Coalition Letter to Senator braun regarding the Promising Pathways Act

Dear Senator Braun,

We are writing as nonprofit organizations that are part of the Patient, Consumer, and Public Health Coalition to share our views regarding the Promising Pathway Act (S. 1906/H.R. 4408). As advocates representing patients, consumers, physicians, and public health experts, we empathize with patients who have limited treatment options for potentially serious diseases. However, the Promising Pathways Act is poorly written and would have unintended consequences of pressuring the FDA to approve drugs that would remain on the market for as long as 8 years without any evidence that they are effective. In contrast, the FDA has already implemented numerous pathways that make it easier for patients facing urgent, very serious unmet medical needs to have free access to experimental drugs or to pay for drugs and biologics with some evidence that they work. Given these better options, it makes no sense to pass S. 1906, which would result in approval of drugs for less serious medical problems that have better treatment options.

  • The Promising Pathway Act is dangerous as well as unnecessary.  FDA’s regulatory standards for drugs to treat rare and serious diseases are currently so flexible that some drugs are prescribed for years before evidence is gathered that indicates they are ineffective and/or unsafe. This is harmful to patients’ health and financial well-being, in some cases consuming a family’s entire financial nest egg for treatments that do more harm than good. Needless to say, it also threatens the financial stability of the Medicare program, creating a threat for the 65 million Americans who are 65 and older or have chronic disabilities and who depend on Medicare for their healthcare.
  • The Promising Pathway Act provides false hope rather than real promise. It is written in a way that would make many drugs eligible for approval that are not urgently needed and would otherwise never be approved.  For example, the pathway is intended for drugs that treat a medical condition that could lead to premature death. Perhaps the bill’s sponsors intended that to mean people whose lives are at risk in the near future, but as written it would include drugs for almost any medical problem that may have potential to improve one’s expected lifespan for a few months decades in the future. For example, since high blood pressure, high cholesterol, and being overweight all have the potential to lead to premature death, any drug treating those conditions could be eligible for the conditional pathway. It would not be restricted to drugs for lethal diseases, rare diseases, or those urgently needed due to lack of available treatments.

The bill would make sense if the FDA were currently requiring too much evidence to support approval.  Research clearly indicates that is not the case.  In fact, research suggests that the FDA is more likely to approve a drug or biologic that has no evidence of a clinically meaningful benefit to patients than it is to reject approval for one that has any evidence that it is likely to work. Unfortunately, in many cases the agency has shown so much flexibility in approving “promising drugs” that billions have been spent on drugs that are never proven to work. And yet, the Promising Pathways Act proposes to weaken these standards further, rather than maximize the chances that innovative new drugs will have evidence suggesting clear benefits that will soon be proven to work based on high quality evidence after it is approved. 

Currently, the law requires “substantial evidence” of safety and effectiveness – typically  at least one well-designed trial.  This bill would allow provisional approval based on relevant early evidence of a “positive therapeutic outcome” but the bill also allows for the use of biomarkers, which by definition are not measures of therapeutic outcome. Equally important, research shows that drugs that look promising in early development often are unable to confirm that they are safe and effective in later, more well-designed trials.

It is unfair to patients to pay for treatments that do not work. It is also unfair to taxpayers who are not prescribed these drugs but pay for them in the form of higher insurance premiums, including higher costs for Medicare coverage.

The bill also relies on registries to gather information about safety and efficacy, but registries have 2 major shortcomings: 1) the data only becomes available years after relatively large numbers of patients have taken the unproven and possibly unsafe medication; and 2) since registries do not include a randomized control group, it may be impossible to determine whether patients taking the drug have a better or worse outcome than patients that do not take the drug. Randomization to a new drug or a comparable group of patients that are not on the drug is much superior to a registry because some patients with a specific disease will inevitably do better than similar patients with the same disease even if they do not get treatment.

Some argue that the FDA should be willing to approve drugs that are not yet proven when patients have a serious disease and no proven treatments are available. However, that option already exists:  FDA’s accelerated approval pathway.  As noted earlier, the FDA is not refusing to approve drugs if there is any evidence that the benefits could outweigh the risks.

The National Organization for Rare Disorders (NORD) and the Pharmaceutical Research and Manufacturers of America (PhRMA) both oppose this bill, despite their strong lobbying for previous efforts to lower FDA approval standards. They recognize that this bill is fatally flawed because it has no safeguards for protecting patients from drugs that have no benefits. The Promising Pathways Act has the potential to greatly tarnish patients’ trust in the FDA and in new treatments approved by the FDA.


American Medical Student Association Wisconsin

Breast Implant Safety Alliance

DES Action USA

Jacobs Institute of Women’s Health

Medical Device Problems

Medshadow Foundation

MRSA Survivors Network

National Center for Health Research

National Women’s Health Network

Our Bodies Ourselves

Patient Safety Action Network

SCAD Alliance

USA Patient Network

Washington Advocates for Patient Safety

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