Category Archives: Policy

Our Comments on the FDA’s Use of Generally Accepted Medical Knowledge Draft Guidance

July 24, 2023

We are pleased to have the opportunity to share our views with the Food and Drug Administration (FDA) on their Guidance: Generally Accepted Scientific Knowledge in Applications for Drug and Biological Products: Nonclinical Information.

We are a nonprofit think tank that conducts, analyzes, and scrutinizes research on a range of health issues, with a particular focus on which prevention strategies and treatments are most effective for which patients and consumers. We do not accept funding from companies that make products that are the subject of our work, so we have no conflicts of interest.

We support the efforts of the FDA to remain flexible and provide a more open application of scientific and regulatory judgment when conducting nonclinical studies that determine in vitro safety and efficacy. We recognize that this has the potential to streamline product development as well as avoid unnecessary animal testing, decrease costs, and hasten new drug approval time.  However, we predict that this will result in some sponsors providing insufficient data to show the safety, toxicology, and efficacy profile of a drug.

For that reason, we do not recommend using Generally Accepted Scientific Knowledge (GASK) as the sole source of nonclinical data in New Drug Applications (NDAs) or Biologic License Applications (BLAs). Instead, GASK should only be used to support sponsor data and should not replace the vital research that is needed to create a robust safety and efficacious drug profile.

Permitting GASK as the sole or primary evidence would often create a level of ambiguity regarding a drug’s true pharmacologic, distributive, and toxicologic effects. We support sponsor communication that is clear and data that are unequivocal, so that potential harms are minimized. We strongly encourage that the GASK used be: 1) long-standing, 2) uncontroversial, and 3) scientifically robust.  However, we note that even when the generally accepted scientific knowledge being relied upon is uncontroversial, long-standing, and scientifically robust, the external application of that information by the sponsor may be inappropriate. For example, even when a drug’s mechanism of action or the biologic pathway that a drug acts on is well understood, the drug should still be tested to assure that there are no downstream or unanticipated effects when in vitro to the full extent possible, and not solely rely on GASK.

In conclusion, we appreciate the opportunity to comment and support the efforts of the FDA to streamline product development and avoid unnecessary animal testing, decrease costs, and hasten new drug approval time. However, this must be better balanced with the need for sponsors to provide clear, unambiguous data regarding the pharmacologic, safety, and effectiveness profile of new drugs and not use GASK as a means of replacing vital research. To that end, we strongly urge the FDA to revise the proposed GASK guidance to require comprehensive and precise language on when using GASK by the sponsor is appropriate.

Testimony of Diana Zuckerman at the CMS Meeting Regarding Transitional Coverage for Emerging Technologies

August 1, 2023

I’m Dr. Diana Zuckerman, president of the National Center for Health Research and Cancer Prevention and Treatment Fund.  Our Center is a public health think tank that focuses on policies and programs that increase the safety and effectiveness of medical products.

We support the goals of the TCET program, but we see an enormous disconnect between the types of evidence that FDA requires for breakthrough devices, especially for 510k devices, and the CED standards that CMS requires for coverage.  We hear a lot about flexibility from FDA and industry but not enough about scientific evidence of safety or effectiveness.  Innovation should be defined to require that a device is proven to be better, not just newer.  We urge CMS to work with industry to make it clear that CMS evidence standards of clinical benefit are very different from the standards required by CDRH.  And we urge CMS to urge companies to provide the evidence needed to be worthy of CMS coverage.

Let’s remember that the standards for FDA for devices are not proof of safety or effectiveness, but rather the “reasonable assurance of safety and effectiveness.”  And often that “reasonable assurance” is not so reassuring.

I want to reiterate the previous comment that even when the FDA requires clinical data, many types of Medicare beneficiaries are under-represented:  older patients, people of color, and women.

I want to thank CMS for this effort to improve access to safe and effective medical products and urge CMS to hold firm to its standard for coverage based on scientific evidence that all medical products are proven to be reasonable and necessary for Medicare patients.

Our Comments on FDA Guidance Regarding Decentralized Clinical Trials for Drugs, Biological Products, and Devices

August 1, 2023

We are pleased to have the opportunity to share our views with the Food and Drug Administration (FDA) on their draft guidance regarding Decentralized Clinical Trials (DCTs) for Drugs, Biological Products, and Devices.

We are a nonprofit think tank that conducts, analyzes, and scrutinizes research on a range of health issues, with a particular focus on which prevention strategies and treatments are most effective for which patients and consumers. We do not accept funding from companies that make products that are the subject of our work, so we have no conflicts of interest.

