Speeding Ineffective and Unsafe Treatments to Market in the Name of Patients? Not So Fast

It is not a bottleneck at the FDA that is to blame. Medical research is simply not producing the breakthroughs needed for breast cancer. And we should not let a reduction of safety and efficacy standards replace our demand for safer and more effective treatments.
07/13/2015 08:18 pm ET Updated Jul 13, 2016

As the head of Breast Cancer Action, a national watchdog organization for the breast cancer movement, I frequently hear from long-time members who are outraged that breast cancer treatments remain largely the same as they were decades ago. Activists and doctors alike worry that the basic treatments for breast cancer continues to be "slash, burn, poison"--more commonly referred to in medical offices as surgery, radiation, chemotherapy--with too few advances benefiting too few women. Too many women continue to be diagnosed with and die from breast cancer.

Why, then, would Breast Cancer Action and other watchdog groups oppose a bill currently speeding throughCongress that some say will help get new medical products and treatments into the hands of patients faster?

Because, quick Food and Drug Administration (FDA) approvals don't help patients if the treatment or technology doesn't work in the first place--or worse if it is harmful!

Supporters of the 21st Century Cures Act (HR 6) say it will help to quickly bring new medical technologies and treatments to the marketplace. It is being promoted as a regulatory update in response to technological innovation and medical advancement. But the truth is that this industry-friendly bill is a give-away to Pharma and Biotech that threatens to reduce standards of safety and efficacy necessary to protect patients.

At Breast Cancer Action, we have been advocating for more effective, less toxic treatments since our founding 25 years ago. From our founders' early direct action on the lawns of Genentech to demand compassionate access to a promising new drug to our work to overturn the human BRCA 1&2 gene patents, we have been working to ensure that treatments, technologies, and tests are accessible to the women who need them. No one knows better than our members how desperately new, better treatments are needed--now.

Yet, we are gravely concerned that 21st Century Cures could lower the bar of evidence for medical devices and drugs in ways that may harm rather than benefit patients. As a watchdog for patients, we know that medical advancement relies on innovative research--not just reducing regulatory review to speed new technologies and treatments to market. Hopeful promises made by Pharma and Biotech about the latest, greatest medical device or drug must be backed by evidence and data that show that these new drugs and devices are indeed safe and effective for patients.

One key way that 21st Century Cures threatens to weaken safety and efficacy standards is by allowing "clinical experience" in place of well-designed randomized controlled clinical trials. Clinical experience--which includes observational studies, patient registries, and insurance claims--should inform and guide research questions, but it cannot replace rigorously-collected research data.

Additionally, the Act proposes new categories of fast drug approvals further threaten to water down standards for safety and efficacy. Shorter clinical trials are cheaper for industry but frequently rely on measures of effect called surrogate endpoints that can be unreliable predictors of ultimate outcome and do not necessarily correlate with benefits for patients. These smaller and faster studies may not catch rare or late-onset yet potentially serious--even life-threatening--side effects. Whether for a new drug or an expanded use of an already approved drug, adequate data is needed to make a determination that benefits outweigh the risks of treatment. 21st Century Cures threatens to undermine the FDA's ability to make such an assessment.

The problem of insufficient advancement of breast cancer treatments is not solved by speeding FDA approvals. For breast cancer patients, it's not as if there is a backlog of safe and effective breast cancer treatments and tests sitting out of reach, awaiting FDA approval. Innovative breakthroughs and dramatic advances in better treatments and technologies are few and far between. All too often, highly anticipated new treatments or tests end up showing very modest incremental benefit, at best.

The real barrier to breast cancer patients receiving more effective, less toxic treatments is scientific, not regulatory. Lowering standards for safety and efficacy will not speed up innovation--and instead threatens to lower the quality of treatments that come to market.

In the US, drug approvals are already faster than in Europe and Canada. And the FDA already has several mechanisms in place to speed promising new therapies to market. Unfortunately, rather than benefiting patients, 21st Century Cures runs the risk of sacrificing safety and efficacy in the name of speed.

Currently, 21st Century Cures is getting bipartisan support for increasing the budget for the National Institutes of Health (NIH). However, no amount of funding for basic research should justify the lowering of scientific standards for any new treatments that result from such research.

At Breast Cancer Action, we refuse to allow safety and efficacy standards to be undermined in the name of patients. Despite the billions of dollars spent in the name of breast cancer, activists have long asked what we have to show for it. Why are 40,000 women dying each and every year from breast cancer? We have seen too few advances that benefit too few people affected by breast cancer.

It is not a bottleneck at the FDA that is to blame. Medical research is simply not producing the breakthroughs needed for breast cancer. And we should not let a reduction of safety and efficacy standards replace our demand for safer and more effective treatments.