U.S. Patients Have Much Greater Access to New Cancer Drugs Than Others Do

New research by scholars at the University of Pittsburgh shows how much better access American patients have to new cancer medicines than their peers in other developed countries:

Of 45 anticancer drug indications approved in the United States between January 1, 2009, and December 31, 2013, 64% (29) were approved by the European Medicines Agency; 76% (34) were approved in Canada; and 71% (32) were approved in Australia between January 1, 2009, and June 30, 2014. The U.S. Medicare program covered all 45 drug indications; the United Kingdom covered 72% (21) of those approved in Europe— only 47% (21) of the drug indications covered by Medicare. Canada and France covered 33% (15) and 42% (19) of the drug indications covered by Medicare, respectively, and Australia was the most restrictive country, covering only 31% (14).

(Y. Zhang, et al., “Comparing the Approval and Coverage Decisions of New Oncology Drugs in the United States and Other Selected Countries,” Journal of Managed Care and Specialty Pharmacy, 2017 Feb;23(2):247-254.

21st Century Cures Act Shows Republicans Can Lead on Heath Reform

(A version of this column was published by Forbes.)

Since the election, there has been a lot of sturm und drang around what the alternative to Obamacare will look like. It looks like we can be highly confident the Republican-majority Congress will repeal Obamacare very quickly starting in January. However, there is some question about what exactly will be repealed.

Last year’s repeal bill, H.R. 3762 repealed Obamacare’s spending and taxes, but not its over regulation of health insurance. Further, Republican politicians have promised not only to repeal Obamacare, but replace it with a better payment system than existed previously. People’s primary concern about the previous system was that people in the individual market could be denied coverage for pre-existing conditions or charged higher premiums as a result of underwriting.

Despite Six-Figure Starting Salaries, FDA Can't Hire Reviewers

Would a starting salary of over $160,000 turn you off? Well, maybe if you had a scientific PhD and had to wait four months before the employer could decide whether to hire you or not, you would find a spot elsewhere.

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 applications is that it is too slow to process hiring.

If it 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.

Mixed News on Generic Drug Approvals

A response to expensive patented medicines is generic competitors. The U.S. has struck a pretty good balance between innovation and low prices through the Hatch-Waxman (1984) Act, which specified patent terms for newly invented medicines, and a pathway for generic competitors to enter the market after a period.

One obstacle to generic entry in recent years was a very slow approval process at the Food and Drug Administration. This led to a backlog, which was unexpected because one important benefit of Hatch-Waxman was that generic competitors did not have to replicate the expensive clinical trials innovative drug-makers had to carry out to receive the FDA’s approval.

The FDA’s Office of Generic Drugs (OGD) considers approving generic copies of drugs upon receipt of an Abbreviated New Drug Application (ANDA) from the manufacturer. The system changed under a law passed in 2012, the Generic Drug User Fee Act (GDUFA). Recent data show improvement:

Who Benefits From the "Right to Try” Experimental Medicines?

The Goldwater Institute has had great success getting states to pass “Right to Try” laws. Right to Try allows a desperately sick patient to take an experimental new medicine before the FDA has approved it.

Thirty-one states have passed Right to Try. Further, U.S. Senator Ron Johnson (R-WI) has tried to get a federal Right to Try law through the U.S. Senate. However, there has been push-back. According to Allison Bateman-House of NYU Langone Medical Center, “there is no confirmed instance of anyone getting a drug through Right to Try.” Jonathan Friedlaender, a survivor of advanced metastatic melanoma, has written a compelling essay in Health Affairs, which concludes Johnson’s proposed federal law would not improve access to experimental medicines.

The problem has two parts:

A Modest Proposal To Reduce The Price Of EpiPens

(A version of this Health Alert was published by Forbes.)

Posturing politicians on Capitol Hill conducted a hearing a few days ago, in which they grilled Heather Bresch, CEO of Mylan. N.V., which makes EpiPens. Prices of EpiPens have skyrocketed in the last few years. According to Aaron E. Carroll, writing in the New York Times, the real (inflation-adjusted) price of EpiPens has risen 4.5 times since 2004.

The politicians were more interested in wagging their fingers and tut-tutting at Ms. Bresch for the amount of money she has made, than actually figuring out a way to lower the price of EpiPens. (By the way, Ms. Bresch testified she has no intention of reducing prices in response to their badgering.)