DCTs have the potential to increase inclusion of underserved or rural communities and patients who are home-bound. It reduces geographic barriers by enabling patients to participate in clinical trials even if they do not live near a study site. This is much more convenient than traveling to a conventional study for medical interventions, data collection, assessments, and follow-up visits. However, the FDA and those conducting DCTs must also recognize how they could inadvertently reduce the enrollment of diverse populations when relying on digital health technologies (DHTs). The inability to access DHTs or lack of familiarity or comfort with DHTs could limit or prevent enrollment of older adults and children, participants living in rural areas, people who speak a language other than English, and people who have lower literacy skills or lower income. We recommend that FDA revise the guidance to incorporate the following strategies when implementing DCTs, including those that use DHTs:

  1. The guidance should incorporate information on how patients who decide to use their own DHTs during DCTs differ from those individuals who are provided a DHT during the study. Patients who regularly use these tools before study participation may differ in terms of several baseline health characteristics, vary in socioeconomic status, or have different health habits than those who do not have DHTs. Data recorded during a trial should include information on a participant’s prior device experience, as well as reasons for use, and include these variables in study analysis, as prior experience with a device could affect outcomes.
  2. DCTs using DHTs should consider and plan for the consistent availability and use of the same DHTs over time, even if newer versions of a device are introduced, as these updates could affect trial performance. Additionally, DHTs that require software on a participant’s phone or computer could undergo multiple app updates over the course of the study, with the potential to add new features that affect data input. Investigators must plan to follow-up with participants whenever major software updates occur.
  3. The FDA guidance mentions that sponsors should consider syncing information recorded by DHTs across different platforms. Digital health sources such as electronic health record (EHR) portals or portals linking data from personal digital devices, can be disconnected during the course of the DCT and require significant additional effort to reconnect. These disconnections can come from password changes and security issues which can result in the loss of data, which may be temporary or permanent depending on the willingness or ability of the participant to reconnect. Planning for this possibility is essential to ensure comprehensive data capture. Study teams should regularly review the completeness and quality of the DHT data over the course of the study to reduce missing data.
  4. The guidance mentions that, “training should be provided to all parties (e.g., trial personnel, local HCPs, and trial participants)” related to the software used in DCTs. This should apply to all DHTs and, as appropriate, should include caregivers who may assist study participants during DCTs.
  5. Although FDA states that researchers “should attempt” to collect data regarding healthcare services provided outside the study site. This does not adequately provide explicit directions on how to provide information regarding unexpected or routine visits to non-study sites, such as an ER visit for an adverse event that may or may not be obviously related to the clinical trial. The FDA guidance recommended that providers outside of the clinical trial not be included in the task log, which tracks health care providers that perform trial-related activities. However, if that information is not collected and evaluated, then significant information about safety issues would inevitably be missed. FDA should explicitly address how potentially important information should be included in the task log and re-evaluate the need to include these data points.

Biden’s Crackdown on ‘Junk’ Plans: Minimal Impact on Payers

Jesus Mesal, Health Payer Specialist, July 14, 2023

The Biden administration’s proposed restrictions on short-term private health plans aim to protect consumers, but they raise questions about the future value potential of a thriving market segment and do little to quell the controversy about insurance criticized by some as “junk.”

Short-term plans offer flexible coverage periods, such as 30 days or three years, and cost 50% to 80% less than individual market coverage. They are not regulated to the same extent as plans offered in the Affordable Care Act insurance marketplaces and can, for example, exclude pre-existing conditions or limit the number of visits or coverage amounts.

The proposal from the Department of Health and Human Services, the Labor Department’s Employee Benefits Security Administration, and the Internal Revenue Service would restrict them to three months, or to four months at a maximum.

These “misleading insurance products” can “trick consumers into buying products that provide little or no coverage when they need it most,” says a joint statement from the agencies.


The plans, devised as a way for consumers to plug short-term gaps in coverage, have remained a source of political contention since the ACA was enacted in 2010. In 2016, the Obama administration limited their coverage period to three months to address concerns that consumers might choose these plans over comprehensive coverage under the ACA.

Two years later, former President Donald Trump reversed the rule, arguing that consumers should have choice. He extended the allowable duration of short-term plans to a year, with option for consumers to renew them for up to three years. Unlike the ACA’s once-a-year open-enrollment period, these plans are accessible at any time during the year.

Growing market

Since the policy swing, the short-term health insurance market has emerged as a thriving segment experiencing significant growth. The firm Persistence Market Research reports that its value reached $41.1billion in 2022, with the Trump administration rule change playing a substantial role in this expansion.

Mixed opinions

Democratic lawmakers have long advocated for measures to limit the impact of short-term plans. They argue that these lower-cost plans provide minimal coverage and have the potential to lure Americans away from more-comprehensive ACA plans.

The proposed rule could increase ACA marketplace enrollment by an estimated 60,000 individuals in the years2026, 2027, and 2028. Enrollment in ACA plans hit 16.3 million people this year, according to HHS.

They are called ‘junk’ plans for a reason,” said Diana Zuckerman, president of the National Center for Health Research. Zuckerman questioned why the Biden administration took so long to take this step and does not agree with the argument that having one of these plans is better than having no coverage at all. Short-term plans end up being more expensive for Americans because many people cannot afford the bills they receive when they do not have coverage for emergencies, she said.

