The British will be able to start receiving the vaccine within days. Their focus will be on protecting the frontline workers and the elderly; those in the highest risk groups. Those in the highest risk groups in the US, on the other hand, will be forced to wait.

On November 9th, Pfizer and BioNTech announced that their vaccine was over 90 percent effective. A press release is no substitute for the actual data from the study, and review of the data is necessary. They applied to the FDA for an Emergency Use Authorization (EUA) on November 30th.  An EUA allows the FDA to streamline the approval process in the case of an emergency, or if running a clinic trial is impossible.

The FDA released a notice in the Federal Register on Nov. 27, giving the public information about the EUA approval meeting. This provided the public with the opportunity to comment on the proposals being discussed, which is the normal process for federal agencies issuing regulatory changes. Finally, on December 10th, the FDA advisory committee will meet to discuss the EUA request. Upon reaching a recommendation, the FDA will choose whether or not to act upon it, which the FDA commissioner Stephen Hahn said in an interview would likely be within a few days.

According to Dr. Hahn, the FDA has been and will continue to review data and generate reports to prepare for the meeting during this time. In his interviews, Dr. Hahn’s description of the process implies that he believes that slow equals safe. But this is a false dichotomy. Rather than setting an arbitrary deadline for how long approvals should take to be complete, approvals should last until a rigorous analysis can be completed. How much additional data will we be able to gather by waiting a week longer than the UK?

The EUA means that this is an emergency. On average COVID-19 deaths amount to over 1,500 per day. For much of the year, excess deaths were even higher than COVID-19 deaths. Whether those deaths are undiagnosed COVID-19 deaths or deaths due to our response does not matter; they are a result of the virus and need to be considered when we are weighing the tradeoffs. We need to consider the risk of the vaccine and the risks of people contracting COVID-19 without the vaccine.

Unfortunately, the FDA has a long history of risk aversion when considering these tradeoffs. Former FDA Chair Scott Gottlieb detailed the culture of risk aversion at the FDA and its pitfalls. Vahid Montazerhodjat and Andrew Lo used Bayesian decision analysis to analyze the FDA approval stance. They also find that the FDA is too conservative, overweighing the risk of ineffective treatments and undervaluing the cost of rejecting effective treatments for severe diseases. This lengthens the process from discovery in the lab to use as treatment, which already sits at an average of over 15 years.

But this risk aversion and slow process is outdated in today’s rapidly advancing world. Technology is progressing at a rapid pace. It took researchers at Moderna just two days to design their vaccine in January, something unimaginable years ago. The rapid speed with which researchers are able to isolate a problem and design a solution tailored to it is only increasing as our technology improves. For example, just this week researchers released that they have solved the 50-year-old protein folding challenge, successfully cultured meat grown in a lab, and discovered a treatment to de-age and regenerate neurons.

Just 10 years ago it would have been impossible for so many people to seamlessly transition to working remotely. It should be no shock that science is seeing these improvements too.

Despite the rapid advances or science and technology, the FDA is still mired in an outdated regulatory process designed for the world of fax machines, not smartphones. The regulatory process is more suited for the pre-internet age, stifled by much slower data collection and the movement of information. We need nimble regulations that are able to keep up, rather than arcane and ritualized procedures.

Of course, you can’t shorten the time to conduct clinical safety and efficacy trials, but the UK allowed Pfizer to submit information on a rolling basis to shorten the approval process timeline. The FDA can do that to make it easier. The FDA could also end the requirement to fax or mail in hard copies of data.

The FDA also has a history of moving the goalposts, and requesting additional data from pharmaceutical developers, lengthening the approval process. After some time on the market, the Multiple Sclerosis drug Tysabri was found to increase the chance of progressive multifocal leukoencephalopathy and was voluntarily withdrawn from the market. Despite the FDA advisory committee recommending only a warning accompanying the package, the FDA required they create a risk map and limited the availability of the drug, which they didn’t require for Rituxan, which was in a similar situation.

Similarly, the FDA added requirements during the EUA process. In October, they announced a 2-month post injection review for side effects for 50 percent of participants. This forced Pfizer to wait an additional month to apply in order to meet the requirement. Eric Topol worked to convince the FDA to add this requirement to lengthen the process. The FDA’s decision was called “bureaucratic jujitsu” by a supporter. I’d call it bureaucratic obstruction which cost the lives of thousands of people forced to wait for the vaccine. Meanwhile patients in the UK are able to receive that vaccine.

The UK approval should inspire us to enact a reform that is far overdue and is a simple alternative that still keeps the protections of a regulatory agency but will allow for some speed. We should have reciprocity agreements with regulators in other countries. This would allow any drug approved for use in another country to be used by American patients. Some countries, like Australia and New Zealand, already take FDA approval into consideration during their approval process.

