Comments to FDA on how device makers can avoid sending “confusing or unclear information” to patients

August 9, 2016


Division of Dockets Management (HFA-305)

Food and Drug Administration

5630 Fishers Lane, Rm. 1061

Rockville, MD 20852.



Comments of members of the Patient, Consumer, and Public Health Coalition

on the draft guidance

Dissemination of Patient-Specific Information from Devices by Device Manufacturers

[Docket No. FDA-2016-D-1264]



Members of the Patient, Consumer, and Public Health Coalition appreciate the opportunity to comment on the draft guidance Dissemination of Patient-Specific Information from Devices by Device Manufacturers.  We generally support the draft guidance but the document needs further clarification, especially the paragraph on the Health Insurance Portability and Accountability Act (HIPAA).

The purpose of the draft guidance is to “clarify that manufacturers may share with a patient  patient-specific information” collected from devices regarding that same patient.  In other words, device companies may share with a patient the information that the device collects about him or her. The draft guidance defines “patient-specific information” as including “recorded patient data, device usages/output statistics, healthcare provider inputs, incidence of alarms, and/or records of device malfunctions or failures.”[1] We agree with FDA that providing the above information “will empower patients to be more engaged with their healthcare providers in making sound medical decisions.”1


FDA recommends that device makers take steps to avoid “disclosure of confusing or unclear information that could be misinterpreted” by patients. The draft guidance does not provide details on how device makers should accomplish this goal.  If the information from the device is summarized, key data could be omitted. Alternatively, if all data is released (e.g. via data dump), the information could be overwhelming and useless to the patient.

Information communicated to patients should be done in a manner that is easy for them to understand.  Only 12 percent of adults have proficient health literacy, according to the National Assessment of Adult Literacy.[2]  This indicates that many patients may not be able to understand information that is complicated or communicated using medical terminology. Patients benefit from interactive, simple to follow, and practical communications that are appropriate to the intellectual and social skills of the patient and the caregiver.[3]

FDA notes that device makers “may share patient-specific information…with patients at the patient’s request without obtaining additional premarket review before doing so.” FDA then cautions that additional information from devices shared with patients by the manufacturer could meet the definition of labeling and would be subject to FDA labeling regulations. Although FDA cites the labeling section of the Federal Food, Drug, and Cosmetic Act (section 201 (m)), the draft guidance does not provide an example of when information shared from a device would meet the labeling definition.

FDA states that often the patient-specific information is “accessible by the patient’s healthcare providers,” or patients may contact the manufacturer directly to obtain the information.  The advantage of receiving the information from a healthcare provider is that the information will more likely be interpreted and put in context, and the patient can ask follow-up questions.   The disadvantage is that the patient will have to pay for the appointment, and may not be able to access the information in a timely manner.

We agree with FDA that patient-specific information shared with patients should be “comprehensive and contemporary” and the information from a patient’s blood pressure device provides a good example. But again, we are concerned that “comprehensive” could become a useless “data dump.”


We agree with FDA that patient-specific information should include “relevant context” so that the information will not be misinterpreted, “thus leading to incorrect or invalid conclusions.” Invalid conclusions could lead to additional tests (i.e. over diagnosis), or false negatives, which could put the patient’s health at risk. We also agree with FDA that device makers who provide patient-specific information should include information “about whom to contact for follow-up information.”


The draft guidance dedicates one paragraph to HIPAA.  It notes that HIPAA protections apply to device makers to prevent the sharing of “individually identifiable health information” but the protections “are not intended to prevent a device manufacturer from sharing patient-specific information with the affected patient.”

A recent article criticized FDA’s definition of “Patient-specific information” because it “appears to be, at least in part, inconsistent with HIPAA’s definition” of Protected Health Information (PHI). The article also notes that “there are a number of instances where a device manufacturer may be a HIPAA-regulated entity.”[4] For example, if a medical device company has contracted with a covered entity (such as a doctor’s office or hospital) so that the device will transmit electronic protected health information directly to the provider, compliance with HIPAA requirements is mandated.[5] This type of scenario is not addressed in the draft guidance.

Others have noted that the guidance appears to offer an incorrect interpretation of HIPAA when it states that device manufacturers are prevented under HIPAA from sharing this information with covered entities, such as health plans and health-care providers that electronically transmit health data, without the patient’s consent.[6]  We agree  that HIPAA was never meant to prohibit patient data collected by devices from being shared with the patients’ own physicians. Clarification of these issues is needed.

Also, nothing is mentioned about encrypting sensitive personal health information, or the risk of data breaches.  The draft guidance should recommend steps device makers can take to mitigate the risk of data breaches, and to make sure the information is not compiled in any databases that are shared with health plans or healthcare providers.


We generally support this brief draft guidance but the HIPAA section needs clarity, and Content section needs more details on how device makers can avoid disseminating “confusing or unclear information” to patients.

 American Medical Women’s Association

Breast Cancer Action

Connecticut Center for Patient Safety

MRSA Survivors Network

National Center for Health Research

National Consumers League

Our Bodies Ourselves

The TMJ Association

Washington Advocates for Patient Safety


The Patient, Consumer, and Public Health Coalition can be reached through Paul Brown at (202) 223-4000 or


[1] Food and Drug Administration (June 10, 2016).  Dissemination of Patient-Specific Information from Devices by Device Manufacturers; Draft Guidance of Industry and Food and Drug Administration Staff.

[2] Quick Guide to Health Literacy. US Department of Health and Human Services.

[3] Schooley B, San Nicolas-Rocca T, Burkhard R. Patient-provider communications in outpatient clinic settings: a clinic-based evaluation of mobile device and multimedia mediated communications for patient education. JMIR Mhealth Uhealth. 2015 Jan 12;3(1):e2.