“When more people have high-quality health insurance, we are all better protected. These plans do not provide the 10 essential health benefits required by the ACA,” she told Health Payer Specialist. “People claim they have a choice, but what we have observed is that due to misleading marketing, many customers do not fully understand what they are purchasing, and it ends up costing millions of dollars for all Americans.”


Our Public Comment on HHS Draft Framework to Support and Accelerate Smoking Cessation

We appreciate the opportunity to submit public comments to the Department of Health and Human Services (HHS) regarding their Draft HHS 2023 Framework to Support and Accelerate Smoking Cessation.

This is an incredibly important issue and we understand that this framework, and its goals, will guide HHS cessation efforts moving forward. We are a nonprofit think tank that conducts, analyzes, and scrutinizes research on a range of health issues, with particular focus on which prevention strategies and treatments are most effective for which patients and consumers. We do not accept funding from companies that make products that are the subject of our work, so we have no conflicts of interest. Below are recommendations for HHS to consider as the smoking cessation framework is developed.


Cigarette smoking is the leading cause of preventable death and illness in the United States. More than 480,000 die annually from smoke-related illnesses and approximately $240 billion was spent on healthcare for smokers in 2018.[1] Safe and effective cessation treatments and techniques are needed to improve the health and longevity of individuals living in the United States. A 2015 study from the Centers for Disease Control and Prevention (CDC) found that 68% of people want to quit smoking, but that desire alone is not enough to maintain a change in the behavior. Research has shown it can take 30 or more tries before a smoker quits successfully.[2],[3] The most common method of quitting for people in the United States is to set a quit deadline and attempt to stop cold turkey on that day. Evidence-based interventions that help assist in quitting have been developed, with some being more successful than others.

It is crucial for HHS to invest in cessation strategies that are safe and effective, including behavioral therapies and nicotine replacement therapies (NRT). Behavioral therapies and nicotine replacement therapies (NRTs) are common, with hundreds of studies looking at which of them are most effective. A 2021 meta-analysis of behavioral therapies found that any form of counseling (e.g., in-person, telephone, self-help, etc.), as well as guaranteed financial incentives, were the most successful forms of therapy.[4] The same study analyzed the economic benefits of behavioral therapies and found that different behavioral therapies were equally cost effective. A 2023 meta-analysis of NRTs looked at 68 studies and found a combination of NRT, such as nicotine gum alongside a nicotine patch, was more effective than using any method individually.[5] The same study also found that higher-dose products are more effective than their lower-dose counterparts and that using NRT prior to quitting smoking can be more effective than starting the NRT after quitting. However, higher-dose products may also have greater risks, and smoking cessation products can have serious psychiatric side effects.[6] Therefore, studies of the efficacy of NRTs compared to each other or to behavioral/counseling treatments need to also consider data on unpleasant or serious side effects.

Some claim that e-cigarettes can be used as a cessation therapy.  This claim has been described as “negligent and misinformed.”[7]  Research in this area needs to be carefully reviewed because despite conflicting findings, there is growing evidence that e-cigarettes are not safe and not effective to aid in smoking cessation.[8],[9] Most of the studies that support e-cigarettes for smoking cessation have flaws making the results questionable (e.g., short term follow up or not randomly assigning participants.) Since vaping is often promoted as a tool to quit smoking, it is essential to have better data to determine whether this is supported by evidence.


Increasing access and coverage of cessation treatments is a key step in decreasing the number of deaths related to smoking. A 2018 study examined patients who were offered smoking cessation treatments by their primary care doctor. The results showed fewer than 20% of smokers accepted any type of therapy.[10] This same study found that White patients were more likely to be offered a prescription for a cessation medication whereas Black patients were more likely to be offered cessation counseling. We recommend HHS determine effective strategies to help primary care providers increase patients’ knowledge about effective cessation therapies and willingness to try them. These key providers can give information to patients about how they can stop smoking and what resources are best at helping them to do so. Providing patients with several effective options, will allow them to find which strategy, or perhaps strategies, work best for aiding their smoking cessation.


Expanding surveillance regarding smoking and cessation behaviors is vital to understand the patterns and trends of the behaviors. Regularly evaluating data from point of sales and compliance with advertising requirements will allow HHS to act on the most up-to-date information and enforce decisions by the court. For example, the FDA banned flavored e-cigarettes after data showed that teenagers used preferred them to traditional tobacco flavors, and that they potentially served as a gateway to nicotine addiction.[11] Similarly, it is important to ensure that advertising restrictions banning the promotion and sales of e-cigarettes aimed at children and teenagers are enforced. We also recommend collecting information about trends in popular cessation therapies such as purchasing trends for NRTs. This will enable HHS to encourage safe and effective cessation methods.