EpiPen: Government is Part of the Problem

Much has been written about the dramatic price hikes for EpiPens, which inject a drug that counters severe allergic reactions (anaphylactic shock). According to Aaron E. Carroll, writing in the New York Times, the real (inflation-adjusted) price of EpiPens has risen 4.5 times since 2004.
Both Carroll and the Wall Street Journal have described how government has allowed EpiPen’’s manufacturer to hike prices so much. EpiPen is complicated, being both a drug and a device. The drug is very inexpensive, and not patented. The device is protected by patents issued in 2005, which expire in 2025.

First, the government made a couple of interventions in the market that allowed the manufacturer to raise prices above the free-market level. The federal government changed its guidelines such that the EpiPens have to be sold in packages of two (while customers might prefer just one, or at least an odd number). Also, the federal government gave public-emergency grants to states on condition they stockpile EpiPens.

Artificial Intelligence, Machine Learning, and the FDA

(A version of this column, with hyperlinks, was published by Forbes.)

In July, the Food and Drug Administration issued guidance on three topics important to the future of medical innovation. These welcome guidelines demonstrate the FDA is doing the best it can to ensure it does not interfere inappropriately with advances in medical technology that rely on processing information.

However, the guidelines also show the FDA will be limited in its ability to respond effectively to future innovations. Current law does not really define the FDA’s powers to regulate devices that depend on advances in artificial intelligence and machine learning, as applied to health care. Guidelines give medical entrepreneurs some comfort the FDA will not impose an undue regulatory burden on them, but they are no substitute for legislation precisely defining the FDA’s powers in the digital age.

Private Sector Vigilance, Not Government Regulation, Protects Patients

Last April, I cheered Arizona for passing a law allowing patients to order blood tests without a doctor’s prescription. The company that lobbied for the change, Theranos, was also interesting because it posted its prices at venues where patients could get blood drawn. It had a widely promoted partnership with Walgreens, which has a strategy of using new technologies to deliver more value-added services in the retail environment.

Since then, Theranos has gotten into trouble for being opaque about how it actually conducts its tests when the samples get back to its labs. This has led to turmoil in the business media and among investors which are interested in backing entrepreneurs with new approaches to lab testing.

Read more at NCPA's Health Policy Blog.

Reciprocal Regulatory Approval To Reduce Drug Prices

Senators Ted Cruz (R-TX) and Mike Lee (R-UT) recently introduced the RESULT Act, which would allow drugs and medical devices approved in certain other countries to be allowed in the U.S. as well. The countries included are European Union members, Israel, Canada, Japan, and Australia.

The benefits of this act would be significant. Professor Daniel Klein of the Mercatus Institute at George Mason University and Professor William L. Davis of the University of Tennessee at Martin have surveyed economists on this policy, and a majority agree it would improve patients’ access to safe and effective drugs.

Read more at NCPA's Health Policy Blog.

Jeb Bush Health Plan: Innovation and Patient Care

By avoiding sound bites and respecting voters’ ability to understand issues, Governor Jeb Bush’s health-reform proposal demonstrates strong leadership. Repeal and replace Obamacare? Sure, Bush is for that, but no Republican politician should win points simply by regurgitating what many citizens fear has become little more than a slogan.

What is especially impressive is that Bush leads with a non-Obamacare issue  – fundamental reform of the Food and Drug Administration (FDA): “It should not cost $1.2 billion to $2.6 billion nor take 12 to 15 years to advance a medicine from discovery to patients, but that is the case under the Food and Drug Administration’s current regulatory mess.”

Read the entire op-ed in The Hill.

Jeb Bush's Health Plan

Jeb Bush’s health plan is out – and it is very good. Bush leads with fundamental reform of the Food and Drug Administration. “It should not cost $1.2 billion to $2.6 billion nor take 12 to 15 years to advance a medicine from discovery to patients, but that is the case under the Food and Drug Administration’s current regulatory mess.”

In recent weeks, we’ve read stories about drugs that have been around for decades, for which prices have been hiked sky-high. These price hikes are carried out by executives taking advantage of obscure FDA rules that impede competition.

Read the entire entry at NCPA's Health Policy Blog.

Martin Shkreli A Creature of FDA Regulation, Not Pharma Industry Greed

Perhaps it is just unfortunate coincidence, but Hillary Clinton’s proposal to impose federal controls on prescription drug prices was perfectly timed to ride the public outrage over a hedge-fund manager turned pharmaceutical executive, Martin Shkreli, who raised the price of a  decades-old drug drug from $13.50 per pill to $750.

