Three Ways the FDA can Fight Cancer

On September 12, President Biden became the latest chief executive with a plan in hand to fight cancer. Biden called for a “moonshot” to beat cancer, rightly noting that, “[w]e know too little about why treatments work for some patients, but a different patient with the same disease, it doesn’t work for. We still lack strategies in developing treatments for some cancers.” While the President mainly called for increased federal research funding, there’s not too much evidence that federal “investment” leads to increased innovation in the long run.

There is plenty the Biden administration can do immediately that would help Americans fight the scourge of devastating disease. No anti-cancer moonshot will be complete without significant reforms at the Food and Drug Administration (FDA). Below, the Taxpayers Protection Alliance (TPA) highlights three ways that FDA reforms can save and improve lives:

Expand over-the-counter (OTC) medication sales

Before a new medication is approved by the FDA, manufacturers and regulators must see eye-to-eye on whether the drug will require a prescription. In theory, prescription status is warranted when medication risks are high and/or the treatment regimen is confusing. Plenty of lifesaving medications, though, currently require physician sign-off despite being low-risk and straightforward to use. And that’s a problem when Americans overwhelmingly use OTCs as their first line of defense and rarely see doctors. Doctor’s visits are expensive, embarrassing, and time-consuming, resulting in patients not seeking out the prescriptions they need.

Even switching a few medications from prescription to OTC could save thousands of lives. An increasing body of evidence shows that statin medications not only significantly reduce the risk of heart attacks, but also reduce, “the risk of developing various cancers, and improve…the outcomes for cancer patients.” Meanwhile, side-effects are minor and resolve once patients stop treatment. Additionally, selective estrogen receptor modulators such as raloxifene can reduce the risk of developing breast cancer by 40 percent. While blood clots are a very real side effect of these medications, risks are low especially if patients don’t have a history of blood clots. Surely, patients who have seen their family members struggle with breast cancer can weigh these risks against the significant benefits of these therapies.

Embrace flexibility in drug evaluation process

To get the FDA’s stamp of approval, manufacturers must show that their therapy is safe and effective for patients. While not explicitly required, the agency is far more likely to approve medications that have gone through two, large-controlled clinical trials. If these trials demonstrate a drug having a statistically significant impact on a specified endpoint (e.g., five-year disease survival), then the sponsor has a decent chance of getting a thumbs-up from regulators.

Developers of drugs for rare diseases, though, often find themselves between a rock and a hard place. If a disease only impacts a smaller sliver of the population, it can be difficult to find enough participants to enroll in a clinical trial. In addition, manufacturers may conclude that it’s cruel to only give the therapy to some, instead of all, sufferers. Some sponsors have tried to circumvent these issues by relying on natural history studies in which drug users are closely monitored to see how the medication is impacting disease progression. These patient experiences can even be compared to historical data from non-users to see how drug users are faring with the new therapy. Unfortunately, though, the FDA hasn’t taken too kindly to natural history data. In 2019 guidance, the agency states, “FDA regulations recognize historical controls as a possible control group (usually reserved for special circumstances); however, inability to control for certain biases could limit the ability of externally controlled trials to demonstrate substantial evidence of effectiveness.”

AVEO Oncology found this out the hard way in 2020 when the FDA rejected its refractory renal cell carcinoma drug (Fotivda) because the company gave patients in the control group of its study access to the medication. Even though the company showed robust results and the therapy had already been approved in the EU (in 2017), the manufacturer had to go back to the drawing board and submit additional data. The FDA made cancer patients wait an additional year before finally approving the medication in 2021.

The FDA has similarly balked at natural history study data for ALS treatments, Hopefully, the FDA advisory panel’s recent vote to recommend approval of a new ALS drug is a sign that things are slowly changing. Going forward, the agency should keep an open mind to compelling data that doesn’t fit into its classical criteria for clinical trials.

Greenlight reduced-risk smoking alternatives

Cigarettes are a big killer. According to the American Cancer Society, “[s]moking causes about 20% of all cancers and about 30% of all cancer deaths in the United States.” Products such as e-cigarettes can give users the sensation of smoking with about 95 percent less risk. Unfortunately, the FDA is determined to make it as hard as possible for smokers to access these life-saving products. The agency has denied several companies’ applications to sell flavored vapes, even though, as Reason editor Jacob Sullum notes, “former smokers who have switched to vaping overwhelmingly prefer nontobacco flavors, and the FDA acknowledges that ‘electronic nicotine delivery systems’…hold great promise as a harm-reducing alternative to cigarettes.”

Unfortunately, federal and state governments care more about waging political battles against vaping companies than combatting smoking. Juul has tentatively agreed to pay a nearly $500 million settlement “in a multistate agreement to settle numerous allegations (without admitting wrongdoing) related to youth vaping.” These large legal and regulatory costs will inevitably be borne by adult vapers. There are about 4 times as many adult vapers as teenage vapers, and it is up to regulators to make sure that adult smokers have all options at their disposal to quit. This means rolling back lawsuits and regulatory actions and allowing e-cigarette makers to save lives.

In Conclusion….

Any “moonshot” to vanquish cancer will require significant regulatory reforms across federal and state governments. The FDA can move the needle on treatment by rethinking its reliance on outdated and ineffective rules.