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No, Updating Federal Drug Regulations Will Not Spawn ‘Dangerous Medicines’


Following last week’s passage of the 21st Century Cures Act, praise for this $6.8 billion law echoed widely, with one stark exception: the law’s provisions granting the Food and Drug Administration more flexibility in the drug review and approval process.

In the run-up to passing the Cures Act and right after President Obama signed this widely supported law, critics have launched two main attacks on the FDA provisions. First, opponents condemn the Cures Act for failing to control drug prices. Second, critics claim the law loosens regulatory standards that “could cause ineffective or dangerous medicines to reach the public.”

Let’s consider each charge in turn, starting with the question of drug prices. I spoke with my friend and former law school classmate John Crowley, CEO of Amicus Therapeutics, Inc., to get his take on this criticism: “Think about it,” he said. “Costs are driven down by innovation and competition. When barriers to both are removed, there will be more drugs on the market and companies will be forced to compete on price.”

Crowley’s interest here is personal, not financial. He has two children with Pompe disease, a severe and often fatal neuromuscular disorder. Frustrated by the slow pace of research for drugs that could help his children, he co-founded a biotech company and raised capital to fund the research. His efforts resulted in the development of a first-generation treatment for Pompe disease, which he credits with saving his children’s lives. The movie “Extraordinary Measures,” starring Brendan Fraser and Harrison Ford, tells this amazing story.

Avik Roy has also written extensively on the impact of FDA regulations and drug prices. His study for the Manhattan Institute thoroughly demonstrates the verity of Crowley’s comments: reforming the FDA’s regulatory process will reduce drug prices. His threepart series at summarizes those findings.

The response? Risible rhetoric. For instance, during debate over the Cures Act, Sen. Bernie Sanders proclaimed: “If you want to lower the outrageous cost of prescription drugs, vote against this bill,” adding “[i]t is time to stand up against the pharmaceutical industry and stand up with the American people who are tired of being ripped off by this extremely greedy industry.” Sen. Elizabeth Warren likewise condemned the FDA provisions and branded Big Pharma a hijacker of the Cures Act.


Two hot takes: First, Sanders and Warren are putting (low) profits above patients. They voted against the Cures Act because they care more about punishing the pharmaceutical industry than finding cures for sick and suffering individuals.

If this has a deja screw you feel to it, it should. We’ve seen this screed from liberals before, such as when then-candidate Obama admitted he would raise capital gains taxes on the rich “out of fairness,” even if it resulted in less revenue for federal use. Second (at least for Warren), let’s add hypocrisy to the mix, since she has no problem associating with “Big Pharma” when it suits her, as demonstrated by her retweet of this praise:


While criticism that the Cures Act is a sop to “Big Pharma” smacks of unserious rhetoric, the second charge, that the Cures Act could result in the FDA approving ineffective or unsafe medicines, sounds more substantive—emphasis on sounds. For instance, consider The New Republic hit piece “Congress Just Quietly Handed Drug Companies A Dangerous Victory.” After spending half the keystrokes attacking the pharmaceutical industry or conservative think-tanks, it warns:

The 21st Century Cures Act allows ‘the potential use of real world evidence’ to support the approval of a novel indication for an approved drug and ‘to help to support or satisfy postapproval study requirements.’ That sounds rather reasonable: who could possibly oppose the use of ‘real world evidence’? The problem, however, is that ‘real world evidence’ actually means here ‘really bad evidence.’ The law defines ‘real world evidence’ as ‘data … derived from sources other than randomized clinical trials,’ which is to say uncontrolled observational data. This is perhaps the bill’s most dangerous clause. Randomized clinical trials are—in the vast majority of cases—the only way to know if a drug actually works, as observational studies can be notoriously unreliable in determining the efficacy of drugs.

Sounds scary. But what does the Cures Act actually say:


So The New Republic ignores the statutory text, which requires the FDA to establish a program to evaluate the potential use of real world evidence, and instead posits to its readers that under the Cures Act, the FDA will approve drugs based on “really bad evidence.”

This scare tactic also ignores other provisions of the Cures Act that will prevent the FDA from approving drugs based on “really bad evidence.” For instance, the Cures Act requires the FDA to define appropriate standards and methodologies for collecting real-world data. Before adopting any such regulations, the FDA must consult with medical professional organizations, patient advocacy groups, consumer organizations, disease research foundations, and academia.