Mrs. Clinton lumps all nominally expensive prescription drugs into the same category of so-called “specialty drugs.” However there is a world of difference between Mr. Shkreli’s Daraprim and specialty drugs like Sovaldi and Harvoni. The latter are newly invented medicines that required many years and huge amounts of capital investment to achieve therapeutic advances for patients suffering Hepatitis C that radically increase their quality of life. They comprise intellectual property protected by patents, which allow the drug makers to compete without fear that their innovations will be copied immediately by manufacturers who made no comparable investment in R&D.

Read the entire column at Forbes.

Is FDA Reporting Drug Shortages Adequately?

For a number of years, there have been critical shortages of certain generic drugs for injection. These are often important cancer drugs. In 2012, I wrote a report that concluded over regulation by the Food and Drug Administration (FDA) was the primary cause of the shortages.

The President and Congress acted, but their actions did not result in improvement for over a year.

Today, the FDA claims to have improved the situation. However, an article in Health Affairs points out that the number of drug shortages reported by the FDA and the number reported by the University of Utah Drug Information Service (UUDIS), the leading private source of this data is diverging dramatically.

Read the entire column at NCPA's Health Policy Blog.

FDA Approves First Biosimilar, Still No Guidance on Names

The Food and Drug Administration has approved the first biosimilar therapy in the U.S.

There is a rough – but very imperfect – parallel between biosimilars and generic drugs, which now make up the vast majority of prescriptions filled in the U.S. The innovative biotech industry long argued that it was harder to make a generic biologic therapy than a generic drug, because the former is made from living sources, not just molecules.

Read the entire column at NCPA's Health Policy Blog.

FDA Allows Direct-To-Consumer Genetic Tests

One year after a very public squabble with genetic-testing company 23andMe, the Food and Drug Administration has decided not only to allow 23andMe to directly market its genetic test to consumer as a diagnostic device, but to free other genetic-testing companies from pre-market review or prescription status.

Read the entire column at NCPA's Health Policy Blog.

21st Century Cures: Waking Up Dormant Drug Therapies

Representative Fred Upton, Chairman of the House Energy & Commerce Committee, recently released a discussion draft of legislative language for the 21st Century Cures Initiative. This initiative attempts nothing less than to “boil the ocean” of regulations and incentives that govern medical innovation in the U.S. The 400-plus-page draft rolls up a number of previously proposed bills (including an updated version of Representative Marsha Blackburn’s SOFTWARE Act, discussed in a previous Health Alert).

A large share of the draft incorporates legislation designed to improve the incentives for inventing new medicines, or finding new uses for old medicines. This is important, because we are facing a crisis in pharmaceutical innovation.

One of these is the Dormant Therapies Act, put forward by Senator Orrin Hatch (R-UT) and Senator Michael Bennet (D-CO). This would give a “dormant therapy” 15 years of marketing exclusivity after approval by the Food and Drug Administration (FDA), significantly more than currently exists.

Read the entire column in Forbes.

Relax: The FDA Will Not Regulate Things That Increase The Flow Of Your Qi

Qi, as I understand it, is a term in Chinese culture that refers to the life force or natural energy that flows in living things and, perhaps, the entire universe. Many Americans know about it through Chinese traditional medicine or martial arts. If you are a fan of such practices, you will be glad to learn that the Food and Drug Administration (FDA) has recently announced that it has no plans to regulate items which claim “to increase, improve, or enhance the flow of qi” as medical devices.

Read the entire column at Forbes.

FDA Regulation of Mobile Health Apps Is A Real Threat

Do you want the Food and Drug Administration (FDA) to regulate your smartphone? Many in the booming digital health industry are indifferent to the risks of the FDA regulating apps as medical devices. As I noted in a recent Health Alert, the FDA’s current doctrine is to allow most new apps onto the market without regulation. However, this openness is defined only in rules written by the FDA itself, not legislation. Rep. Marsha Blackburn and others in Congress have proposed to amend the law to prevent the FDA from overreaching.

Entrepreneurs and patients who think that the FDA’s current posture is eternal should be aware of a campaign to regulate apps for health. Here’s a recent article from Mother Jones:

Read the entire article at NCPA's Health Policy Blog.

Drug Research And Its Discontents: Does It Really Cost $2.6 Billion To Develop a New Drug?

My previous column discussed the high cost of researching and developing a new pharmaceutical compound. I noted that the latest estimate, by the Tufts Center for the Study of Drug Development, is “controversial”, and promised to address the controversy. Well, here goes.

Read the entire column at Forbes.