Promoting well-designed research to support and accelerate smoking cessation will help achieve the Cancer Moonshot’s goal to reduce the cancer death rate by 50% over the next 25 years. Better research is needed comparing the effects of different cessation therapies for different demographic groups.  Across races, people who quit smoking, particularly early in their life, have reduced risk of all-cause mortality.[12] However, there is not enough research on the most effective cessation therapies for specific populations. A small study conducted by researchers at the University of Miami found there were greater decreases in perceived stress for African Americans and Hispanics after receiving behavioral therapy for smoking cessation, but the study did not look at the effectiveness of other cessation therapies.[13]


[1] Centers for Disease Control and Prevention. (2022, July 28). Costs and expenditures. Centers for Disease Control and Prevention.

[2] Babb, S., Malarcher, A., Schauer, G., Asman, K., & Jamal, A. (2017). Quitting Smoking Among Adults – United States, 2000-2015. MMWR. Morbidity and mortality weekly report65(52), 1457–1464.

[3] Chaiton, M., Diemert, L., Cohen, J. E… & Schwartz, R. (2016). Estimating the number of quit attempts it takes to quit smoking successfully in a longitudinal cohort of smokers. BMJ open6(6), e011045.

[4] Hartmann-Boyce, J., Livingstone-Banks, J., Ordóñez-Mena, J. M… & Aveyard, P. (2021). Behavioural interventions for smoking cessation: an overview and network meta-analysis. The Cochrane database of systematic reviews1, CD013229.

[5] Theodoulou, A., Chepkin, S. C., Ye, W… & Lindson, N. (2023). Different doses, durations and modes of delivery of nicotine replacement therapy for smoking cessation. The Cochrane database of systematic reviews6(6), CD013308.

[6] Campbell, A. R., & Anderson, K. D. (2010). Mental health stability in veterans with posttraumatic stress disorder receiving varenicline. American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists67(21), 1832–1837.

[7] McAlinden, K. D., Eapen, M. S., Lu, W., Sharma, P., & Sohal, S. S. (2020). The rise of electronic nicotine delivery systems and the emergence of electronic-cigarette-driven disease. American journal of physiology. Lung cellular and molecular physiology319(4), L585–L595.

[8] Laucks, P., & Salzman, G. A. (2020). The Dangers of Vaping. Missouri medicine117(2), 159–164.

[9] Venkata, A. N., Palagiri, R. D. R., & Vaithilingam, S. (2021). Vaping epidemic in US teens: problem and solutions. Current opinion in pulmonary medicine27(2), 88–94.

[10] Hooks-Anderson, D. R., Salas, J., Secrest, S., Skiöld-Hanlin, S., & Scherrer, J. F. (2018). Association between race and receipt of counselling or medication for smoking cessation in primary care. Family practice35(2), 160–165.

[11] Leventhal, A. M., Goldenson, N. I., Cho, J., Kirkpatrick, M. G., McConnell, R. S., Stone, M. D., Pang, R. D., Audrain-McGovern, J., & Barrington-Trimis, J. L. (2019). Flavored E-cigarette Use and Progression of Vaping in Adolescents. Pediatrics144(5), e20190789.

[12] Thomson, B., Emberson, J., Lacey, B… & Islami, F. (2022). Association Between Smoking, Smoking Cessation, and Mortality by Race, Ethnicity, and Sex Among US Adults. JAMA network open5(10), e2231480.

[13] Webb Hooper, M., & Kolar, S. K. (2015). Distress, race/ethnicity and smoking cessation in treatment-seekers: implications for disparity elimination. Addiction (Abingdon, England)110(9), 1495–1504.

NCHR Comments on FDA’s Survey on Quantitative Claims in Direct-to-Consumer Prescription Drug Advertising


June 26, 2023

The National Center for Health Research (NCHR) appreciates the opportunity to submit public comments on the notice by the Food and Drug Administration regarding their Survey on Quantitative Claims in Direct-to-Consumer (DTC) Prescription Drug Advertising.

NCHR is a non-profit think tank that conducts, analyzes, and scrutinizes research on a range of health issues, with particular focus on which prevention strategies and treatments are most effective for which patients and consumers. We do not accept funding from companies that make products that are the subject of our work, so we have no conflicts of interest.

We strongly support the efforts of the Office of Prescription Drug Promotion (OPDP) to protect public health by conducting research to evaluate patients’ understanding of quantitative information provided in prescription drug advertising. We work closely with patients and consumers and are well aware that many people do not understand quantitative claims made in drug advertisements.1 The information collected in this survey is essential to ensure the public is able to comprehend drug information in order to make informed medical decisions.

We appreciate the clear example provided in the notice regarding the type of information FDA is seeking to collect (i.e. claims describing medians). Some of the statements included were clear and direct (e.g., “People treated with Drug X lived for a median of 8 months” in combination with the explanation that “In people receiving Drug X, this means that about half lived more than 8 months and about half lived less than 8 months”). Research to evaluate patients’ understanding of such explanations will provide crucial information about individuals’ level of comprehension regarding quantitative information.  