It’s Ridiculous to Say This Unleashes Untested Drugs

Critics similarly attempt to frighten the public by claiming the Cures Act “would pressure the FDA to approve new antibiotics, antifungals, and possibly other drugs through a new and faster pathway called the ‘limited population antibacterial drug’ pathway. Through this expedited pathway, new medicines would be approved and labeled for use in patients with ‘unmet medical needs,’ even though these drugs had never been tested in these specific patient populations.”

However, if you compare that synopsis with the statutory language, you’ll see that this doomsday warning is also unfounded. The Cures Act specifies that the limited population antibiotic pathway is only available where a medical condition is serious or life-threatening and there is an unmet need. The Cures Act further requires the FDA to consider “the benefit-risk profile of such drug” by considering “the severity, rarity, or prevalence of the infection the drug is intended to treat and the availability or lack of alternative treatment in such limited population.”

Thus, contrary to the frightening portrayal of the limited population pathway, the FDA is not about to unleash untested and harmful antibiotics on the public at large.  Rather, the pathway provides real hope to those suffering from serious or life-threatening bacterial infections that cannot be treated because of resistance to currently available treatments.

Individuals like Shalini Zachariah Mendelsohn, whose battle with an infection known as MRSA the Heartland Institute highlighted earlier this year. Mendelsohn suffered from a chronic sinus infection caused by a highly resistant strain of MRSA. None of the FDA-approved antibiotics proved effective. Mendelsohn found a cure halfway around the world in the Republic of Georgia, where she underwent phage therapy. Phage therapy uses a virus to destroy a bacteria. It is highly effective, but, as the CEO of San Francisco-based Phase International told Heartland, “FDA’s testing process stands between patients with treatable bacterial infections and bacteriophage therapy.” He added:

What needs to be done in order to make this highly effective antibacterial available is the FDA needs to change their testing model when performing clinical studies. Currently, they want to test phages-like antibiotics, a very long and expensive process. Humans and bacteriophages, which are ubiquitous in the environment, have coexisted since humans arrived on the Earth, and thus they are very safe.

Mendelsohn’s case is not unique. But it is impossible to know the number of people who suffer from antibiotic-resistant bacterial infections who could be cured by phage therapy because, as Roy pointed out in his piece, “[w]hen promising treatments are kept off the market, the patients who fail to benefit go unseen.” However, the Cures Act provides hope that we will find this out and, in my opinion, we’ll find it out soon when a plethora of new clinical phage trials begin, as well as more trials for traditional antibiotics.

This Is about Updating, Not Ending, Regulations

Critics respond by condemning as dangerous stories about individuals benefitting from unapproved drugs. That’s ironic given that opponents of the Cures Act are quick to cart out stories of past catastrophic drug failures, such as seen in the 1960s when pregnant women took thalidomide to combat morning sickness and ended up delivering babies with severe deformities, including missing limbs.

The Cures Act creates a necessary flexibility in the FDA review and approval process in light of twenty-first-century scientific advances.

These battling narratives, however, demonstrate the first key takeaway in this discussion: stringent regulatory standards harm patients by keeping promising treatments off of the market, but lax standards harm patients by exposing them to dangerous drugs.

But there is a second and more important lesson, which Crowley highlighted for me: The Cures Act does not adopt lax standards. Rather, it creates a necessary flexibility in the FDA review and approval process in light of twenty-first-century scientific advances. Crowley explained, “the scientific advancements in the twenty-first century are really remarkable, but the FDA’s regulatory structure is stuck in the twentieth century.”

Crowley also stressed that the Cures Act does not allow the FDA to ignore clinical trials in light of real-world evidence. Instead, it provides a framework for the FDA to review the totality of the data. “Consider,” he said, “when your doctor is deciding whether to prescribe a certain medication for you, the FDA label is helpful, but the doctor will consider many other things. He will look to his experience, the experience of other colleagues, other patients, and the results of other studies.”

The Cures Act gives the FDA this same broad focus. With the additional financial resources and more flexible hiring it provides, Crowley added, the FDA will be able to recruit the highly skilled experts needed to evaluate all of the evidence to assure that effective and safe drugs reach the patients who so desperately need them as quickly as possible. These provisions are not dangerous—the status quo is.

Disclosures: As the mother of a young son with cystic fibrosis, Cleveland has a vested interest in the continued development of drugs to treat and eventually cure this disease. Her family’s 401(k) and retirement savings include diverse holdings, including mutual funds in both the health care and pharmaceutical sectors; it also includes immaterial investments in several individual biotech stocks.