The Zika loopholes

The FDA has a smart-sounding program to drive research for neglected diseases—with two big flaws.

Zika.jpgPolitico; March 25, 2016. By Willo Brock, Rachel Cohen, Jason Cone and Lindsay McKenna. Last week, the Senate voted to add Zika to the list of neglected diseases for which pharmaceutical companies and other entities doing research and development for neglected diseases can receive a fast-track review for another product from the Food and Drug Administration. At first glance this seems like an apt move—the program was designed to encourage the development of treatments and vaccines for neglected diseases, and Zika is a disease for which there are currently no treatments or vaccines.
But the program, created in 2007, is in urgent need of reform. It has two key loopholes that fail to guarantee it will really help people who suffer from neglected diseases like Zika, Chagas disease and tuberculosis. As the House takes up the Senate-passed legislation, lawmakers have an important opportunity to make sure the program truly encourages the research and development of new and innovative medicines for neglected diseases.

The program was intended to respond to a real, pressing need: Neglected diseases without appropriate treatment or prevention options. We have seen the human cost of neglect most recently with the Ebola outbreak, where thousands died from a disease for which no approved vaccine or treatment exists. And we’ve seen it for decades with silent killers like Chagas disease, African sleeping sickness, and drug-resistant tuberculosis – where patients struggle with old, toxic and ineffective treatments. It’s estimated that more than 300,000 people in the U.S. alone are infected with the parasite that causes Chagas.

In 2007, Congress passed legislation to promote research for new treatments and vaccines for such neglected diseases. If a company, research institution or organization successfully registers a product with the FDA from a list of eligible neglected diseases, it is rewarded with a voucher, known as an FDA priority review voucher, allowing it to fast-track any other product in its portfolio through the FDA regulatory process. A PRV can be redeemed by the sponsor or sold to another company, making them economically valuable. In August, a pharmaceutical company purchased a voucher for $350 million.

It may seem strange that vouchers are non-disease-specific and tradeable but this isn’t a fatal flaw in the program: It was designed as a way to encourage companies and others to embark on the expensive research and development process of new treatments and vaccines for neglected diseases. Allowing pharmaceutical companies to fast-track any of their products creates a greater incentive that they’ll invest in neglected diseases.

However, two changes must be made to ensure the program works as intended.

First, there is no novelty requirement to obtain a PRV for a neglected tropical disease. In other words, a PRV can be awarded for an old medicine that has been used for years for the treatment or prevention of that neglected disease, as long as it has not been registered with the FDA. In fact, this is exactly how the first PRV was awarded: In 2009Novartis obtained a PRV for an existing malaria treatment. It happened again in 2014, when a PRV was awarded to Knight Therapeutics for a leishmaniasis treatment, miltefosine, that has been in use since 2004, well before the PRV program was even created.

Second, the PRV does not provide any guarantee that the people who most need the drug or vaccine will ever get it. Under the current scheme, if a group develops a Zika vaccine next year, it could receive a voucher even if it does not register the product in affected countries or if it prices the product out of reach for people in those countries. For instance, treatment providers have struggled to access miltefosine at an affordable price and have experienced considerable registration and supply issues for years.

The need for reform is urgent to avoid further abuse. Now, the biotech company KaloBios is also trying to game the PRV program. Until his indictment and arrest, KaloBios was led by Martin Shkreli, who gained notoriety for raising the price of a drug used to treat a deadly parasitic infection affecting people with HIV and cancer by 5,000 percent overnight. KaloBios is trying to buy the rights to a decades-old treatment for Chagas disease and register it in the U.S. in order to obtain a PRV. The drug is currently only available to U.S. patients through a compassionate use program run by the Centers for Disease Control & Prevention. The company announced plans to sell this drug, which costs a little under $100 in Latin America, at about $90,000 per treatment. Under the current legislation, KaloBios could also sell the PRV for millions without doing any innovation for Chagas patients at the same time it drastically inhibits access to existing treatment for those in need.

In November, seven global health organizations sent a letter to Senate Committee on Health, Education, Labor, and Pensions leadership asking them to fix critical flaws in the program by introducing a requirement that the products earning a PRV be new and accessible to the people who urgently need them. Now, as the House considers adding Zika to the list of neglected diseases eligible for a PRV, this is a unique opportunity to restore the original intent of the program.

Lawmakers should add two provisions to the bill, one requiring that PRVs only be granted to products that are truly novel in terms of introducing new therapeutic or preventative options for patients, and the other requiring companies to consider how products to treat or prevent neglected diseases will be made available and affordable to the people affected by neglected diseases. These changes will help ensure that the PRV program achieves what it should: Encourage investment in innovative products, and make them available to the communities and treatment providers who need them most.

Jason Cone is the executive director of Doctors Without Borders/Médecins Sans Frontières (MSF)-USA. Rachel M. Cohen is the regional executive director for the Drugs for Neglected Diseases initiative North America. Lindsay McKenna is the TB/HIV project officer for the Treatment Action Group. Willo Brock is the senior vice president of external affairs for the Global Alliance for TB Drug Development.

This entry was posted in Innovation, Neglected Diseases, Priority Review. Bookmark the permalink.

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