October 3, 2017

The Honorable Michael C. Burgess, MD
Chairman, Health Subcommittee
Energy and Commerce Committee
United States House of Representatives
Washington, DC 20515

The Honorable Gene Green
Ranking Member, Health Subcommittee
Energy and Commerce Committee
United States House of Representatives
Washington, DC 20515

Dear Chairman Burgess and Ranking Member Green:

Treatment Action Group (TAG) appreciates the opportunity to submit these comments to the Health Subcommittee of the U.S. House of Representatives Energy and Commerce Committee, in association with its hearing, “Examining Patient Access to Investigational Drugs,” and the deliberations of H.R. 1020, the Compassionate Freedom of Choice Act of 2017, and S. 204, the Right to Try Act of 2017. TAG is an independent, activist and community-based research and policy think tank fighting for better treatment, prevention, a vaccine, and a cure for HIV, tuberculosis (TB), and hepatitis C virus (HCV). For 25 years, TAG has strongly advocated for expedited access to drugs and biologics with the greatest potential to save human lives. However, we also remain committed to stringent regulatory practices designed to minimize risk, confirm efficacy, and to protect consumers from harmful commercialization practices.

In July 2017, TAG joined with National Center for Health Research in urging the Senate to reject S. 204.[1] We reiterate here that any legislation aiming to circumvent existing expanded access processes authorized and monitored by the Food and Drug Administration (FDA) to help and protect patients with serious or life-threatening illnesses is unnecessary and dangerous.

In the early 1990s, due in large part to the influence of HIV/AIDS activism, the FDA formalized compassionate use and expanded access programs to provide patients with serious or life-threatening diseases with access to experimental drugs that have demonstrated reasonable safety and potential efficacy in phase II clinical trials, and are undergoing further investigation in phase III trials. These programs have been a lifeline for U.S. residents living with HIV, particularly those with virus resistant to approved antiretrovirals (ARVs), those unable to tolerate approved ARV options, and those unable to access phase III trials due to enrollment, distance, or entry criteria restrictions. They have also been vital for people affected by other life-threatening conditions, such as forms of TB and cancer that approved treatment options cannot cure.

There is empirical evidence that existing expanded access programs are sufficient for patients with serious or life-threatening illnesses. A 2016 analysis conducted by the FDA Center for Drug Evaluation and Research (CDER) found that more than 1,000 expanded access applications are received by the agency each year, the vast majority of which (99.7%) are allowed to proceed.[2] A follow-up analysis confirmed the high number of expanded access applications approved by CDER, while also underscoring FDA commitments to patient protections.[3] Between January 2005 and December 2014, 99.3% of almost 9,000 expanded access applications were approved, with only 38 emergency treatment investigational new drugs (INDs) denied and 23 non-emergency treatment INDs not allowed to proceed. The most common reasons for denying emergency INDs was that the patient was stable on current therapy and that it was not deemed an emergency. The most common reasons for not allowing non-emergency expanded access INDs to proceed were incomplete application, unsafe dosing, demonstrated lack of efficacy for intended use, availability of adequate alternative therapies, and inadequate information provided in the application on which to base a decision.

TAG strongly supports the needs of people living with HIV, TB, and HCV to access promising drugs and biologics as quickly as possible and remains committed to the continuity of ethical and scientifically sound mechanisms in place to ensure patients with limited or no treatment options have access to the most promising investigational agents. Right-to-Try legislation in no way improves on these mechanisms and only stands to compromise patient safety and, additionally, create a lax legal and regulatory environment for the pharmaceutical industry. We urge the Health Subcommittee of the Energy and Commerce Committee to consider the following:

• H.R. 1020 and S. 204, effectively undermine the current requirement that pharmaceutical companies develop, implement, and complete the registrational trials necessary to confirm safety and efficacy in patients with serious and life-threatening illnesses. Not only are these data necessary to support FDA approval indications, they are essential to clinicians and patients in making informed treatment decisions. Legislation that allows manufacturers to circumvent stringent regulatory approval requirements to instead focus on commercializing its products to desperate patients—particularly with statutory language freeing manufacturers of any liability[4]—is a step in the wrong direction.
• No risk analysis or evidence of potential efficacy is required in H.R. 1020 or S. 204. In fact, the proposed legislation stipulates the removal of the FDA from any safety and efficacy determinations and, no less worrisome, would prevent the agency from collecting data from clinicians treating patients with an investigational agent that can be used in safety and efficacy determinations if/when the agent is submitted for approval.[5] Existing expanded access programs not only allow for access to experimental agents, they contribute to the data sets that inform approval, labeling, and best practices—which protect patients, their providers, and companies alike.

TAG believes it is reasonable and necessary to allow the FDA to retain its regulatory oversight for expanded access and compassionate use programs in order to help mitigate safety concerns, ensure preliminary efficacy to guide risk-benefit determinations, and buttress the need for clinical trial data to inform registrational approval and prescribing practices. Especially since the agency approved more than 99% of expanded access requests, FDA’s role in reviewing preapproval access requests is clearly not an impediment, and provides important oversight. H.R. 1020 and S. 204 do nothing for people who are terminally ill. It instead aims to curtail vital FDA stringency requirements that have not only largely succeeded in protecting public health, but continue to be effectively streamlined to hasten access to investigational and approvable drugs and biologics for those who need them most.

Respectfully submitted,

Tim Horn
Deputy Executive Director, HIV & HCV Programs

Erica Lessem
TB/HIV Project Director