Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act Introduced

Earlier this month, U.S. Senator Kay Hagan (D-NC) introduced a bill that would accelerate the Food and Drug Administration (FDA) approval process to get targeted treatments to patients with serious or life-threatening diseases. Known as the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act, the legislation would cover therapies for conditions that do not currently have treatments, targeted therapies for serious illnesses and drugs that could provide significant improvement over standard care. 

Senator Kagan said her proposed legislation is reminiscent of the system that advanced HIV and cancer drugs back in the 1990’s.  “But for patients suffering today from some diseases for which there are no current treatments or for those patients who are clutching to the hope that a better treatment will be developed for their rare diseases, medical advances cannot come fast enough,” said Hagan. 

The TREAT Act would accelerate the review and approval process for medicines that treat an unmet medical need, significantly advance the standard of care, or are highly targeted therapies for serious or life-threatening diseases or conditions.  The bill is being referred to the Senate Health, Education, Labor and Pensions (HELP) Committee, which Hagan is a member of. 

In a press release from Kagan announcing the bill, she noted how the legislation will ensure the best science and greater innovation in medical treatments with better access to expertise, while maintaining the FDA’s high safety and effectiveness standards.  The TREAT Act does this by:  

• Enhancing FDA access to external Scientific and medical expertise. This is critical in areas where research is on the cutting edge.
  • It allows the FDA Commissioner to better utilize waivers when potential conflicts of interest are outweighed by the need to have input from leading medical and scientific experts.
• Including patient advocates and disease research organizations in Advisory Committee reviews.
• Ensuring that FDA provides drug sponsors with explanations of why their applications are rejected so that they might address concerns, improve treatment and get the medications to patients
• Updating the FDA Mission Statement to reflect FDA’s role in advancing medical innovation
• Creating a Management Review Board to enable the FDA to keep pace with the latest scientific advances
• Establishing a Chief Innovation Director to ensure rapid development, testing and review of new drugs and devices
• Establishing Chief Medical Policy Officers to address emerging medical and scientific policy issues at the Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), and the Center for Devices and Radiological Health (CDRH).
• Establishing a Clinical Informatics Coordinator to develop, implement and promote FDA’s use of Health Information Technology (HIT) in clinical research.

The TREAT act does not authorize any government funding, and is not expected to receive any funding through the appropriations process.  Instead, this bill transforms FDA’s internal processes to ensure more consistency and clarity. 

The Biotechnology Industry Organization (BIO) praised Sen. Kay Hagan (D-NC) for introducing the (TREAT) Act.  In fact, BIO President and CEO Jim Greenwood maintained that the TREAT Act will “help unleash the promise of biotechnology to speed life-saving cures, novel medicines and scientific breakthroughs  to patients living with debilitating diseases such as cancer, HIV/AIDS, Parkinson’s and diabetes.”   

“The TREAT Act will help break the institutional barriers to a faster idea-to-market pathway and speed new cures to patients most desperately in need.  Through this bill, Senator Hagan has offered renewed hope to patients desperately in need of cures and breakthrough new medicines as well as a boost to our nation’s economy.  The legislation will FDA retain its leadership position as the global ‘gold standard’ for regulatory science and consumer protection. 

“This bill will modernize the FDA and allow the agency to keep up with the fast pace of American scientific breakthroughs.  Further, it will ensure a transparent and more predictable pathway for the development of safe and effective next generation medicines and cures while maintaining U.S. leadership in biomedical innovation.  A more streamlined and forward-thinking regulatory review process will ensure that innovative biotech firms can focus their resources where they belong – in research and development that leads to breakthrough medicines and cures, and job creation – rather than navigating an overly complicated regulatory process that discourages the innovation that leads to cures.  

“This new legislation includes a provision to modernize the Accelerated Approval pathway to expedite the development of modern, targeted, and personalized therapies for patients suffering from serious and life-threatening diseases, while preserving robust standards for safety and effectiveness.  The pathway would apply only to treatments for diseases or conditions that are serious or life-threatening.  

The accelerated approval process, instituted in 1992 at the behest of AIDS activists, gives the FDA a mechanism to tentatively approve drugs after phase II if a drug shows promise in a serious disease using a surrogate endpoint. The FDA website explains that, “instead of having to wait to learn if a drug actually can extend the survival of cancer patients, the FDA might now approve a drug based on evidence that the drug shrinks tumors because tumor shrinkage is considered reasonably likely to predict a real clinical benefit.  In this example, an approval based upon tumor shrinkage can occur far sooner than waiting to learn whether patients actually lived longer.” 