[4] Weinrieb JM, Weeda JM (June 15, 2016). FDA Publishes Draft Guidance on Dissemination of Patient-Specific Data—But Doesn’t Say Much About HIPAA.  OFW Law.

[5] Hartford, J (August 25, 2015). Are your medical devices HIPAA compliant? MDDI DeviceTalk.

[6] Williamson MD (June 13, 2016). FDA Guidance on Device Data Sharing Aligns With HIPAA: Attorney. Bloomberg BNA.


Coalition urges FDA to protect patients & consumers by requiring biosimilar companies to update their labels when safety issues arise

August 2, 2016


Division of Dockets Management (HFA-305)

Food and Drug Administration

5630 Fishers Lane, Rm. 1061

Rockville, MD 20852



Comments of members of the Patient, Consumer, and Public Health Coalition


Labeling for Biosimilar Products; Draft Guidance



Members of the Patient, Consumer, and Public Health Coalition support the Food and Drug Administration’s (FDA) draft guidance on “Labeling for Biosimilar Products.”  The draft guidance will protect patients and consumers by requiring biosimilar companies to update their labels when safety issues arise.


The Biologics Price Competition and Innovation Act of 2009, signed into law as part of the Affordable Care Act, created an abbreviated pathway to license biosimilar products.[i] A product is a biosimilar if it has no clinically meaningful differences compared to the reference product in terms of “safety, purity, and potency.”1 Because there is no meaningful difference, we agree with the FDA that biosimilars labeling should include a description of the clinical data that supported safety and efficacy of the reference product.  We agree with FDA that the biosimilar prescribing information (package insert) relies mainly on the safety and effectiveness information from the labeling for the reference product.[ii] 

Specific Recommendations on Content of Biosimilar Product Labeling

FDA also notes that biosimilar products’ labels may differ from the reference product labeling (have “appropriate product-specific modifications”) in order to conform to the Physician Labeling Rule (PLR) and the Pregnancy and Lactation Labeling Rule (PLLR), and other safety issues.  We agree since this will add to the safer use of biosimilars.  Also, according to a recent survey of European physicians, they “prefer more product-specific information in the biosimilar label.”[iii]

 Revising Biosimilar Product Labeling

We strongly agree with the FDA that “all holders of marketing applications for biological products have an ongoing obligation to ensure their labeling is accurate and up to date” (emphasis added).1  We see no reason why this should not include PLR and PLLR information for the reference product. To ensure that the product is used safely, both the reference product and the biosimilar product application holders must be able to update their labeling.


We strongly support the “Labeling for Biosimilar Products” draft guidance.  It will protect patients and consumers by ensuring the important labeling safety information is updated for both biosimilars and the reference products.

American Medical Student Association

American Medical Women’s Association

Center for Medical Consumers

Connecticut Center for Patient Safety


MRSA Survivors Network

National Center for Health Research

National Physicians Alliance

National Women’s Health Network

Quinolone Vigilance Foundation

Washington Advocates for Patient Safety



 The Patient, Consumer, and Public Health Coalition can be reached through Paul Brown at (202) 223-4000 or at


[i] Federal Register (April 4, 2016). Food and Drug Administration draft guidance “Labeling for Biosimilar Products.”

[ii] Food and Drug Administration (April 1, 2016).  News & Events Form our perspective: Biosimilar product labeling. 2/3

[iii] Hallersten AFürst WMezzasalma R (June 2016). Physicians prefer greater detail in the biosimilar label (SmPC) – Results of a survey across seven European countries.  Regul Toxicol Pharmacol.

Coalition Members Strongly Support FDA’s Proposed Ban on Inhumane Electrical Stimulation Devices

July 25, 2016


Division of Dockets Management (HFA-305)

Food and Drug Administration

5630 Fishers Lane, Rm. 1061

Rockville, MD 20852.



Comments of members of the Patient, Consumer, and Public Health Coalition on

Proposed Rule To Ban Electrical Stimulation Devices Used To

Treat Self-Injurious or Aggressive Behavior

[Docket No. FDA-2016-N-1111]


Members of the Patient, Consumer, and Public Health Coalition strongly support the FDA’s proposed rule to ban electrical stimulation devices (ESDs)* used to treat self-injurious behavior (SIB) or aggressive behavior (AB). ESD treatments are ineffective and have serious side effects, and there are alternative, less stressful treatments available.

The goal of ESDs is to reduce self-injurious or aggressive behavior by punishing it with shocks.  However, the strategy of preventing violent behavior in any situation by using a painful punishment is rejected by most experts.  That is why the overwhelming majority of SIB and AB patients are not treated with ESDs.  Only one facility in the U.S. manufactures and uses ESDs, the Judge Rotenberg Educational Center, Inc. (JRC). Even if ESDs were a safe, effective, and humane treatment, those used by JRC as recently as 2012 had not been cleared by the FDA. FDA has sent warning letters to the Center at least three times stating that the devices are in violation of FDA regulations because they have been modified to increase the voltage they provide but a new application has not been filed with the FDA.[i], [ii]

Because of advances in human rights and behavioral therapy, nearly half of the States prohibit the use of ESDs.[iii] The United Nations has said use of ESDs, such as those used by the Rotenberg Center, constitutes a violation of the UN Convention Against Torture, and would not be legal if used even against convicted terrorists.[iv], [v]


ESDs have a long list of harms including “depression, PTSD, anxiety, fear, substitution of other negative behaviors, worsening of underlying symptoms, and learned helplessness, as well as the physical risks of pain, and skin burns.”3 The devices are associated with an increased risk of “suicidality, chronic stress, neuropathy, and injuries from falling.”3  They are associated with “nightmares, flashbacks of panic and rage, hypervigilance, insensitivity to fatigue or pain, changes in sleep patterns, loss of interest, difficulty concentrating, and withdrawal from usual activity.”3

We agree with the FDA that adverse events are likely to be under-reported because the children and adults subjected to ESD often have problems that make it difficult to persuasively communicate to anyone who would be likely to report adverse events to the FDA.   In addition, the individuals administering ESDs are unlikely to be taught or encouraged by their employer (who requires them to administer ESDs) to report adverse events resulting from ESD “treatment.”


