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Proposed Drug and Device Laws Should Be Pushed to 2017

The Healthcare Blog - Thu, 07/14/2016 - 10:29

Senate leaders now say they won’t consider companion legislation to the House-passed 21st Century Cures Act until September, after months of delay.  Lawmakers would then have to reconcile the differing House and Senate versions, presumably by year’s end during a lame-duck Congress.

We believe the summer delay is a good thing, and that Congress should actually extend consideration of the complex legislation into 2017 when must-pass FDA funding through industry user-fees will be on the congressional calendar.   That way, lawmakers can debate the implications of the proposed bills in the context of the resources FDA needs.

Why further delay?  Because the legislation—which makes substantial changes to the way the Food and Drug Administration (FDA) approves drugs and devices—is flawed.  As currently crafted, it lowers standards for drug and device approvals and safety, and risks adding to the rising cost of prescription drugs.
The ostensible rationale for the legislation—being pushed by drug and device companies—is that the FDA stifles innovation and advances in treatment by approving drugs and devices too slowly compared with other countries.

That premise is faulty.  Nearly two-thirds of the novel drugs approved in 2015, for example—29 of 45, 64 percent — were approved in the United States before being approved in any other country. The proportion was even higher in 2012 and 2013. Moreover, the majority of those drugs (60 percent) took advantage of existing FDA expedited review programs—fast track, breakthrough, priority review, and accelerated approval—and nearly half (47 percent) were approved to treat rare or orphan diseases.

As for devices, research shows that “it takes the same amount of time or less for patients to gain access to innovative, high-risk medical devices” in the U.S. compared to Germany, France, Italy, and Britain.

The House and Senate bills ignore the above facts. They essentially seek to speed-up the approval process by relaxing FDA’s safety and effectiveness standards. And to make that more palatable, sponsors have attached the changes to increases in funding for the National Institutes of Health and the FDA.
But while the public supports increases in biomedical research funding, it is deeply skeptical about lowering the standards for drug and device approval. In the most recent poll on this issue, just under 60 percent of Americans opposed changing federal regulations to speed the development and approval of drugs, according to a STAT-Harvard poll released in May. Respondents’ main concern: faster approval would allow products on the market that don’t work or are unsafe.

The survey echoed a previous poll by Consumers Union that found 82 percent of Americans believe that preventing safety problems is more important than limiting safety testing to speed the clearance or approval of devices or promote innovation.

The House’s 21st Century Cures Act received broad bipartisan support in part because it put a significant amount of money for research on the table. The House bill pledges a $9 billion increase in mandatory funding for the National Institutes of Health over five years, gaining the support of universities and medical schools. It also promises $550 million to the FDA (which, according to the CBO’s analysis, would not fully pay for the additional workload the Act assigns the FDA).

The drug and device industries intensely lobbied House members to pass the legislation. The Pharmaceutical Research and Manufacturing Association (PhRMA) increased its lobbying from $4 million to $5.4 million in the quarter before the 21st Century Cures Act passed. The Advanced Medical Technology Association upped its spending from $550,000 to $740,000 in the same quarter. The Senate’s lobbying database listed more than 1,100 lobbyists working on the legislation.

The Senate legislation—actually, 19 separate bills—is an improvement over the House’s 21st Century Cures Act.  But, on balance, the Senate bills are still weighted heavily in favor of speeding medical products to market by weakening FDA approval standards.

New drugs and devices are often an improvement over existing products, of course.  And when they clearly are, FDA has established pathways to get them to market and patients as fast as possible. The agency, for example, grants more than one-third of requests from industry for “breakthrough” designation for new drugs. But the history of medicine is replete with examples of drugs and devices that caused more harm than good, some of which were approved too hastily — such as Avastin for breast cancer, Vioxx for arthritis, and metal-on-metal hip implants.  Innovative drugs and devices simply must be required to actually work and not harm patients.

Below we list the Senate bills that, in our view, increase risks to patients and those we think would improve public health.
Increases risks and should be rejected or significantly modified:

The MEDTECH Act would prevent the FDA from collecting adverse events due to flawed electronic medical records, and from recalling certain types of defective medical software. Some of this software has had life-threatening flaws in the past, such as oncology electronic medical record systems that calculated and recorded incorrect drug dosages for highly toxic chemotherapy drugs.

The PATH Act would allow antibiotics to be approved with minimal evidence of safety and effectiveness through a “limited population” approval pathway. But, antibiotics approved in this way are promoted by companies so that they are more widely prescribed in order to increase sales. As they are, we won’t have information about whether they’re actually safe or effective for those groups of patients.

The Advancing Breakthrough Devices for Patients Act would encourage shorter and smaller clinical trials for medical devices. Abbreviated clinical trials will make it difficult, if not impossible, to include sufficient participation from subpopulations such as women, seniors, and racial and ethnic minorities in the analysis of the trials. In an increasingly diverse America, this is unacceptable.