The U.S. government can limit reciprocity to countries that have a proven track record of approving safe and effective drugs. This would include countries in the European Union, Canada, Japan, South Korea, and Australia. This reciprocity would reduce delay and limit wasting time and effort applying to multiple regulatory bodies. Comparing the approved drugs in the UK and the US, researchers have found no significant difference in the safety between the countries, suggesting that MHRA approval is no less safe than FDA approval.

Ensuring that the COVID-19 vaccine is safe and effective is important, no one is arguing that. The question is how long a process is necessary, considering the continuing death toll. While the FDA abides by their bureaucratic process, the MHRA has acted quickly and approved the Pfizer vaccine, allowing people in the UK to begin to be vaccinated.

The FDA approval process, even in their Emergency Use Authorization, is a long process, taking time and costing us lives.

Conor Norris is a research analyst at the Knee Center for the Study of Occupational Regulation at Saint Francis University. He graduated from George Mason University with an MA in economics. 

Fortunately for patients tired of waiting for swift medical attention, health apps are providing solutions.

Health apps use smartphone technology to provide medical assistance or access to medical professionals. More than 318,000 health apps are currently available, and according to a national survey, 65 percent of smartphone users downloaded at least one health app and used it daily.

Unlike much other medical innovation and technology, health apps largely do not require FDA approval. The only exceptions are when the apps are considered to be medical devices or if they accompany medical devices.

Despite widespread use of health apps, their regulatory standing is under constant scrutiny. Some advocates of more FDA involvement and regulation point to the risk of unauthorized use of a patient’s medical information or the danger that apps will provide fraudulent medical advice.

Although these concerns are well intended, they are largely misguided. Health apps are providing patients with necessary services where they are underprovided.

Across the globe, health apps in China are providing widespread relief for rheumatic diseases.

Over 100 million Chinese citizens suffer from some form of rheumatic diseases, but only 5,000 rheumatologists are available to see patients. Further, many of these physicians practice in major cities, which may require rural patients to travel long distances and stay overnight in nearby hotels to receive care.

Fortunately, a health app developed through the Smart System Disease Management (SSDM) provides rheumatic disease patients with online consultations, which saves time and money. From February 2015 to June 2017 over 4,000 patients used the app for such consultations. The app also provided services cheaper, with online consultations costing nearly seven times less than travel and in-person consultations.

Similar to the United States, India faces a crippling physician shortage, with only 150,000 medical specialists serving nearly a billion citizens. Similar to China, access to an Indian physician can be time-consuming and prohibitively costly for patients residing outside urban areas.

In response to this problem, tech entrepreneurs Satish Kannan and Enbasekar D created the health app DocsApp. DocsApp links patients with a variety of medical specialists and to provide medical consultations within 30 minutes. “Around 70% of all health problems can be handled online,” Kannan told Forbes.

Since June, nearly 1,200 Indian physicians have provided over 400,000 consultations through DocApp. With over $2 million in proceeds, DocApp’s inventors hope it will become, “the world’s largest online hospital” and “help 100 million patients across India and the Asian markets,” Kannan says.

Health apps are not only providing access, they are also saving lives.

Earlier this year, North Texas limo driver Thomas Moran suffered a heart attack waiting for a client. Moran’s attack occurred with no warning and left little time to get medical attention.

Fortunately, he received swift medical attention and suffered no permanent damage thanks to the health app Pulsara. Pulsara links users with EMS teams, emergency departments, and hospital cardiac catheterization teams to provide immediate notice of a cardiac event. Grateful to be alive, Moran told a local television news program, “All I know is that they saved my life and I’m very happy to be walking around.”

Where the national goal to provide medical attention for a heart attack victim is 90 minutes, Moran received medical attention in 26 minutes.

In addition to providing consultations and saving heart attack victims, health apps are also being used to screen for pancreatic cancer, manage diabetes, track medications to treat glaucoma, predict epileptic seizures, detect concussions, and combat opioid addiction, among other uses.

Health apps are providing much-needed access and services to millions of patients on a global scale. Efforts to curtail their use, despite the best intentions, only work to deny access to those who may need it most. It would be folly to ask patients to wait in long lines or for federal regulators to verify quality when so many are receiving help every day.

The legislation they are attacking is Senate Bill 204. The bill would enable terminally ill patients to access experimental drugs without the FDA’s approval. Patients could receive access with the help of their doctor, consent from the drug producer, and approval by their state government approval. If passed, Senate Bill 204 would provide a decentralized method to prolong the lives of the terminally ill.

Since 2001, 38 states have passed right-to-try legislation. Last August, the bill passed in the U.S. Senate with unanimous consent. In the past few months, top political figures, including President Trump and Vice President Pence, have thrown their support behind the bill and called for the House of Representatives to act quickly and pass it. But widespread grassroots and political support now appears at an impasse.