Although the scientific literature reports that ESD shocks can immediately interrupt SIB or AB, these studies have been criticized for “weak study design, failure to control for concomitant treatments, small size, lack of peer review, and conflicts of interest.”3  We agree with the FDA that “the evidence is inadequate to establish that ESDs improve individuals’ underlying conditions or…reduce or cease the target behavior to achieve durable long-term reduction of the target behavior.” The established risks are even more unacceptable given the lack of evidence of benefits.

Alternative treatments

There is clear research evidence that positive reinforcement is more effective and longer-lasting than negative reinforcement (punishment).  We agree with the FDA that positive-based behavioral approaches are effective at reducing self-injurious and aggressive behavior, providing greater benefits and less risk than ESDs.  We also agree with the FDA that “addressing the underlying causes of SIB and AB…rather than suppressing behaviors with shocks not only avoids the risks posed by ESDs, but can achieve durable, long-term benefits.”


ESDs are not established to be effective for most self-injurious or aggressive behaviors and represent a substantial and unreasonable risk of injury.  They are also inhumane. For the above reasons, we strongly support the ban on electrical stimulation devices.

American Medical Women’s Association


MRSA Survivors Network

National Center for Health Research

National Physicians Alliance

Washington Advocates for Patient Safety



The Patient, Consumer, and Public Health Coalition can be reached through Paul Brown at (202) 223-4000 or

*We are aware that ESDs are not used in electroconvulsive therapy (ECT or electroshock therapy) and that this proposed rule does not apply to ECT devices.


[i] Willingham E (April 22, 2016).  FDA Proposes Ban On Electric Shock Devices Used On Autistic Children. Forbes.

[ii] Food and Drug Administration (December 6, 2012).  Inspections, Compliance, Enforcement, and Criminal Investigations: Warning Letter CMS#367480, the Judge Rotenberg Educational Center.

[iii] Federal Register (April 25, 2016). Food and Drug Administration: Banned Devices; Proposal To Ban Electrical Stimulation Devices Used To Treat Self-Injurious or Aggressive Behavior; Proposed Rule. [Docket No. FDA-2016-N-1111]

[iv] Ahem, L (October 2, 2010). Disabled children at Mass. School are tortured, not treated. The Washington Post.

[v] Burkholder A (August 5, 2014). Controversy over shocking people with autism, behavioral disorders. CBS News.

Coalition Urges FDA to Improve Draft Guidance for Using Electronic Health Records in Clinical Studies

July 18, 2016

Food and Drug Administration

Division of Dockets Management (HFA-305)

5630 Fishers Lane, Rm. 1061

Rockville, MD 20852


Comments of members of the Patient, Consumer, and Public Health Coalition

on the draft guidance

Use of Electronic Health Record Data in Clinical Investigations

[Docket No. FDA-2016-D-1224]


A major goal of the draft guidance is to “facilitate the use of EHR data in clinical investigations.”1  Although EHRs are not under the direct control of the FDA (the systems belong to healthcare organizations and institutions that use them), the Agency does need to develop a clear process for accepting EHR data for use in clinical investigations.  The draft guidance is a step in the right direction but needs to better address key issues.

The draft guidance addresses how the FDA will ensure the EHR data’s validity, reliability, and integrity, and how the agency will verify the integrity of the EHR data during on-site inspections and audits of the organizations that control the data.

On-site inspections

We agree with the draft guidance that clinical investigators must retain all paper and electronic source documents for as long as required by regulations, and EHR data must meet FDA’s inspection, record keeping and record retention requirements.  And, importantly, study monitors must have “suitable access to all relevant subject information pertaining to a clinical investigation.” 2

However, we are concerned that the draft guidance does not mention how often FDA will do on-site inspections.  If FDA does not establish a schedule of on-site inspections, they may never be done and this could lead to poor quality data.

Quality and integrity

The draft guidance states that “when EHRs are used as a source of data in clinical investigations, sponsors should ensure that the data are ‘attributable, legible, contemporaneous, original and accurate (ALCOA).’”  If the data does not meet all five of these attributes, will the data be rejected by the FDA?

An inherent problem with using EHRs for clinical investigations is that EHRs are currently designed to assist in delivering health care — not to generate medical evidence.  A July 2016 Article in JAMA Internal Medicine notes that “practices like blinding, randomization, or standardized-event recording are more difficult to implement in systems that are oriented toward care.  Health care systems represent inefficient environments to which to learn about differential effects of novel drugs.”3 The draft guidance notes that the use of EHRs for clinical investigations may require additional “considerations, planning, and management” but it does not go into detail on how to accomplish this.

The draft guidance recommends, but does not require, the use of certified EHR technology.  The U.S. Department of Health and Human Services’ Office of the National Coordinator for Health Information Technology (ONC) has a voluntary certification program for health IT.  EHR technology certified by ONC’S Program meets privacy and security protection requirements.

The draft guidance allows EHRs not certified by ONC to be used for clinical investigations as long as they include adequate controls such as limiting access to the electronic systems, identifying the authors of records, providing audit trails, ensuring records are retained for FDA inspections, and assuring patient privacy rights.  Although the adequate controls address key issues, there would be more consistency in investigations, if all EHRs for clinical investigations were required to use certified EHR technology.

