Letter to HELP Committee Re: Innovation Bills

Dear Senator _______,

As members of the Patient, Consumer, and Public Health Coalition, we are writing to express our views as consumers, physicians, scientists, and public health experts regarding the health care legislation scheduled to be marked up on March 9, 2016. We want all Americans to have the best possible medical treatments, and that includes making sure that the safety and effectiveness of medical products are not compromised in the process.

That is why we are concerned that the focus of these bills is on getting medical products to market more quickly, instead of making sure they are safe and effective. Whether creating a new breakthrough pathway for devices (which already are approved based on much lower standards than drugs) or deregulating health IT software, for example, patients will be at risk. The Food and Drug Administration’s (FDA) primary mission is to protect the public health by “assuring the safety, efficacy and security” of drugs, biological products, and medical devices.[i] In your role as a Senator, it is critical that any legislation you support advances the FDA’s role in protecting public health.

The MEDTECH Act (S. 1101)

We strongly oppose this bill because it would remove potentially life-saving and life-threatening health IT software entirely from FDA’s regulatory oversight. If this bill becomes law, the FDA would no longer gather adverse event reports and it would not issue recalls for IT devices that have life-threatening flaws.

A new study by the National Center for Health Research found that over the last 5 years, more than 600 different software devices totaling over 1.4 million units were recalled for high-risk and moderate-risk patient safety issues. Almost 200,000 units were recalled for posing a life-threatening risk to patients. Although most of these recalls are officially referred to as voluntary on the part of device companies, experts agree that most recalls would not occur without FDA regulatory authority. That is because the FDA is responsible for compiling and making public the reports of adverse events, and determines the level of risk posed by the device and therefore how the recall will be implemented. The FDA also has the authority to remove a device from the market if it isn’t voluntarily recalled, which is sometimes necessary to save patients’ lives.

Advancing Breakthrough Devices for Patients Act (S. 1077)

We strongly oppose the Advancing Breakthrough Devices for Patients Act, because it sets a low bar to qualify for “breakthrough” status, micromanages the FDA, lowers standards for safety and effectiveness, and uses a promotional term that misleads the public. In order to qualify for breakthrough status, a device simply must be “in the best interest of patients.” How does that qualify as a breakthrough? Such a vague criterion will encourage many device companies to apply for “breakthrough” status, overwhelming the resources of the FDA. A recent study, published in JAMA, found that patients assumed that “breakthrough drugs” would greatly benefit them;” they were much less enthusiastic when they found out that breakthrough drugs were merely “promising” drugs that were approved based on preliminary, inconclusive data.[ii]

The bill undermines FDA’s professional judgment by telling the agency what type of statistics it should use (Bayesian), recommending the “adoption of shorter or smaller clinical trials,” stating who should be on device review teams, and requiring the Secretary to agree to a legally binding clinical trial plan in writing at the outset. This is particularly problematic because new safety information is often learned during the development process, requiring study modifications to avoid harming patients. Furthermore, smaller clinical trials inevitably have fewer women, people of color, and patients over 65 – often too few to ensure that the device is safe and effective for those groups – which comprise the majority of patients.

The bill would also push FDA to rely on post-market studies rather than ensuring safety or effectiveness before hospitals and patients pay for the devices. A 2015 GAO report found that most required post-market device studies are never completed. In other words, if this bill passes, there would be no conclusive evidence pre-market or post-market. Patients should not be used as unwitting guinea pigs, paying for the privilege of being harmed by untested medical devices.

