pediatric disease or NTD can receive a voucher guaranteeing shorter review timeframe for a future product.

These vouchers are seen as hugely valuable as they can be sold on the open market; in August AbbVie payed a record $350 million to purchase one from United Therapeutics.

"It doesn't matter if it's a material threat, if it's Zika, we have to have a way to go about this," Rep. Marsha Blackburn (R-TN) said.

Responding, Greenwood called the PRV program a "no-brainer" for MCMs.

Congress is currently considering a bill that would do just that. If passed, the Strengthening Public health Emergency Response Act would add "any disease or other agent that is determined to be a material threat under section 319F-2(c)(2)(A)(ii) of the Public Health Service Act" to the PRV program.

"You don't sell those countermeasures at Wal-Mart," Greenwood said. "The companies are willing to take the risk that maybe they will fail at the science, but the investors are not willing to take the risk that if they succeed the federal government is not going to be prepared to reward them."

Government Partners

Anthony Fauci, director of the NIH's National Institute of Allergy and Infectious Disease (NIAID), echoed this sentiment at separate House and Senate hearings on Zika earlier this week, saying his agency was unable to find a partner to develop a West Nile virus vaccine.

"Even though we went through Phase I with good safety and immunogenicity, we could not find a pharmaceutical company that wanted to partner with us because it was felt that this was not something that would have a good profit," Fauci said.

During today's hearing, Rep. Paul Tonko (D-NY) asked whether government programs that fund the development of MCMs, such as the HHS Centers for Innovation in Advanced Development and Manufacturing, are helping make a difference in driving private sector interest.

"They can play a very critical role. For example, new diagnostics have a very difficult time getting approval to be paid for, so that discourages innovative biotech companies from making them. Imagine the difference it would make it we had a rapid diagnostic test for Zika [or] Ebola," said Tara O'Toole, former under secretary of the Science and Technology Directorate in the Department of Homeland Security.

"The technologies for a whole host of new diagnostics are out there, the path to making money on them is very, very troubled … from a regulatory point of view … and the return on investment is not nearly as great," she said.

Another issue, according to O'Toole, is that funding for the government's strategic stockpile of vaccines has stayed flat, making it difficult to replenish expired products and pay for newly developed ones.

Former Secretary of the Department of Health and Human Services Donna Shalala suggested that an alternative to the national stockpile would be to focus on the production of vaccines to rapidly respond to new outbreaks.

To accelerate the development diagnostics, O'Toole suggested that the government could maintain a "curated bank" of disease samples that are considered to be a potential public threat. This, she said would help companies "much more rapidly give FDA useful data on how well their test works."

Energy & Commerce Hearing

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Categories: Biologics and biotechnology, In vitro diagnostics, Research and development, Regulatory strategy, Submission and registration, News, US, Latin America and Caribbean, FDA

Tags: Biodefense, Blue Ribbon Study Panel on Biodefense, Medical Countermeasures, MCMs, Priority Review Voucher, PRV, Zika Virus, Ebola, Bioterrorism

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