Food and Drug Administration (FDA) Commissioner Scott Gottlieb has adopted increased drug availability and decreased prices as a central tenet of his leadership of the agency. In a blog post published on the FDA website on October 2, Gottlieb laid out the FDA’s role in strong terms. Gottlieb said,
I consider access to medicine a matter of public health. If consumers are priced out of the drugs they need, that’s a public health concern that FDA should address, within the scope of its mandate and authorities.
While FDA doesn’t control drug pricing, our policies do affect competition in the market. This is the nexus of our current efforts on drug pricing.
Gottlieb pointed to a number of agency strategies to this end. They include improving the efficiency of the generic drug approval process, closing loopholes that allow major drug companies to inhibit generic competition, and increasing generic competition to “complex drugs.”
Complex drugs include expensive medicines such as some injectable drugs and metered dose inhalers intended for asthma sufferers. It is difficult to produce a generic version of these drugs, which means less competition. According to Gottlieb, some of these drugs are not patent protected but still face little to no generic competition. Complex drugs can also include drug-device combination products such as EpiPen.
The Commissioner has also taken a role in the “Right to Try” debate. Gottlieb testified in a hearing of the U.S. House of Representatives Energy and Commerce Committee. The hearing, titled “Examining Patient Access to Investigational Drugs” and held on October 3, focused on two pieces of legislation aimed at increasing the availability of drugs intended to treat chronic or terminal illnesses that the FDA has not approved.
One bill discussed, S. 204, commonly known as the Trickett Wendler Right to Try Act of 2017, was already passed by the Senate in August. The other bill is H.R. 1020, the “Compassionate Freedom of Choice Act of 2017.”
In his written testimony for the hearing, Gottlieb noted that he has a personal as well as professional interest in the legislation discussed. Gottlieb wrote:
Access to investigational products for patients facing serious or immediately life-threatening diseases is not an abstract issue to me. As a cancer survivor who used a commercially-available combination therapy in an off-label manner, I understand, on a very personal level, that patients who are fighting serious or life-threatening diseases want the flexibility to try new therapeutic approaches, including access to investigational medical products, particularly when there is no other FDA-approved treatment option.
He went on to state that the FDA has recently taken actions and intends to take further steps streamline its Expanded Access program, an FDA initiative that seeks to make certain investigational treatments available to patients. These actions include speeding up the application and approval timeline as well as improving public knowledge and awareness about the program.
As far as the legislation discussed at the hearing, Gottlieb’s testimony praised S. 204 but suggested a few changes. One suggested change was that the definition used in S. 204 should be altered from those whose illness is “life-threatening” to those who are suffering from a “terminal condition.” Gottlieb recommended clearly defining “terminal illness,” as well as clarifying that existing regulations around the marketing of drugs and the ability of the FDA to take enforcement actions apply to the new laws as well.