13 minute read
The Luckbox Roundtable
from February/March 2023
Mark Cuban, the nation’s newest retail drug impresario, joins our expert panel to weigh in on the state of American pharma. Here’s what our panelists say lies ahead. (Luckbox lightly edited the responses for brevity and clarity.)
BY JAMES MELTON & JEFF JOSEPH
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PANELISTS
Dr. Mikkael Sekeres: CHIEF OF THE DIVISION OF HEMATOLOGY, SYLVESTER COMPREHENSIVE CANCER CENTER AT THE UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE. AUTHOR OF DRUGS AND THE FDA: SAFETY, EFFICACY, AND THE PUBLIC’S TRUST.
Mark Cuban: ENTREPRENEUR, VENTURE CAPITALIST, TELEVISION PERSONALITY, OWNER OF THE DALLAS MAVERICKS AND INVESTOR IN MARK CUBAN COST PLUS DRUG COMPANY.
Dr. Caleb Alexander: PROFESSOR OF EPIDEMIOLOGY AND MEDICINE AT JOHNS HOPKINS BLOOMBERG SCHOOL OF PUBLIC HEALTH WHERE HE SERVES AS CODIRECTOR OF THE CENTER FOR DRUG SAFETY AND EFFECTIVENESS.
Gerald Posner: INVESTIGATIVE JOURNALIST AND AUTHOR OF 13 BOOKS, INCLUDING PHARMA: GREED, LIES, AND THE POISONING OF AMERICA.
LUCKBOX: Only 25% of Americans view of the pharmaceutical industry favorably. What’s the biggest problem?
POSNER: It’s remarkable that positive ratings for pharma are declining after it came to the rescue on COVID-19 by developing lifesaving vaccines and therapeutics in record time. There’s no question the pharma industry stepped up to the plate when it was most necessary during the pandemic. Pharma has gotten a lot of bad press over the last few years over the opioid crisis and deserved it in many cases. If you ask the average person what they think about drug pricing, most will have heard about insulin prices. They’ve read an article saying insulin is too expensive and somebody is not eating the right food because of the cost.
CUBAN: Lack of transparency and trust. No one understands how prices are set and why prices always go up. That’s going to upset everyone and is why we made cost plus drugs transparent.
SEKERES: During the pandemic we saw science, drug development and regulatory science unfold in real time. But a lot of people in this country also saw science is not always a linear process. And we saw information that was wrong communicated about COVID. I remember how much time we spent washing our hands when COVID was just breaking, not realizing the importance of masking. And then you had frequent vaccines rolled out, and they were free. More recently, you’ve heard that the government isn’t going to subsidize this anymore. People are going to have to pay for it.
ALEXANDER: On the one hand, we continue to see enormous innovation within the industry and remarkable scientific breakthroughs brought to market. On the other hand, millions of Americans are burdened and, in some cases, devastated by the cost of prescription drugs. The modern pharmaceutical industry is a mixed bag. We have remarkable breakthroughs and scientific innovations. But for every blockbuster, plenty of other products are marginal innovations at best.
Government officials and executives at Twitter may have suppressed the Great Barrington Declaration (GBD), an open letter signed by scientists harboring “grave concerns” about COVID-19 lockdowns. Assuming that happened, was it harmful?
CUBAN: If information were suppressed, no one would have known about GBD. Everyone who supports GBD has a Substack account and a YouTube channel. All of them appeared on Fox and every other supportive platform—all of which reach more people than Twitter. What Twitter did by taking requests from the Trump administration and then Biden was a non-event.
POSNER: Censoring any group because you think that’s not the ‘truth’ at the moment when you’re not doctors—which they weren’t at Twitter —boy, that’s fraught with problems. If you want to make some idea go viral as a conspiracy theory—I’m telling you as someone who studied conspiracy theories—then tell one group they can’t have something. And that will be the one everyone thinks was the truth. So, it was problematic on so many levels. It’s unfortunate because the public airing of those disagreements is fine. Even during the pandemic, you’re going to get the experts at the government level at the FDA and others saying, ‘this is what we’ve decided, and this is what we’re going to do. And that’s what really matters.’ But it’s unfortunate that the Twitter files show the public had a right to have that information out there.