In using EHRs for clinical investigations, the draft guidance recommends that extracted data is checked for consistency and completeness.  It also recommends that corrections be made when errors are found “to properly align the source data with the extracted data.”  We are concerned that unless FDA spot checks the data, errors will not be corrected.

The draft guidance notes that software updates could “affect the reliability and integrity of EHR data entering the sponsor’s electronic system.”  This is a major concern.  A recent review of FDA data 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 moderate or high risk patient safety issues.  For example, oncology electronic medical record systems were recalled because they erroneously switched patient data and failed to warn physicians about dangerous drug reactions.

Interoperability & audit trails

The draft guidance encourages the use of interoperable systems in order to “reduce errors in data transcription and provide data that is more accurate.”  We agree that interoperability will reduce transcription errors but if the data was incorrect to begin with, it will not provide more accurate data.

The draft guidance states that adequate methods of audit trails are needed to monitor, track and document all changes made to information in the EHR regarding the clinical investigation. However, the draft guidance fails to define the phrase “adequate methods.”

One of the major risks associated with EHRs are data breaches.  Data breaches could negatively affect a subject’s employment or ability to get insurance.  The draft guidance states, “Sponsors should consider whether there are any reasonably foreseeable risks with the use of EHRs…that must be described to the subject in the informed consent.”  We recommend that the risk of data breaches be communicated to subjects at least twice—once in writing and once again verbally.


EHRs can provide investigators “access to real-time and longitudinal health care data”1and can provide post-trial follow-up information on safety and efficacy of medical products.  However, we think this draft guidance still needs work.  It does not address how inaccuracies in the data will be corrected, how often FDA will do on-site inspections, and ways to mitigate the risks of data breaches.

American Medical Women’s Association

MRSA Survivors Network

National Center for Health Research

Our Bodies Ourselves

Quinolone Vigilance Foundation

Washington Advocates for Patient Safety


The Patient, Consumer, and Public Health Coalition can be reached through Paul Brown at (202) 223-4000 or

Congress Shouldn’t Pass The 21st Century Cures Act In A Summer Rush

By Paul Brown, Tracy Rupp, and Steven Findlay, Health Affairs

July 11, 2016

The full Senate may in the next few days consider companion legislation to the 21st Century Cures Act that passed the House last year. The legislation—currently 19 separate bills—makes substantial changes to the way the Food and Drug Administration (FDA) approves drugs and devices. Set to adjourn for an extended election-year summer recess on July 15, the clock is ticking. The congressional calendar in the fall is full and the Senate may simply not have the time to take up the complex legislation, and reconcile it with the House version, before the November elections.

We believe that’s a good thing. The legislation, while containing some useful measures, is flawed. Instead of hastily agreeing to it, Congress should postpone consideration until 2017 and attach the best of the 19 bills (see below) to must-pass legislation on FDA funding through industry user fees.

At that time, we believe the Senate should reject those bills that lower standards for drug and device approvals and safety, and risk adding to the rising cost of prescription drugs.

This is a story two years in the making. Proponents of the proposed legislation—drug and device companies, and members of both parties in the House and Senate—argue that the FDA stifles innovation and advances in treatment by approving drugs and devices too slowly compared to other countries.

That premise is faulty. Nearly two-thirds of the novel drugs approved in 2015, for example—29 of 45, 64 percent — were approved in the United States before being approved in any other country. The proportion was even higher in 2012 and 2013. The majority of these drugs (60 percent) took advantage of existing FDA expedited review programs—fast track, breakthrough, priority review, and accelerated approval—and nearly half (47 percent) were approved to treat rare or orphan diseases.

As for devices, research shows that “it takes the same amount of time or less for patients to gain access to innovative, high-risk medical devices” in the U.S. as compared to Germany, France, Italy, and Britain.

The House and Senate bills ignore the above facts. They essentially seek to speed-up the approval process by relaxing FDA’s safety and effectiveness standards. And to make that more palatable, sponsors have attached the changes to increases in funding for the National Institutes of Health and the FDA.

But while the public supports increases in biomedical research funding, it is deeply skeptical about lowering the standards for drug and device approval. In the most recent poll on this issue, just under 60 percent of Americans opposed changing federal regulations to speed the development and approval of drugs; 38 percent favored speedier FDA action, according to a STAT-Harvard poll released in May. Respondents’ main concern: faster approval would allow products on the market that don’t work or are unsafe.

The survey echoed a previous poll by Consumers Union that found 82 percent of Americans believe that preventing safety problems is more important than limiting safety testing to speed the clearance or approval of devices or promote innovation.

The 21st Century Cures Act received broad bipartisan support in the House in part because it put a significant amount of money for research on the table. The House bill pledges a $9 billion increase in mandatory funding for the National Institutes of Health over five years, gaining the support of universities and medical schools. It also promises $550 million to the FDA (which, according to the CBO’s analysis, would not fully pay for the additional workload the Act assigns the FDA).

The drug and device industries intensely lobbied House members to pass the legislation. The Pharmaceutical Research and Manufacturing Association (PhRMA) increased its lobbying from $4 million to $5.4 million in the quarter before the 21st Century Cures Act passed. The Advanced Medical Technology Association upped its spending from $550,000 to $740,000 in the same quarter. The Senate’s lobbying database listed more than 1,100 lobbyists working on the legislation. Many patient groups have also voiced their support for the legislation, based on their belief that the bill would result in more treatment options. An analysis of public comments for the House bill completed by Avalere noted that 43 percent were from patient advocacy groups.

The Senate bills are an improvement over the House’s 21st Century Cures Act. But, on balance, they are still weighted heavily in favor of speeding medical products to market by weakening FDA approval standards.