The problem with the accelerated approval process notes Forbes, “is that it is only used in very narrow areas, such as cancer and AIDS, where the FDA has to its satisfaction established surrogate markers that it believes to correlate to broader clinical outcomes (such as tumor shrinkage being a surrogate for cancer survival).  But there are a lot of areas where surrogate markers aren’t as easy to define.  In addition, the FDA focuses on using the accelerated approval process to address life-threatening diseases like cancer, neglecting serious but more chronic ailments such as diabetes, where the cost of clinical trials is prohibitive.” 

Discussion 

Despite Hagan’s efforts, a recent article from Forbes alleged that the pharmaceutical industry “helped to kill an earlier version of the TREAT Act, which would have created an entirely new “progressive and exceptional approval” pathway for innovative new drugs: something that could have turbocharged drug development in several fields.” 

According to the article, “the new pathway would have addressed a much broader range of drugs than the existing accelerated approval process, such as: the first drug approved for a specific and identifiable disease subpopulation; patients unresponsive to, or intolerant of, existing treatments; improvement in patient outcomes due to either improved safety or efficacy; or “otherwise satisfy an unmet medical need.” 

Unlike the accelerated approval process, which requires an FDA-sanctioned surrogate marker, the new process would involve a case-by-case negotiation between the industry sponsor and the FDA, in which both sides could come to an agreement as to what would demonstrate an early signal of clinical benefit.  If confirmatory phase III studies didn’t reproduce that benefit, the FDA could revoke its approval, as with the Avastin case. 

“Sen. Hagan’s proposal would have been devastating to the big pharma R&D oligopoly. If small biotech companies could get their drugs tentatively approved after inexpensive phase II studies, they would have far less need to partner those drugs with big pharma. They could keep the upside themselves and attract far more interest from investors. Big pharma, on the other hand, would be without its largest source for innovative new medicines: the small biotech farm team.” 

Accordingly, the article maintained that PhRMA objected to Sen. Hagan’s proposal, instead arguing for window-dressing reforms to the existing accelerated approval process. Instead of allowing for phase II data to achieve approval for a wide range of drugs, the new TREAT Act merely allows for the existing, surrogate marker-driven accelerated approval process to address “highly targeted therapies for distinct subpopulations.” Relative to the bold, innovative proposal contained in the bill’s original version, the new version amounts to window dressing. 

“There were four major groups that played a role in the change,” according to Forbes. On the pro-innovation side were the biotechnology industry, as represented by BIO, and reformist legislators on Capitol Hill, such as Sen. Hagan and Sen. Mike Enzi (R., Wyo.). In favor of window dressing were PhRMA and the FDA, which “wasn’t ready” for the change.  Forbes also noted that “certain consumer activist groups raised concerns that the change would allow unsafe drugs to make it to market.” 

The FDA, too, is incentivized to promote “safety,” by slowing down drug development, than to look out for the interests of ill patients who need novel medicines. Last month, Steve Usdin of BioCentury interviewed FDA Commissioner Janet Woodcock, who poured cold water on Hagan’s progressive approval proposal, calling it “a new scheme for getting drugs on the market that substantially lowers the standards for safety or for effectiveness.” 

It is likely that this legislation will try to be placed in the fifth reauthorization of the Prescription Drug User Fee Act, the law that has come to govern the pharmaceutical regulatory process at the FDA. 

Conclusion 

BIO President Greenwood asserted that, “It is simply unacceptable that in the United States, as patients suffer and die, the time to take a new drug from discovery to patient is 10 to 15 years.”  He noted how “regulatory uncertainty is having a major negative impact on the private funding of biomedical innovation, deteriorating the ability of our member companies to deliver cures and new medicines.”  Sixty-one percent of venture capitalists now cite FDA regulatory challenges as having the highest impact on their investment decisions, and 40% expect to decrease investment in the development of new therapies.   

“The need to reform the investment and regulatory environment for biotechnology innovation has never been more urgent.  Only by transforming the policy environment can we develop new cures and medicines, grow a robust 21st century innovation economy, maintain our nation’s global leadership in biotechnology, continue to create high-paying jobs in the U.S and prevent the tsunami of additional health care costs that will be associated with the aging baby-boomer population,” said Greenwood. 

He noted how Biotech companies hold the greatest promise for finding cures to life-threatening diseases. There are currently 800 new treatments being developed for cancer by biotech companies, 300 for heart disease, 200 for diabetes, and 100 for Alzheimer’s. When biotech finds cures it saves lives and dollars. Reducing cancer death rates by just 10 percent would have a $4 trillion economic impact. Delaying the onset of Alzheimer’s by just five years could save up to $50 billion per year.