We strongly recommend that determining patients’ and consumers’ comprehension of information regarding relative risk, absolute risk, relative benefit, and absolute benefit should also be evaluated by the FDA in this surveyFor example, how does the public understand a statement that a drug reduces the relative risk of recurrence by 37% or increases the absolute chances of 5-year survival by 3%? Information about relative risk or relative benefit is often used in advertising because those numbers are inevitably larger and seem more impressive than the absolute difference in risk or benefit. According to the FDA’s own research, 65% of physicians believe DTC ads confuse patients about the relative risks and benefits of prescription drugs.2 Unfortunately, patients are not the only ones confused by these statistics.

The FDA notice states that survey questions will be informed by consumer feedback elicited in one-on-one interviews. We support this method of data collection but request that the FDA be more specific about how many interviews OPDP plans to conduct to compile this information, and what type of demographic diversity they will require when selecting people to be interviewed. A relatively small or homogeneous selection of individuals to interview one-on-one could result in unintentional bias in the survey, which in turn would have implications for the results of the estimated 1,100 completed surveys.

We urge you to consider our recommendations, which are intended to enhance the quality of the survey. We would be happy to help recruit patients for your survey or interviews.



1. Sullivan, H. W., O’Donoghue, A. C., Lynch, M., Johnson, M., Davis, C., & Rupert, D. J. (2019). The Effect of Including Quantitative Information on Multiple Endpoints in Direct-to-Consumer Prescription Drug Television Advertisements. Medical decision making : an international journal of the Society for Medical Decision Making, 39(8), 975–985.

2. Food & Drug Administration. (2015). The Impact of Direct-to-Consumer Advertising. 

NCHR Comments on USPSTF Draft Recommendation on Breast Cancer Screening

June 6, 2023

We are pleased to have the opportunity to express our views regarding the U.S. Preventive Services Task Force Draft Recommendation Statement regarding breast cancer screening.

The National Center for Health Research (NCHR) is a nonprofit think tank that conducts, analyzes, and scrutinizes research on a range of health issues, with a particular focus on which prevention strategies and treatments are most effective for which patients and consumers. We do not accept funding from companies that make products that are the subject of our work, so we have no conflicts of interest.

While we support the task’s draft recommendation that women who are at average risk of breast cancer should undergo a screening every other year rather than annually, we are concerned that the task force’s recommendation of lowering the age of screening mammography from 50 years old to 40 years old is broadly applied to all women, rather than directed at groups most at risk. The guidelines are only supposed to be regarding women of average risk of breast cancer, and information is widely available to indicate some women are at higher risk because of genetic predispositions, smoking, obesity, family history, and other factors.  Dense breasts are also a risk factor, but unfortunately breast density also makes mammography less accurate[1] and tends to be especially high for women under the age of 50[2].

It is especially important to note that the most recent research, which may post-date the writing of these draft recommendations, had findings that suggested that Black females should start screening approximately 8 years earlier than White women, and that Hispanic and Asian and Pacific Islander females could start even later[3]. For that reason, we urge the USPSTF to consider whether the recommendation to start at age 40 should only apply to Black women and to other women who also have higher than average risk of breast cancer at a younger age, whereas starting at age 50 or even later is scientifically supported for other racial/ethnic groups that have been studied.

We agree with the USPSTF that there is not enough evidence to recommend screening mammography for women 75 years old or older.

We also agree that biennial mammogram screening has benefits that outweigh the risks for most women between the ages of 50-74 and for women at high risk between the ages of 40-50, there is currently insufficient evidence that using additional screening tools, such as using an MRI following a screening mammogram, is beneficial even for women with dense breasts, unless a diagnosis is needed when abnormalities are shown during the mammogram.

We understand that the USPSTF may be reluctant to suggest different screening schedules for Black women or for any specific group of women, but we urge the Task Force to focus on the scientific data. In this case, that includes different recommendations based on race and ethnicity data. Women are capable of understanding why the age to start mammography screening may be different for women with different risk factors. What is confusing is when some physician groups recommend annual mammograms for all women starting at age 40, even though the data do not support that recommendation. USPSTF should not compromise its standards to be more similar to those recommendations.

NCHR is grateful for the opportunity to comment on this USPSTF draft recommendation. The National Center for Health Research can be reached at or (202) 223-4000.



1) Kolb TM, Lichy J, Newhouse JH. Comparison of the performance of screening mammography, physical examination, and breast US and evaluation of factors that influence them: an analysis of 27,825 patient evaluations. Radiology. 2002 Oct;225(1):165-75. doi: 10.1148/radiol.2251011667. PMID: 12355001.