New drugs and devices can, of course, be an improvement over existing products. And when they clearly are, FDA has established pathways to get them to market and patients as fast as possible. The agency, for example, grants more than one-third of requests from industry for “breakthrough” designation for new drugs. But the history of medicine is replete with examples of drugs and devices that caused more harm than good, some of which were approved too hastily — such as Avastin for breast cancer, Vioxx for arthritis, metal-on-metal hip implants, and power morcellators. Innovative drugs and devices simply must be required to actually work and not harm patients. Methods of testing drugs and devices in patients are improving all the time, with new tools that help detect products that warrant further testing or could be dangerous. The default mode of this technical process must always be “safety first.”

Below are our concerns with some of the Senate bills followed by a list of bills we believe would improve public health.

Senate bills that would, in our opinion, increase risks to patients:

  • The MEDTECH Act would prevent the FDA from collecting adverse events due to flawed electronic medical records, and from recalling certain types of defective medical software. Some of this software has had life-threatening flaws in the past, such as oncology electronic medical record systems that calculated and recorded incorrect drug dosages for highly toxic chemotherapy drugs.
  • The PATH Act would allow antibiotics to be approved with minimal evidence of safety and effectiveness through a “limited population” approval pathway. But, antibiotics approved in this way are promoted by companies so that they are more widely prescribed in order to increase sales. As they are, we won’t have information about whether they’re actually safe or effective for those groups of patients. For example, fluoroquinolones are today widely overprescribed for urinary tract infections despite guidelines recommending other antibiotics and data linking their overuse to the development of resistance.
  • The Advancing Breakthrough Devices for Patients Act would encourage shorter and smaller clinical trials for medical devices. Abbreviated clinical trials will make it difficult, if not impossible, to include sufficient participation from subpopulations such as women, seniors, and racial and ethnic minorities in the analysis of the trials. For example, a recent study of high-risk medical devices found that the median number of participants was only 65 patients. With so few patients, it’s difficult to draw reliable conclusions about safety and effectiveness, especially for subgroups of patients. In an increasingly diverse America, this is unacceptable. The bill also pushes FDA to rely on post-market studies for devices rather than ensuring safety effectiveness before hospitals and patients pay for the products.
  • The Advancing Hope Act would continue the existing pediatric priority review voucher program through 2022. The program is currently set to expire at the end of September. The program’s ability to stimulate innovation is questionable: a recent GAO review of the program concluded that the six drugs for which vouchers have been awarded so far were in development before the program existed. By allowing drug makers to buy a priority review, the bill removes FDA’s ability to set its work priorities and resource allocations based on public health needs. In a time of threats such as the Zika virus, our government agencies must be able to prioritize public health, and not be bound by vouchers that were sold to the highest corporate bidder.

Senate bills likely to promote innovation and protect public health:

  • The Preventing Superbugs and Protecting Patients Act will help prevent drug-resistant infections from contaminated duodenoscopes and other reusable medical devices that have caused harm and deaths. If enacted, this bill will require certain reusable medical devices to have validated cleaning, disinfection, and sterilization procedures prior to marketing. This will reduce the likelihood that these devices will transmit dangerous infections from patient to patient.
  • The Advancing Research for Neurological Diseases Act will help people suffering from neurological diseases by providing critical information to researchers as they work on new treatments and cures. The bill requires the Department of Health and Human Services to collect neurological disease surveillance data, such as demographic information, risk factors, outcomes, and treatments. Providing such information is likely to enhance understanding of these diseases and the development of new treatments.
  • The Next Generation Researchers Act recognizes the importance of investing in the brightest young researchers to ensure that the United States remains at the forefront of biomedical research.
  • The FDA and NIH Workforce Authorities Modernization Act will make it easier for FDA to recruit and retain top scientific and technical experts by making salaries more competitive with those offered by industry. It will also lead to the development of standards for regenerative medicine — such as replacing, engineering, or regenerating human cells, tissues, or organs to restore or establish normal function.

Lawmakers are also considering adding the REGROW Act to the Senate’s package of bills. The bill would allow complex regenerative medicine therapies to be conditionally approved based on preliminary evidence. Since at least half of all drugs fail in the last stage of testing, many patients could end up receiving therapies that are later found to be unsafe or ineffective.

Looking Ahead

On Saturday June 25, six former FDA commissioners from Democratic and Republican administrations suggested at the Aspen Ideas Festival that Congress make the agency independent of the Department of Health and Human Services — similar to the Securities Exchange Commission, for example. With regulatory purview over products that represent a quarter of the U.S. economy, the group said the FDA is harmed by an unstable federal budget process and persistent political meddling. The group said they would issue a white paper on their proposal for the next administration. That’s another reason why Congress should postpone consideration of these bills until 2017.

To see original article, click here.

Coalition Letter to Senators regarding the Senate’s Biomedical Bills (21st Century Cures companion legislation)

July 5, 2016

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 Senate’s biomedical bills (21st Century Cures companion legislation).

We want all Americans to have the best possible medical treatments but we are concerned that the primary 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 expedited 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.

We are very concerned about unintended consequences of expedited approvals. If drugs are approved based on preliminary evidence and then later found to be ineffective or unsafe, potentially billions of dollars could be wasted by Medicare (taxpayers) and private insurers. Consumers are worried about skyrocketing drug prices. A Bloomberg analysis found that 30 of 39 drugs they reviewed had price increases more than double the rate of inflation from 2009 to 2015, even after discounts were factored in.[1]Innovative new drugs and devices will do nothing to improve health or save lives if they don’t work or if most Americans can’t afford them.

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.[2] As a Senator, it is critical that any legislation you support advances the FDA’s role in protecting public health. Below are bills that we urge you to oppose followed by bills we would like you to support.