ALEXANDER: Unfortunately, we’ve learned the hard way that we were not as well prepared for COVID-19 as we should have been—as a country and as a society. There are a lot of lessons to learn from the pandemic, not the least of which is the threat misinformation poses to public health. Information and misinformation have been weaponized and used for political purposes or other purposes contrary to public health. If there’s one thing that we can learn from the pandemic, it’s the importance of investing in our public health system to ensure a more rapid, coordinated, and robust response to the next pandemic.
SEKERES: The basis for our understanding of science and the development of new therapies is transparency, communication and vetting ideas. If I submit an article to a scientific journal, it will undergo external review, where people will comment on it. Increasingly, scientists put their findings online and open them up to public comment before sending something to a journal. So, the basis of science is transparency, feedback from colleagues and eventually arriving at a source of truth. So, any censorship of that is ultimately going to hurt science.
What did we learn from Operation Warp Speed that might apply to the development of other vaccines?
ALEXANDER: When there is the right political will, and the forces are properly aligned, we can bring tremendous innovations to market and do so much more quickly than historically has been the case. There were many efforts by many individuals and organizations—and parts of the federal government—that allowed vaccine development to progress at the speed that it did. The speed with which the COVID vaccines were developed—and their remarkable safety and effectiveness—reflects what’s possible when there is the right momentum and alignment across many, many parties. So, that’s the main take-home for me. The whole drug development framework should take a close look at the achievements that were possible.
SEKERES: We learned the FDA could move quickly if adequately resourced. The FDA needs the resources to bring every drug for life-threatening diseases to market within a year. Similarly, we provided enormous government resources to some of the manufacturers of these vaccines to get the trials up and running. You had motivated medical centers and a motivated public enroll in these trials very quickly to get an answer.
POSNER: I would hope some lessons were learned and could be implemented into the future operating procedures. But I am a pessimist about that. The system responds best and fastest to a crisis. There’s nothing that could have made the FDA operate better than it did. Like everyone in Washington, the FDA responds to public pressure. And the pharma companies know that very well. That’s why they have mastered the art of creating patient advocacy groups for specific diseases. They find the most vulnerable of those patients to go to Capitol Hill and testify before Congress. Then Congress asks the FDA why aren’t [they] approving these drugs, and two months later something comes out.
President Biden has announced a “Cancer Moonshot” aimed at rebooting an Obama-era initiative to slice the cancer death rate in half within 25 years while improving the lives of cancer victims.
SEKERES: Increasingly, we’re recognizing that cancer represents thousands of diagnoses. Leukemia, for example, isn’t just one thing. It’s dozens or hundreds of diagnoses of leukemia. So, within those narrower and narrower subtypes, yes, we are going to cure some people. But are we going to eliminate cancer as we know it in 25 years? No. One big reason is that is, during the pandemic, a third of the workforce for conducting clinical trials left as part of the great resignation. It will be hard to cure cancer with a third of resources to support clinical trials gone.
POSNER: Biden’s goal is admirable. The problem is that the idea of curing cancer or slashing rates by half is an over-promise made by every president since Richard Nixon declared a ‘war on cancer’ in 1971. The moonshot metaphor, which Biden has relied upon for six years, is simply the latest in fifty years of presidents talking tough about cancer. Nixon was the first to use that moonshot symbolism. The problem is that the moonshot metaphor is too simplistic and creates unrealistic expectations for many patients. As Mikkael points out, medical researchers realized over time that cancer was a far more complex disease with many variations. There would be no single cure but the battle against it would be more nuanced and take much longer, with some victories and plenty of setbacks.
The fees pharmaceutical companies pay to expedite FDA approval of their new drugs are said to provide 50% to 75% of the agency’s drug review budget. Does that threaten the FDA’s independence?
CUBAN: I don’t think the FDA inherently plays favor ites. They know they are responsible for saving lives and no one at the FDA gains financially from their decisions. The challenge is the optics of it. There will always be people who criticize the economics of the pharma business. But that’s the reality of how we do things in the U.S. We don’t want to pay more in taxes, so we find other sources of revenue for our agencies.