It appears significant changes to the bill are likely if it is to pass through the House. As one article notes, “a change to the wording (or a new bill altogether) is inevitable.” Similarly, FDA Commissioner Scott Gottlieb is pushing for a narrowed definition of “terminally ill.”

But why now, after passing in red and blue states and bipartisan support from the Senate, is there such a pressing need to moderate the bill? There are many criticism of the proposed legislation. I find few of them are convincing and none of them a cause to dilute the bill.

Perhaps the most common argument against right-to-try is that the FDA’s compassionate use program largely serves the same purpose and approves most applications to access experimental drugs. I have written on the shortcomings of this approach here.

Another common criticism is that physicians do not know enough about experimental drugs to effectively supervise patients seeking right-to-try access. Thus, experimental-drug access may not be handled appropriately.

But this argument ignores that physicians and pharmaceutical companies commonly find effective uses for drugs without the FDA. So called off-label drug prescriptions–prescribing drugs for purposes other than their approved FDA use–accounts for about 25 percent of all prescriptions. This rate steeply increases when treating cancer and other terminal diseases.

Then there is concern that the bill provides legal protection for drug providers, which in the words of one CNBC article, allows drug companies to “circumvent the FDA and sell dangerous medicine to desperate people.”

The term “dangerous” is misleading. All drugs, whether approved or unapproved by the FDA, are potentially dangerous. According to one estimate, taking a daily aspirin is responsible for over 3,000 deaths a year. The relevant issue for policy makers, medical professionals, and patients is the relative risk of taking experimental medication.

But who would know whether the risk is worth taking better than the patient, the doctor, and the drug provider? The FDA, with all its expertise, doesn’t know why previous treatments failed or why other parties agree that unapproved mediation is worth a try. When the known alternative is certain death, we would expect patients to willingly accept a fair amount of risk.

Although chances may be slim, right-to-try has prolonged lives in the past. In 2012, Ted Harada was dying from ALS. His condition granted him access to an experimental procedure through Georgia’s right-to-try laws. In an unprecedented medical breakthrough, Ted was able to stop and reverse his ALS symptoms!

Not only was Ted’s recovery the first of its kind but, according to an article from the Washington Post, “Ted was told the treatment would not help him” and that “he was part of a Phase I safety trial, whose sole purpose was to prove the procedure would not kill him.” If Ted wasn’t granted access (very few were), he could not afford to wait an additional 9 years for FDA approval.

Lastly, some have raised concern that right-to-try access might be too expensive for terminally ill patients. In the words of Wayne State University Physician David Gorski,

A terminally ill patient could easily go bankrupt before he dies, and many couldn’t access experimental therapeutics through such laws in any event because they simply don’t have the money or the fundraising wherewithal to do so.

This passage highlights an important economic reality. The expensive and time-consuming process to have a drug approved can prohibit drug companies from engaging in efforts which do not impact their product’s ability to get approved. This includes opportunities for the terminally ill.

Consider the case of Josh Hardy, who suffered from a rare and deadly form of kidney cancer. In 2014, after receiving a serious inflection stemming from a bone marrow transplant, Josh’s physicians asked Chimerix Inc for experimental access to a drug they believed could help him. Unfortunately, Chimerix did not have the funds to provide experimental access and needed to devote its financial resources to the formal approval process. Josh was denied access twice.

Fortunately, a social media campaign by Josh’s family and coverage from major media outlets provoked the FDA to grant Chimerix, “an immediate initiation of a pilot trial.” Thankfully, Josh was eventually granted access.

Upon deeper inspection, it seems the conventional arguments against right-to-try come up short. But then why is the legislation under fire and likely to be revised? Unfortunately, the answer may be politics.

Consider remarks by Alison Bateman-House, an assistant professor in the Division of Medical Ethics at the NYU School of Medicine, who asked in her recent Forbes piece, “Why, then, do right-to-try advocates want to scapegoat the FDA?” Her answer? “Simple. Right to try was conceived by — and has been heavily lobbied by — an antiregulatory libertarian group.”

Many are making similar comments. A Los Angeles Times piece entitled “Right-to-try Laws are Hazardous to Your Health–and Now They’re Backed by the Koch Brothers” similarly states:

On the surface, right-to-try laws look like humanitarian relief from regulatory barriers for last-gasp treatments. Under the surface, however, they’re insidious. They don’t offer patients any legal or financial safeguards, they don’t improve access to drugs, and their real goal — here’s where the Koch brothers come in — is to weaken drug regulations in a way that will hurt every American

When attempts to criticize right-to-try legislation are unconvincing and efforts to defend current legislation flounder, playing politics becomes the only strategy left. Unfortunately, recent delays and attempts to curtail recent legislation may be a sign it’s working.

That day the American College of Cardiology (ACC), working with the American Heart Association (AHA), released new guidelines regarding what constitutes high blood pressure. Since 2003, a reading below 140/90 was considered normal. Now, any blood pressure over 120/90 is considered hypertension.