We urge you to oppose:

The MEDTECH Act (S. 1101)

The MEDTECH Act removes health IT software from FDA’s regulatory oversight. If this bill becomes law, the FDA would no longer gather information about life-threatening flaws in medical software and no longer have the authority to recall those deadly devices. Dangerous flaws are commonly caused by glitches in software design but can also result if the software is easily hacked. A National Center for Health Research study found that if medical software is removed from FDA regulatory oversight, millions of patients would be at risk from defective software. For example, oncology electronic medical record systems were recalled in the past because they calculated and recorded incorrect drug dosages. Clinical decision support systems used during surgery were recalled because they erroneously switched patient data and failed to warn physicians about dangerous drug reactions.

Advancing Breakthrough Devices for Patients Act (S. 1077)

The Advancing Breakthrough Devices for Patients Act lowers standards for safety and effectiveness for devices. Only 3% of all medical devices are studied in clinical trials, and most are already small. This bill would encourage even smaller clinical trials, which would inevitably have fewer women, people of color, and patients over age 65 (often too few to ensure that the device is safe and effective for those groups). The bill would also push FDA to rely on post-market studies rather than ensuring safety and effectiveness before hospitals and patients pay for the devices. Remember that these are already the riskiest 3% of medical devices! As a result, public and private insurers will spend billions on medical devices that might later be found to be unsafe or ineffective.

The PATH Act (S. 185)

While the amended version that was voted out of the HELP Committee was greatly improved over the 21st Century Cures version, we do not support the concept of a limited population approval pathway for antibiotics. Antibiotics approved via a pathway such as this will have minimal evidence of safety and effectiveness but will inevitably become widely prescribed to more general populations with less severe infections. As a result, resistance will develop and a potentially promising drug will become ineffective.

The Advancing Hope Act (S. 1878)

The ability of the Advancing Hope Act to stimulate innovation through the issuance of priority review vouchers is questionable and it does not efficiently use FDA resources to benefit public health. The priority review vouchers have so far been used for drugs already under development in the U.S. or already licensed in other countries.[3]Furthermore, by allowing sponsors to buy a priority review, the bill removes FDA’s ability to set its work priorities and resource allocations based on the public health merit of the project.[4] In a time of dangerous threats like Zika and Ebola virus, we expect our government agencies to be able to prioritize public health, not to be bound by priority review vouchers that were sold to the highest corporate bidder.

The Combination Product Regulatory Fairness Act (S. 1767)

We strongly oppose the Combination Product Regulatory Fairness Act because it would result in more combination products being classified as devices and thus reviewed with less scientific evidence when it would be better for patients if the FDA used the more rigorous drug review process. This bill also restricts the FDA’s ability to later seek additional clinical data if it discovers new information related to safety and effectiveness. This is dangerous for patients.

The REGROW Act (S. 2689)

While not included in the bills voted out of the Senate HELP Committee, the REGROW Act has been mentioned as a priority by the Republican leadership for inclusion in the Senate biomedical bills. This bill would allow complex biologic therapies to be conditionally approved based on very preliminary evidence and then prescribed to patients without proof that the new therapy actually works or is safe. Notably, the Alliance for Regenerative Medicine (ARM), which includes many of the best regenerative medicine researchers, does NOT support the REGROW Act. If passed, this Act could lead to CMS wasting billions of dollars on medications that were approved by the FDA but do more harm than good. Although strongly criticized by researchers and ethicists, this bill has support from a billionaire who has donated $2 million to a Senate super-PAC.

We urge you to support:

The Preventing Superbugs and Protecting Patients Act (S. 2503)

Drug-resistant infections from contaminated duodenoscopes and other reusable devices have hurt and killed many people throughout the United States. This legislation and the safety recommendations in the recent HELP minority staff report will help prevent this from happening in the future. We strongly urge you to support this legislation that will reduce and eliminate these deadly infections.

The Advancing Research for Neurological Diseases Act (S. 849)

The bill would provide a foundation for the Department of Health and Human Services (HHS) to learn more about the many factors involved in neurological diseases, such as geographic clusters of diagnoses, variances in the gender ratio, disease burden, and changes in health care practices. This could truly be a game-changer for people suffering from neurological diseases and provide critical information to researchers as they work on new treatments and cures.

As you consider combining only the best of these bills into a comprehensive legislative package, it is essential to provide additional mandatory funding for the NIH and FDA so that there are no unfunded mandates in the legislation and there is sufficient support to develop and monitor safe and effective medical treatments.  We particularly urge substantial new funding for the NIH to develop new antibiotics targeted to resistant bacteria, as well as to FDA and CDC to ensure stewardship of antibiotics already on the market. We must ensure that these institutions remain at the forefront of science and medicine for years to come and that patients can trust their drugs and medical devices to improve their health rather than harm it.



Breast Cancer Action

Mothers Against Medical Error

MRSA Survivors Network

National Center for Health Research

National Consumers League

National Women’s Health Network

Quinolone Vigilance Foundation

The TMJ Association

Washington Advocates for Patient Safety

Woody Matters


[1] Langreth R, Keller M, Cannon C (June 29, 2016). Decoding Big Pharma’s Secret Drug Pricing Practices. Bloomberg. Available at:

[2] Food and Drug Administration. FDA Mission from the agency’s Web site. Accessed March 3,2016.

[3] Branswell H (November 28, 2015). How a system to help treat rare diseases broke down,STAT.

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

Faster Drug Approval: Winners and Losers

By Kristen Fischer, HealthZette

July 1, 2016

[…] Recently, due to calls for novel treatments that move faster from the (lab) bench to the bedside, a legislative effort is attempting to speed up that process. The 21st Century Cures Act would allow companies to submit evidence of safety based on patient stories instead of actual clinical trials.