2) Barrette, Lori, Breast Density: What Women Should Know” University of Rochester Medical Center. 15 October 2015. <>

3) Chen T, Kharazmi E, Fallah M. Race and Ethnicity–Adjusted Age Recommendation for Initiating Breast Cancer Screening. JAMA Netw Open. 2023;6(4):e238893. doi:10.1001/jamanetworkopen.2023.8893

Patient, Consumer, and Public Health Coalition Letter to FDA Commissioner Califf Regarding FDA Advisory Committee Meetings

July 12, 2023

The Honorable Robert Califf, MD
Food and Drug Administration
10903 New Hampshire Ave.
Silver Springs, MD 20993

Dear Commissioner Califf,

As members of the Patient, Consumer, and Public Health Coalition, we appreciate your recent comments reaffirming your commitment to improving the format of Advisory Committees and look forward to working with you throughout these efforts. However, we also want to express our strong concerns when the Food and Drug Administration (FDA) Center Directors publicly undermine or privately overrule the recommendations of their own scientists’ and statisticians’ regarding applications for accelerated approval and full approval.

At our meeting with you on March 1, you expressed your view that some nonprofit leaders and academic researchers were inaccurately and unfairly assuming that FDA Advisory Committee members were more knowledgeable than the FDA scientific reviewers who had spent months reviewing the data and other information provided by sponsors. What we have seen for several years, particularly in recent months, is that the FDA Advisory Committee members often agree with scientific and statistical concerns and conclusions expressed in the FDA scientific memoranda provided to them to review, and it is the Center Directors or other officials that are overriding the views of FDA scientific reviewers.

Below we have summarized a few recent examples where the FDA decision conflicted with FDA scientists and statisticians summaries that were provided to FDA Advisory Committee members and the public.

FDA staff who presented the data at the FDA Advisory Committee meeting on May 12 regarding the accelerated approval application for Sarepta’s gene therapy Elevidys made it very clear that they did not feel remotely confident that the benefits outweighed the risks for boys ages 4-7. For example, they stated that the surrogate endpoint for the drug is not “reasonably likely to predict clinical benefit” in support of accelerated approval. They also stated that although the data appeared to be more promising for boys ages 4-5, the post-hoc analysis could not be trusted. However, in his remarks, Center Director Peter Marks told the Advisory Committee that the FDA should show flexibility by granting approval. We consider this flexibility especially problematic because FDA had previously granted accelerated approval to three other Sarepta drugs, none of which have completed their confirmatory trials. In fact, the confirmatory trial for Exondys 51, which was approved in 2016, were due in November 2020 but instead was not even started until July 2020.[1]

Despite Dr. Marks’ persuasive remarks at the Advisory Committee meeting, members only narrowly voted (8-6) in favor of the gene therapy, and even those who voted in favor expressed numerous concerns about the data. It has been reported that Dr. Marks overruled the recommendations of all FDA staff when he granted accelerated approval to Elevidys, consistent with what was clear for all to see at the Advisory Committee meeting.[2] Sarepta immediately announced that the treatment would cost $3.2 million per patient. Meanwhile, Sarepta is charging up to $1 million per patient per year for Exondys 51 – approximately four times the cost the company estimated in 2016. In fact, Sarepta has so far earned a total of $2.5 billion in sales from Exondys 51 and the two other Duchenne drugs granted accelerated approval.[3]

Unfortunately, the unilateral decision by CBER Director Peter Marks to overrule his own staff is troubling and harmful to the reputation of the FDA, as was the similar decision by CDER Director Janet Woodcock regarding Exondys 51 in 2016. These are just two of numerous examples where FDA Center Directors have gone against the recommendations of its own scientists and advisors to grant approval for a drug when the safety and effectiveness were not consistent with FDA requirements for approval. Two other recent examples include the controversial approval of Aduhelm (initially for all Alzheimer’s patients although it was only tested on patients with mild cognitive impairment) and Relyvrio for the treatment of patients with amyotrophic lateral sclerosis (ALS). These drugs will be marketed for years without confirmation of clinical benefit.

The June 28, 2023 Advisory Committee meeting regarding a drug for Fibrodysplasia ossificans progressive (FOP) is a somewhat different example. As was the case with Elevidys, the written memorandum by FDA staff expressed strong concerns about the scientific evidence: The primary end point was not met; the historical comparison sample was inappropriate; the data were manipulated post hoc in questionable ways; the nominal benefits were unreliable due to the wide confidence intervals; and there were increases in flare-ups – the very symptom that the drug was supposed to reduce. However, unlike the Elevidys meeting, the oral presentations by the FDA scientific and statistical staff were very obviously watered down versions of their written analyses. In fact, the main FDA presenter often seemed to be speaking on behalf of the sponsor, not the FDA. While still expressing concerns about whether the drug was safe and effective, the FDA speakers’ oral presentations contradicted the written FDA documents by stating that they were confident that the treatment probably had benefit and the risks of flare-ups were probably not so serious – an odd statement given that flare-ups were the outcome measure intended to be reduced by the treatment. Those statements were dramatically inconsistent with the written document summarizing the same analyses. This would have been worrisome but more justifiable for an accelerated approval, since the drug was intended to fulfill an unmet need for a terrible disease; however, the FDA meeting was considering full approval. FDA’s oral statements were so persuasive that several members of the Advisory Committee stated that the “FDA reassurances” convinced them that the benefits probably outweighed the risks, thus persuading them to vote in favor of the drug despite their strong reservations.

