October 27, 2016

The Honorable Nancy Pelosi
Democratic Leader
United States House of Representatives
Washington, DC 20515

Subject: Reconsideration of support for impending 21st Century Cures Act (H.R. 6) bill

Dear Minority Leader Pelosi:

On behalf of Treatment Action Group (TAG), I write to you strongly urging for the reconsideration of your support for the passage of the 21st Century Cures Act. Even as an impending and new version of the bill is set to be introduced by Energy and Commerce Chairman, Rep. Fred Upton – we are concerned that several provisions carried from the prior iteration of the bill (H.R. 6) may continue to undermine public health and jeopardize patient safety. These concerns include lowering standards for the FDA in determining approval for prospective drugs and devices, increasing the likelihood of antimicrobial resistance, to granting extended market exclusivity for new drugs.

TAG is an independent, science-based research and policy think-tank fighting for better treatment, vaccine and a cure for HIV/AIDS, tuberculosis (TB), and hepatitis C (HCV). We work closely and interface extensively with U.S. research and regulatory institutions to centralize community engagement and ethically accelerate vital research.

Although TAG supports provisions of the 21st Century Cures Act that provides a needed fiscal boost to the NIH to conduct vital research and supporting young scientists to work on cures, we caution that iterations of the bill are ultimately flawed. Specifically, the key concerns with the bill is that it allows for:

Bypassing phase III trials: Phase III trials remain the gold standard by which approvals for new drugs and devices are rigorously assessed for their clinical efficacy and safety in patients. An early version of the 21st Century Cures Act, however, would have allowed for drug manufacturers to bypass conducting phase III trials. Instead, data after conducting phase II trials – such as biomarkers, surrogate markers, patient testimonials, and case studies – would accordingly be sufficient and valid in determining FDA approval under the act. Allowing for drug and device developers to bypass phase III trials substantially weakens the regulatory authority of the FDA in utilizing sound evidence-based standards for approving new medical technologies, while placing unethical and unnecessary risk upon patients with potentially unsafe and harmful treatments. We strongly you to reject any proposals that include this provision.

Monopolizing pricing, limiting access: The act permits an additional 6-month exclusivity period to an already granted exclusivity period, if a drug is given an orphan designation to treat a rare disease. The exclusivity period, however, extends to all indications of the drug, including the orphan indication. In effect, this creates a monopoly in the pricing of the drug that can thereby limit patient access to a potentially lifesaving treatment even for a broad set of health conditions. According to the Congressional Budget Office, extending the exclusivity just six months would cost federal taxpayers $869 million through Medicaid, Medicare and other federal health programs from 2016-2025. Extended exclusivity may also continue to restrict the entry of cost-saving generic medications into the market, especially during a time when increasing drug costs and limited access continues to be an issue garnering intense media spotlight and public scrutiny.

Approving Drugs With Lower Safety Standards: The legislation would allow for approval for new antibiotics without conventional clinical trials in the event of treating patients facing situations of unmet medical needs or life-threating diseases. Other forms of less reliable evidence could be then used to supplant hard clinical data to meet requisite approval standards of the FDA. While any antibiotics approved through this pathway would be reflected on drug labeling, but even with disclaimers there is very little evidence to suggest that health practitioners restrict prescribing to appropriate patients.

Accelerating antimicrobial resistance (AMR): In fact, the bill would also incentivize health care institutions and professionals through financial incentives for dispensing new, yet unproven, antibiotics. This in turn encourages the pervasive use or overuse of unproven antibiotics and thereby increases the risk of the development of AMR – growing public health threat currently being undertaken by governments and global health institutions.

Despite being underfunded, the FDA as a regulatory agency leads the world in its efficiency in approving new and innovative medical technologies. For example, the agency already provides several pathways and mechanisms to accelerate the approval of drugs and devices. Over an 11- year period, 56 percent of new drugs were approved via accelerated pathways. Research has additionally shown that the FDA approves new therapeutic agents much faster than its stringent regulatory counterpart agencies in Europe and Canada. To target the FDA as a barrier to medical innovation, a crux of this bill, is unfounded.

Furthermore, reducing the regulatory power of the FDA is not the solution to accelerate the proliferation of new treatments, cures and vaccines. In fact, increasing the ability and strengthening the capacity of the FDA to handle drug applications and approvals may increase the pipeline of new medical technologies. The problem with bringing new cures to market has less to do with the FDA standing in the way of patients, and more to do with the need for government supported basic science and R&D, to create more new cures, diagnostics, vaccines and other therapies. Increasing NIH funding to advance current basic sciences, diagnostics, treatments, vaccines, prevention and operational research would be immensely impactful, especially for neglected diseases like TB that heavily relies on publicly-funded research provided through the leadership of the US government. But any promise of funding for research cannot be done in exchange for jeopardizing patient safety and risking access to unproven, potentially unsafe drugs and devices.

We recognize that you are faced with a challenge in crafting and moving forward legislation with limited time at the end of year during a lame-duck session of Congress. We also recognize that the 21st Century Cures Act has received considerable bipartisan praise and support. But instead of yielding to bipartisan pressure to pass the bill hastily, we implore you to strongly reconsider and withdraw your support for the 21st Century Cure Act on the basis that it will greatly strip the regulatory power of the FDA and will be more harmful, than hopeful, to public health.

We thank you for your continued leadership in the fight against HIV/AIDS and other global health issues. Should you have any questions, please contact TAG policy staff Kenyon Farrow at kenyon.farrow@treatmentactiongroup.org and Suraj Madoori at suraj.madoori@treatmentactiongroup.org.

Sincerely,

Mark Harrington
Executive Director
Treatment Action Group