The House already passed its own version; the Senate is expected to review it in the coming weeks, STAT reported.

While the measure could speed up the process, some people fear it would also remove a lot of quality control. And when it comes to health, it’s good to know that the medications you take have been tested extensively and are backed by reliable data.

Another concern is any new fast-track process would enable pharmaceutical companies, for the most part, to dictate the efficacy of a drug instead of the FDA as a third party.

Patience for Patients

[…] Dr. David Gortler, a former FDA senior medical officer and current drug safety and FDA policy expert at, told LifeZette he doesn’t agree with speeding up the process because lawmakers typically don’t have an extensive pharmacology background. Circumventing the time-tested FDA process will not help Americans.

“They [lawmakers trying to speed up the process] don’t trust these people who have dedicated their lives to pharmacology and medicine,” Gortler said.

People think they know better, but they are really just putting their lives at risk, he added. “They don’t trust doctors or pharmacists. They trust GNC.”

In a Washington Post editorial, Susan F. Wood, an associate professor at George Washington University’s Milken Institute School of Public Health and a former FDA assistant commissioner, explained that the 21st Century Cures Act is based on an assumption that there will be more cures if drugs and devices are studied more quickly on fewer patients.

She, along with Diana Zuckerman, president of the National Center for Health Research, believes the studies would be too small to allow safety and effectiveness findings to be broken down into subgroups such as male and female.

“This embrace of smaller, more preliminary studies could drastically lower scientific standards. When fewer people are studied, it is more likely that a drug will seem safe and effective even if it has dangerous side effects for many patients — who may not have been included in those small studies,” they wrote.


To see original article, click here.

Republicans seize on reports critiquing FDA to push for agency reforms

Sheila Kaplan, STAT News

June 15, 2016

Has Congress’s watchdog agency just given Tennessee Senator Lamar Alexander the ammunition he needs to push a stalled biomedical reform through the Senate?

The Government Accountability Office on Wednesday issued two critical reports suggesting that the Food and Drug Administration isn’t properly planning how to keep pace with medical science.

One report notes that the FDA lacks goals, targets, and time frames for keeping up with scientific advances — and also fails to track the money it spends on these efforts. The other finds fault with the FDA’s strategic plan, which is supposed to speed approvals of drugs and devices, especially combination medical products, such as heart stents that also deliver blood thinners to prevent clots.

The reports land just as time is winding down for the Senate to pass its version of the 21st Century Cures Act, which the House has already overwhelmingly approved. The bill aims to get drugs and devices to market more quickly.


Some Democrats say it weakens FDA standards on drug and device approval too much. Critics also want more funding for both the FDA and the National Institutes of Health. To try to move things along, Alexander chopped the legislation into a series of smaller bills, but funding remains a sticking point.

A few weeks ago, Alexander took to the floor to urge action. “There is no excuse whatever for us not to get a result this year. And it would be extraordinarily disappointing to millions of Americans if we did not,” he said.

It’s not clear whether the GAO’s concerns about the FDA would be addressed by the 21st Century Cures bill, or by the Senate’s package of biomedical bills.

Neither the HELP committee Democrats nor the FDA were available for immediate comment.

Diana Zuckerman, a critic of the bills and the president of the National Center for Health Research, said the FDA is already focused too much on speeding up drug and device approvals and is not paying enough attention to “doing its job to protect the health and safety of patients and consumers.”

To see original article, click here.

Coalition letter urging Senators to oppose S. 2912, the Trickett Wendler Right to Try Act of 2016

June 14, 2016

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 S. 2912, the Trickett Wendler Right to Try Act of 2016. We strongly urge you to NOT co-sponsor this bill. This bill is bad for patients, bad for doctors, bad for drug development, and bad for science.

As you may know, the history of the FDA is a history of Congress responding to medical tragedies by requiring FDA to do more to protect patients from harmful medical products. Thalidomide birth defects and deaths and infertility from the Dalkon Shield IUD are just two of many examples. Although the AIDS crisis resulted in a more flexible approach for deadly diseases for which there were no treatments, today’s HIV/AIDS advocates are among the FDA’s strongest supporters, because the scientific evidence that FDA required of pharmaceutical companies resulted in effective treatments and prevention strategies. The FDA’s essential role continues to be to achieve a balance between patients’ rights and societal good. It is critical that, in your role as a Senator, you support the agency in upholding this delicate balance.

The idea behind this bill and other Right to Try laws is to give terminally ill patients access to experimental drugs that have passed phase I testing – testing that is often done on healthy volunteers, not people with serious illnesses. While this type of legislation promises cures, it offers false hope. Since most phase 1 trials include less than 30 patients and are designed only to identify the most immediate side effects, they provide very little information about the drug’s safety and effectiveness. In fact, most drugs with promising phase 1 test results are later found to be too risky or ineffective for patients to use. This is especially true for cancer drugs. Under the proposed bill, desperate patients are likely to be exploited by unethical companies that want to sell their unproven treatments at prices that would bankrupt many families. Health insurance companies will not pay for these unproven treatments – if they did, insurance premiums would skyrocket as patients harmed by unproven treatments required more expensive remedial care.

In the recent past, access to experimental bone marrow transplants for breast cancer was expedited due to an early benefit seen in preliminary data. It was not until many women were harmed and some even died that researchers realized a terrible mistake had been made. If doctors and patients can be fooled into trying such a painful and toxic treatment, imagine how often patients would be fooled by treatments that are less painful but ineffective and expensive snake oil.