The Advancing Hope Act (S. 1878)

We strongly oppose the Advancing Hope Act since its ability to stimulate innovation is questionable and it does not efficiently use FDA resources to benefit public health. The purported intent of the bill to encourage new treatments for rare pediatric diseases is admirable, but there is no evidence that the program will accomplish this goal. In fact, the priority review vouchers have so far been used for drugs already under review in the U.S. or already licensed in other countries.[iii] Recently, FDA officials stated that they have seen “no evidence that the program is effective.”[iv]

Another serious flaw in the program is that it does not allow the FDA to prioritize matters of the highest public health importance. According to FDA Office of New Drugs Director, John Jenkins, “These programs allow sponsors to ‘purchase’ a priority review and remove FDA’s ability to set its work priorities and resource allocations based on the public health merit of the project.”[v]

While the program has not resulted in new treatments for children, it has dramatically increased profits for pharmaceutical companies. The vouchers have been sold for prices ranging from $67.5 million to $350 million.4   Knight Therapeutics spent about $10 million moving a drug through the FDA’s priority voucher program and then sold the voucher for $125 million.

The Medical Countermeasure Innovation Act (S. 2055)

We strongly oppose the Medical Countermeasure Innovation Act. The bill establishes yet another priority review voucher program for medical countermeasures. We discussed the reasons why we oppose priority vouchers above. The bill needlessly burdens the FDA and adds bureaucratic red tape by requiring the Secretary of HHS to send an “excuse letter” to Congress each month it is not able to complete the final animal rule guidance, regardless of whether the FDA has sufficient information to do so. It also subjects HHS to pressure from congressmen who want their state’s company to receive contracts.

The Combination Product Regulatory Fairness Act (S. 1767)

We strongly oppose the Combination Product Regulatory Fairness Act because it requires the FDA to review more combination products as devices rather than as drugs. It does this by requiring that jurisdiction as drugs or biologics not be assigned “solely because” of chemical action in the human body. Devices are held to a much lower standard for approval than drugs, so this shortchanges patient safeguards. In addition, the bill requires the FDA and sponsor to develop a binding clinical trial plan early in the development process. This would restrict the FDA’s ability to later seek additional clinical data if it discovers new information related to safety and effectiveness. That is dangerous for patients.

Patient-Focused Impact Assessment Act of 2015 (S. 1597)

We oppose this bill as written, which adds a new layer of bureaucracy to the FDA. While we strongly believe that the FDA should consider patient perspectives, it is essential that those perspectives be independent of industry funding and pressure. Groups funded by industry are likely to champion the interests of industry. As written, the bill provides a mechanism for industry to promote drugs directly to consumers for off-label uses.

We were pleased to support the legislation that the HELP Committee passed last month, although disappointed that none of our suggested improvements were considered. We are very disappointed that these bills are so biased against the interests of our nonprofit member organizations, who represent millions of patients, consumers, scientists, and public health researchers and advocates. Our members depend on the FDA to assure that medical products are safe and effective, and these bills undermine that mission.


Breast Cancer Action

Center for Medical Consumers



Mothers Against Medical Error

MRSA Survivors Network

National Center for Health Research

National Consumers League

National Organization for Women

National Women’s Health Network

Our Bodies Ourselves

The TMJ Association

Union of Concerned Scientists, Center for Science and Democracy

Washington Advocates for Patient Safety


[i] Food and Drug Administration. FDA Mission from the agency’s Web site. Accessed March 3, 2016. http://www.fda.gov/downloads/aboutfda/reportsmanualsforms/reports/budgetreports/ucm298331.pdf

[ii] Krishnamurti T, Woloshin S, Schwartz LM, et al. A Randomized Trial Testing US Food and Drug Administration “Breakthrough” Language. JAMA Intern Med. 2015;175(11):1856-1858.

[iii] Branswell H (November 28, 2015). “How a system to help treat rare diseases broke down,” STAT. http://www.statnews.com/2015/11/28/priority-review-vouchers-rare-diseases/

[iv] U.S. Government Accountability Office (March 2, 2016). Too Early to Gauge Effectiveness of FDA’s Pediatric Voucher Program. http://www.gao.gov/products/GAO-16-319?utm_medium=email&utm_source=govdelivery

[v] McCaughan M (October 6, 2015). Priority Review Vouchers: FDA Has “Concerns,” Pharma & Medtech Business Intelligence.

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