POSNER: There’s no quid pro quo because of these expedited fees. Does that constrain FDA’s enforcement ability? It is possible to be an aggressive regulator and enforce the rules, notwithstanding where the funding comes from. As an investigative reporter, I’d prefer in a perfect world that half of the budget didn’t come from the industry you’re regulating. But if you take away that funding, someone must give more money to the FDA, and no one wants to do that.
ALEXANDER: The FDA typically does remarkably good work. It has many different mechanisms to keep industry at arm’s length. But there are also many cases where regulators failed to do so. It creates concern about the perception of conflicts of interest. It’s a complex issue. It’s not a plain vanilla matter. The federal government has yet to step up and say, ‘We want to provide this money so you don’t have to rely upon user fees.’
SEKERES: In the history of the FDA, some people were fired because they had conflicts of interest. However, in my interactions with the FDA, I met a dedicated team who wanted to do the right thing and thought about the public’s safety all the time. The only way to resolve this potential conflict of interest is adequate funding from Congress to the FDA.
Does the drug approval process need improvement? If so, how?
POSNER: The FDA is underfunded and overmatched. I’ve spoken to people working in different divisions in the FDA over the last few years—and this is pre-COVID—and they were drowning. They were trying their best to keep their heads above water. And they’re earning a government salary. They say somebody in the private sector often tempts them with two to three times the salary to jump out. And so, some of the best people are leaving while others stay. Do they need a larger budget? Absolutely. Yes.
ALEXANDER: On the one hand, the United States is at the front of the pack in innovation in the pharmaceutical marketplace. But enormous inefficiencies, costly delays and, in some cases, inadequate safeguards exist. There are reasons to look at what was done during the pandemic, learn from that, and further improve and refine our drug development system.
SEKERES: People who are in clinical trials can trust that they are receiving—at the very least—the standard of care for their condition. And researchers conduct trials safely and with a lot of oversight. There’s not any other country that does that better than we do. However, to get that sort of oversight, pharmaceutical companies often contract out the supervision of clinical trials to groups called contract research organizations, or CROs. There’s a perverse incentive in that the CROs’ bill is based on how much work they do. We have essentially incentivized them to put more regulatory infrastructure into trials. So, how we conduct clinical trials here in the U.S. is safe, but oversight has gotten out of control to the point where the clinical trials have become very costly, laborious and take a long time to complete.
What should the U.S. do to make drugs more accessible and affordable?
CUBAN: More transparency. Less dependence on insurance companies. I don’t think the interests of insurance companies and patients align.
SEKERES: One thing that’s done differently in Europe is regulatory agencies consider costs at the same time they’re considering approval. And if they’re not getting enough bang for their buck—if a drug isn’t prolonging survival in a meaningful way—they’re going to say, ‘Nope, we’re not going to pay a lot for that drug.’
What are your thoughts on Mark Cuban’s new Cost Plus Drugs platform?
ALEXANDER: We need innovation in pharmaceutical pricing. A lot of good ideas have been put on the table about how to lower drug prices. But unfortunately, there has yet to be the political will to adopt policies that get this done. [Cuban’s initiative] is a very important step. And I think the transparency of his model has broad appeal.
POSNER: To use a very hackneyed expression, critics of the industry say what is needed is out-of-the-box thinking. And Cuban has done that. There’s no question he’s taken a model that he thinks will keep prices competitively low for patients and, at the same time, be profitable enough to run as a business. Now, it sounds simple when you think of its essence, but it’s not. If it were simple, everybody would be doing it. But I think that if anybody can do this, it’s a person like Cuban.
SEKERES: It’s great to make medications affordable to patients. I know of at least one patient who is taking advantage of that. An important aspect of it is to make sure that the drug supply that’s going through that initiative is shored up. Part of the [Food and Drug Administration’s] job is to determine the relative balance of safety and efficacy of drugs—to get them to the U.S. population and to approve a drug for marketing purposes. The FDA also must ensure the consistency of drug manufacturing. And that can become harder when drug supply chains are outside this country. One thing I do know about the Cuban initiative is the drug pricing is awesome. That’s particularly true for the sorts of drugs that I prescribe—some of which might cost $120,000 a year.