This legislation would subject patients to a great risk of harm and they would have no legal recourse if things go wrong. In fact, this bill and similar state Right to Try laws reflect the libertarian philosophy of the Goldwater Institute that created them: the patient is on his or her own without any protections or safeguards. The bill explicitly states that drug and device companies as well as physicians cannot be held liable for any tragic outcomes, even if the company or a physician misled the patient. Even worse, if a patient suffers a terrible medical complication from a Right to Try treatment, the insurance company would not need to pay for the medical care that is needed to treat that complication. The only thing worse than a terminal illness is being terminally ill and suffering a major complication as the guinea pig in an experimental treatment that you had to pay for.

There are many other problems with the bill, but perhaps the most important thing for you to know is that the FDA’s current compassionate use program already provides patients with the opportunity to try experimental drugs, while offering some protections to patients and society. For example, while physicians and drug companies are required to report the outcomes of experimental treatments obtained through the FDA’s compassionate use program, Right to Try laws do not carry such stipulations. Indeed, this bill strips all protections from patients who would use it. Bypassing the oversight of the FDA is not in the best interest of patients or the public health.

The one shortcoming of the FDA’s current compassionate use (“expanded access”) program is also in the Right to Try bill: the company is not required to provide the experimental drug to the desperate patient. In some cases, that’s because the company doesn’t have enough doses to provide to patients not in a clinical trial, and in other cases, the company believes that the patient is more likely to be harmed by the product than helped by it.

In conclusion, Right to Try laws such as S. 2912 will do more harm than good for patients and society. The bill is opposed by experts in the field as well as by legitimate drug companies. We urge you to oppose the Trickett Wendler Right to Try Act of 2016.


American Medical Women’s Association

Connecticut Center for Patient Safety

DES Action

Jacobs Institute of Women’s Health

MedShadow Foundation

National Center for Health Research

National Physicians Alliance



The Patient, Consumer, and Public Health Coalition can be reached through Tracy Rupp at 202-223-4000 or


Coalition’s comments to the FDA on User Fees for Over-the-Counter Drugs

June 10, 2016

Comments of Members of the Patient, Consumer, and Public Health Coalition at the Public Meeting on OTC Monograph User Fees

Thank you for the opportunity to speak today.  My name is Paul Brown and I am speaking on behalf of 10 members of the Patient, Consumer, and Public Health Coalition.  Our informal coalition of nonprofit organizations represents the interests of patients, consumers, health-care professionals, scientists, and public health experts.

We support providing FDA with additional resources through the implementation of OTC user fees, so the agency can fulfill its mission of protecting the public from dangerous and ineffective OTC products.  Additional funding will help FDA to finalize the remaining OTC monographs, and allow patients and consumers to have confidence in the OTC products they use every day.

Since the OTC drug review process was established in 1972, FDA has struggled to complete the safety and efficacy evaluations of ingredients used in OTC drug products.  More than 40 years later, the monograph process still has not been completed for all ingredients and conditions of use.

As a result, many OTC products without a general recognition of safety and effectiveness (GRASE) determination continue to be marketed, leaving millions of American vulnerable to potentially unsafe products.

A staff of only 18 people cannot effectively regulate 800 active ingredients for over 1,400 different therapeutic uses. We are particularly concerned about how the current process limits FDA’s ability to require new warnings or other labeling changes to address emerging safety or effectiveness issues in a timely manner.

We strongly urge FDA to include funding in user fees to address emerging safety and effectiveness issues.  FDA needs resources to provide ongoing surveillance of marketed products and move quickly when safety signals arise.

This is especially important for products used by children. When the monographs were first developed in the 1970s, FDA lacked specific data on use in infants and children.  So, FDA did what was scientifically customary at the time and extrapolated the data by simply reducing adult doses by a percentage.

Our understanding of pediatric dosing has grown since then and, as a result, data from actual use in the pediatric population is preferred.  Many products continue to be given to infants and children without sufficient safety and effectiveness data. OTC user fees are needed to support the reexamination of the use of these products in children.

OTC product user fees should also support the development of product formulation standards. The monographs set forth the conditions under which a specific active ingredient used in a drug product is generally recognized as safe (GRAS) and generally recognized as effective (GRAE) and not misbranded.

The monographs, however, generally do not dictate what other non-active ingredients can be added, or other aspects of the formulation. For example, we know that many product formulation variables affect how much of a tablet’s medication dose is absorbed. The regulatory science behind generic drugs has shown us that excipients and manufacturing quality control must factor into the determination of a product’s safety and effectiveness. Therefore, we recommend development of standards for drug products, not just their ingredients; and we urge FDA to include funding for this in user fees. (As United States Pharmacopeia (USP) has recommended.)

A user fee system for OTC drugs will have to take into account the way OTC drugs come to market.  Since the monograph system is ingredient-based and not product-based and, since sponsors of monograph drugs are not required to obtain FDA approval prior to marketing, the fee structure will have important differences as compared to that used by the prescription drug programs.

We recommend that user fees be structured as a product listing fee based on a sliding scale proportionate to the complexity and reviewing resources required. We feel this mechanism would provide the agency with a stable and predictable source of funding for the OTC division.

We would avoid structuring the fee as a facility fee since it may have the unintended consequence of pushing sponsors to consolidate operations into as few facilities as possible. This could impact the supply chain and cause OTC drug shortages, if a facility is removed from operation.

In summary:

  • We support the establishment of a user fee program for OTC drugs so that all OTC monographs can be finalized.
  • We urge you to include funding in user fees to address emerging safety and effectiveness issues as well to reexamine use of certain OTC products in infants and children. Additional resources are critical to protect public health.

Thank you for your attention.

American Medical Student Association
National Center for Health Research
Our Bodies Ourselves
Quinolone Vigilance Foundation
The TMJ Association
Washington Advocates for Patient Safety