At an FDA Advisory Committee meeting in September 2022, FDA’s scientific summary of the confirmatory trials of PI3K inhibitor duvelisib (Copiktra) concluded that the risks outweigh the benefits for patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).  However, despite decreases in survival, the product retains its accelerated approval and FDA has not announced whether it intends to support those conclusions.

It is our understanding that FDA Advisory Committees are supposed to provide independent experts to review the FDA analyses, and not be unduly influenced by FDA staff or officials’ urging members how they should vote. Moreover, we agreed with your remarks on March 1 that FDA scientists and statisticians are the experts with the greatest knowledge of the data and the issues involved. It undermines the integrity of the FDA, the morale of FDA staff, and the public’s faith in FDA decisions when FDA officials grant an approval that contradicts its own scientists and statisticians. Equally important, overriding the scientific staff harms patients and contributes to a healthcare system that is financially unsustainable.

You have made it clear in numerous public statements that you are concerned about the format of Advisory Committee meetings, and we are as well. We urge you to speak out about the importance of FDA decisions supporting the FDA’s own scientific and statistical analyses. Please consider the following recommendations as you work to improve the format of the Advisory Committees:

  1. Provide training to FDA Advisory Committee members to help them understand the statistical analyses that are an essential part of all Advisory Committee meetings, and ensure they respect the importance of understanding and considering scientific evidence as part of their advisory role;
  2. Require that the FDA scientific and statistical staff who write the FDA memoranda for Advisory Committee meetings have the scientific freedom to express their own views, and that those views are accurately presented in FDA oral presentations at the meeting;
  3. Encourage Center Directors and other FDA officials attending Advisory Committee meetings to refrain from making comments that can be interpreted as encouraging committee members to vote a particular way; when FDA officials attend these meetings, their remarks should make it clear that FDA wants to hear their views and not to influence their votes.
  4. Remind Center Directors and other FDA officials that overruling the views of their own scientific and statistical staff undermines the public trust, and should be avoided, especially when the scientific staff are in consensus.


American Medical Student Association Wisconsin

Breast Cancer Action

Doctors for America

Government Information Watch

Jacobs Institute of Women’s Health

Medical Device Problems


Mothers Against Medical Error

MRSA Survivors Network

National Center for Health Research

National Women’s Health Network

Patient Safety Action Network

TMJ Association

USA Patient Network

Washington Advocates for Patient Safety




1. A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON). Available:

2. Becker, Z. (2023). In approving Sarepta’s DMD gene therapy, FDA’s Peter Marks overruled reviewer’s rejection. Fierce Pharma. Retrieved from Accessed July. 5, 2023.

3. Langreth, R., Rutherford, F., Milton, I., & Campbell, M. (2023). Sarepta’s gene therapy gets FDA accelerated approval: Are fast-tracked drugs safe? Benefits Pro. Accessed July. 5, 2023.

Meeting of Patient, Consumer, and Public Health Coalition with Commissioner Califf and Key FDA Officials

March 1, 2023

Location: FDA White Oak Campus, Silver Spring, MD-Building 1, Great Room

FDA attendees: Robert Califf (Commissioner, virtual due to exposure to viruses), Peter Marks (Director, CBER, virtual), Owen Faris (OPEQ Principal Deputy Director, CDRH, virtual); In person: Julia Tierney (Chief of Staff, Office of the Commissioner), Jacqueline Corrigan-Curay, (Principal Deputy Director, CDER), Dayle Cristinizio (Director, Stakeholder Engagement, Office of External Affairs), Hilary Maston (Chief Medical Officer), Namandje Bumpus (Chief Scientist, AdCom responsibilities), Kaveeta Vasisht (Assoc Commissioner for Women’s Health), Jennifer Dooren (Director Communications and Public Engagement, Food Safety).

Coalition Attendees: In Person: Thomas Eagen (NCHR), Maria Gmitro (BISA), Denise Hyater-Lindenmuth (NWHN), Patricia Kelmar (U.S. PIRG), Suzanne Robotti (MedShadow), Kim Witczak (WoodyMatters), Diana Zuckerman (NCHR).  Virtual: Tahir Amin (I-MAK), Wendy Dolin (MISSD), Helen Haskell (MAME), Rex Johnson (WAPS), Katherine Leon (SCAD Alliance), Judy Norsigan (OBOS), Linda Radach (PSAN), Reshma Ramachandran (DFA), Brian Ronholm (Consumer Reports), Tess Schulman (Medical Device Problems), Robin Strongin (NCL), Dru West (USAPN), Sophia Phillips (on behalf of the Coalition).