These changes mean nearly half of Americans have high blood pressure, a considerable increase from about 30 percent under the old guidelines. If hypertension rates before these changes were described as an epidemic and a silent killer, the new rates will likely replace the silence with alarm and calls to action.

As a first call to action, authors from the AHA and the ACC also provided new recommendations monitor and treat hypertension, which include emphasizing a “team approach,” stress management, self-monitoring equipment, more educational materials, and drastic dietary changes.

But how are these changes going to be implemented? Historically, efforts to combat public health problems eschew competition in the market in favor of using the political process.

These most recent changes are no exception. Both the ACC and the AHA openly advocate using government to advance their interests. According to the Center for Responsive Politics, in 2016 both organizations spent just under $3 million to lobby the federal government.

Whenever government works with special-interest groups to set industry standards it is important to examine the underlying incentives. When government imposes more stringent health standards, more resources are devoted to meet them. This can include more physicians, medical devices, prescriptions, dietary products, and other health-related goods. This is lucrative for many healthcare professionals.

Perhaps more importantly, we should ask: How successful has government been in advancing health in the past? Answering this question reveals a repetitive, and concerning, pattern.

In 1998, the industry standard for what constituted obesity was also changed per the recommendation of the National Institute for Heart, Lung and Blood Institute. In this case, obesity rates adjusted for older populations, which typically weigh more, were eliminated. The result was a swift rise in obesity rates and the arrival of the obesity epidemic.

Widespread concern over America’s growing waistline prompted a call for government action. In the words of University of California, San Francisco, pediatric endocrinologist Robert Lustig, “These [efforts to curb obesity] are things that have to be done at a governmental level, and government has to get off its ass.”

Government did. The results were widespread, invasive, and costly policies.

Among a few of the policies stemming from the obesity epidemic were regulations limiting portion sizes, taxing sugary foods, banning fast-food restaurants in some low-income areas, and mandating required nutrition labels. The government also began funding costly educational programs. In 2014, the Division of Nutrition, Physical Activity, and Obesity, which was started to deter obesity, accounted for nearly $48 million in federal spending alone.

For all these efforts, little progress has been made. From 1999 to 2016, total obesity rates increased about 9 percent. Further, evidence indicates obesity policies may work to promote obesity rather than prevent or reduce it. Research finds taxing fast-food restaurants leads to over-consuming fatty foods at home and taxes on sugary foods do not reduce their consumption enough to motivate weight loss. Both may work to worsen dietary choices. Evidence that nutritional labels help consumers make health-conscious choices is mixed at best.

Evidence that special-interest groups benefit from these policy changes is, on the other hand, very strong. As of 2009, the medical costs of obesity were estimated at $147 billion. Previously non-existent, obesity medicine has become its own specialty. Medical schools, researchers, and other healthcare professionals receive large federal grants to study the causes and treatment for obesity.

With a newfound increase in hypertension rates, history may be poised to repeat itself. The incentive for special interests to use government to advance their goals remains the same. Government’s overreaching involvement in public health remains the same. Only the diagnosis changed.

To be fair, hypertension can be a serious if left uncontrolled and can result in a heart attack, stroke, and other life-threatening cardiovascular complications. But hypertension’s seriousness is all the more reason to question the incentives behind its changing standards and the use of government to prevent or treat it.

With history in mind, I think it’s fair to take recent call to actions with, ironically, a big grain of salt.

This is the situation the Food and Drug Administration finds itself in, according to the Washington Post:

The Food and Drug Administration has more than 700 job vacancies in its division that approves new drugs, and top officials say the agency is struggling to hire and retain staff because pharmaceutical companies lure them away.

“They can pay them roughly twice as much as we can,” Janet Woodcock, who directs the FDA’s Center for Drug Evaluation and Research (CDER), said at a rare-diseases summit recently in Arlington, Va.

(Sidney Lupkin & Sarah Jane Tribble, “Despite ramped-up hiring, FDA continues to grapple with hundreds of vacancies,” Washington Post, November 1, 2016.)

As I’ve discussed before, the FDA is not short of money. On the contrary, its budget for drug approvals has increased significantly over the years. However, one reason it cannot hire enough staff to review new drug applications is that its hiring process is too slow.

If the agency cannot hire regulatory staff efficiently, how will it ever process drug approvals efficiently?

The fundamental problem is that the FDA is a monopoly, protected by government. Its staff do not suffer if new medicines and devices are not approved in a timely manner. Rather, patients, investors, and innovators suffer. The FDA has lots of reason to complain, because that is how it increases its budget.

However, it has no incentive to become more productive or efficient in approving new therapies. A bigger budget just makes the FDA bigger, but not better. Patients need more freedom to use new therapies without having their access strangled by the FDA.