On behalf of the Coalition members at the meeting, Dr. Diana Zuckerman offered our support to the Commissioner and the FDA, and thanked the Commissioner for his efforts to improve the Accelerated Approval program by requiring confirmatory trials be started prior to granting accelerated approval. Patricia Kelmar discussed members’ support for the FDA to regulate lab-developed tests to ensure their accuracy. Kim Witczak and Suzanne Robotti discussed possible improvements to the FDA Advisory Committee process, based on their perspectives as consumer representatives on two FDA Advisory Committees.  After the meeting was completed, Coalition members who were attending in person had informal discussions with several FDA officials.

Joint Letter from from Cancer Groups and Patient-Centered Nonprofits about VALID Act in Omnibus

December 16, 2022

The Honorable Patty Murray
Senate Health, Education, Labor & Pensions Committee
Washington, DC 20510

The Honorable Richard Burr
Ranking Member
Senate Health, Education, Labor & Pensions Committee
Washington, DC 20510

The Honorable Frank Pallone
House Energy & Commerce Committee
Washington, DC 20515

The Honorable Cathy McMorris Rodgers
Ranking Member
House Energy & Commerce Committee
Washington, DC 20515

Dear Chair Murray, Ranking Member Burr, Chair Pallone, and Ranking Member Rodgers:

We are writing to express our strong support for your bipartisan support for the VALID Act earlier this year, and to urge you to include it in the Omnibus Spending bill with a few small improvements. Our concern is that women and men who are at high risk of breast cancer or other types of cancer are not currently able to have confidence that genetic tests used for screening and diagnosis are accurate. Our members were shocked to learn that the FDA only regulates diagnostic tests sold by companies, not those sold by laboratories. We have many examples of patients who have been terribly harmed when lab-developed diagnostic tests that were widely sold have been dangerously inaccurate. There are currently approximately 100,000 different lab developed tests that would be grandfathered under the current bill language, and none of them would be required to submit evidence now or in the future to prove that their tests are accurate. Even the highest risk lab developed tests that are currently being sold could continue to be sold without any restrictions, and it is unclear if serious adverse events caused by inaccurate tests would be reported to the FDA. That is unacceptable to us as patient-centered organizations.

Your constituents deserve better. In addition to the extensive grandfathering, the bill would categorize lab-developed tests as low, moderate, or high-risk. Low-risk would be essentially unregulated, moderate risk would  require some evidence but not be tested for accuracy (similar to the 510(k) review process). As you know, the 510(k) pathway is used to review approximately 95% of all medical devices, including most implanted devices, and it has been strongly criticized by physicians, patients, and public health experts because many of those devices are high risk, not moderate risk as the FDA claims.

As described in the current VALID provisions in the bill, only the highest risk devices would be required to provide clear evidence of accuracy, and the definition of high risk is unclear and based on the FDA’s track record is likely to exclude many tests that could cause irreparable harm, such as genetic tests that are used to inform people that they are at very high risk of breast cancer, ovarian cancer, stomach cancer, and other very serious diseases. Since many patients who are told that they tested positive for these genetic mutations have those important bodily organs surgically removed, those genetic tests should be considered high-risk. It would be equally tragic for a person to have an organ unnecessarily removed due to an inaccurate positive test result, or for a person to be incorrectly told they did not have a life-threatening genetic mutation, due to a false test negative result. Although we are focusing on breast cancer in this letter, it is well-known that most prenatal genetic testing is inaccurate, thus resulting in terminating wanted pregnancies because they were erroneously told their baby had a fatal birth defect. For that reason we strongly urge you to include genetic testing in the definition of “high risk” tests in the bill, and not allow grandfathering of high risk tests.

In addition to these shortcomings, we are concerned that academic medical centers are lobbying to be excluded from FDA regulation. While academic medical centers have an important role to play in U.S. healthcare, their tests are not monitored by the FDA, CMS, or any other independent entity to ensure they are accurate.  As a result, there are clear examples of tests that were dangerously inaccurate.  Any exemption for academic medical centers’ lab-developed tests should be extremely narrow, to target tests used for individuals, not for large numbers of patients.  High-risk tests should always be regulated.

In conclusion, we strongly recommend including an improved version of the VALID Act, as described above, in the Omnibus bill, in order to better protect patients and consumers from inaccurate and unreliable lab-developed tests.


Breast Implant Safety Alliance

Cancer Prevention and Treatment Fund

National Women’s Health Network

Not Putting on a Shirt

Our Bodies Ourselves

Patient Safety Action Network

USA Patient Network

Washington Advocates for Patient Safety



Cc: Members of Senate Health, Education, Labor, and Pension Committee and Members of Energy & Commerce Health Subcommittee