The Advancing Hope Act would continue the existing pediatric priority review voucher program through 2022. The program is currently set to expire at the end of September. The program’s ability to stimulate innovation is questionable: a recent GAO review of the program concluded that the six drugs for which vouchers have been awarded so far were in development before the program existed. By allowing drug makers to buy a priority review, the bill removes FDA’s ability to set its work priorities and resource allocations based on public health needs. In a time of threats such as the Zika virus, our government agencies must be able to prioritize public health, and not be bound by vouchers that were sold to the highest corporate bidder.
Would promote innovation and protect public health, and should be passed:

The Preventing Superbugs and Protecting Patients Act will help prevent drug-resistant infections from contaminated duodenoscopes and other reusable medical devices that have caused harm and deaths. If enacted, this bill will require certain reusable medical devices to have validated cleaning, disinfection, and sterilization procedures.

The Next Generation Researchers Act  invests in the brightest young researchers to ensure that the United States remains at the forefront of biomedical research.

The Promoting Biomedical Research and Public Health for Patients Act reduces unnecessary administrative burdens on researchers and encourages compliance with reporting requirements.

The FDA and NIH Workforce Authorities Modernization Act will make it easier for FDA to recruit and retain top scientific and technical experts by making salaries more competitive with those offered by industry. It will also lead to the development of standards for regenerative medicine — such as regenerating human cells, tissues, or organs to restore or establish normal function.

Lawmakers are also considering adding the REGROW Act to the Senate’s package of bills. The bill would allow complex regenerative medicine therapies to be conditionally approved based on preliminary evidence. Since at least half of all drugs fail in the last stage of testing, many patients could end up receiving therapies that are later found to be unsafe or ineffective.
Looking Ahead

On Saturday June 25, six former FDA commissioners from Democratic and Republican administrations suggested at the Aspen Ideas Festival that Congress make the agency independent of the Department of Health and Human Services — similar to the Securities Exchange Commission, for example. With regulatory purview over products that represent a quarter of the U.S. economy, the group said the FDA is harmed by an unstable federal budget process and persistent political meddling. The group said they would issue a white paper on their proposal for the next administration.

That’s another reason why Congress should postpone consideration of this legislation until 2017.

A version of this post was previously published on the Health Affairs blog.

Paul Brown is Government Relations Manager and Tracy Rupp is Director of Public Health Policy Initiatives at the National Center for Health Research.  Steven Findlay is an independent health care journalist and consumer advocate.

Categories: OIG Advisory Opinions

From Takeoff to Touchdown, It’s About Engaging the Patient

The Healthcare Blog - Thu, 07/14/2016 - 00:00

                                   SPONSORED CONTENT

In my personal time away from my role at Deloitte, I am a private pilot and passionate volunteer for a charity that facilitates free air transportation for children and adults with medical conditions who need to get to treatment far from home.  In my interactions with these patients I hear how important communication is to their well-being. I also hear how outreach from life sciences companies enables improvements in their lives and puts them back at the center of the health ecosystem.

It is not controversial to say that patients must be at the center of the current and the future healthcare ecosystem; however, it may be to admit that today they are not well served by this ecosystem. The need to enable and effectively support care coordination across health care professionals (HCPs), providers, and other care team members to drive effective and appropriate use of pharmaceutical products is something I hear my clients in health care asking for on a daily basis. My clients ask for strategies that will enable them to build loyalty through extraordinary execution of their patient engagement programs, and through that loyalty drive adherence and better health outcomes. This ability to deliver consistent, high-quality, reliable service across all channels of engagement is a game changer. Recently Deloitte Consulting leaders discussed this subject on a webinar for our clients.

When these programs are well implemented they provide transparent information to build trust, while anticipating how to proactively respond to patients’ and their care team’s needs.  Insight and strategic intelligence enable our clients to demonstrate measurable benefits of their treatments and to provide guidance about how cost aligns to outcomes.

So what does a great patient engagement program platform look like? It provides consistent high-touch interactions across channels (e.g., companion app, portal, phone, text, and chat).  It enables the care network to collaborate with HCPs, families and other care team members. It provides a vehicle for internal and external data analysis to bring unique insights into patient treatment and interactions and demonstrate real-world evidence. All it does all of this while aggregating patient data from multiple sources and connecting both pharmaceutical and external vendor data to create a single view of patient interactions. All of these capabilities are easily empowered by a cloud-based, scalable technology platform like Salesforce Health Cloud. In fact, Deloitte Digital announced our approach to the market on March 2 of this year and will soon be making another major announcement as our platform advances.

In my personal interactions with the patients I fly, I can see the importance of a well-defined patient engagement program that feeds real-world evidence and intelligence back into the product-development lifecycle. This output data of the health care system is the key to creating a learning loop that will enable life sciences companies to continue to make quantum leaps in gaining the best possible outcomes from current products and feeding the funnel to find new treatments for these families.  Hopefully this means better results for patients and fewer flights for me.

Chris Zant is a Principal in Life Sciences for Deloitte Digital.


Categories: OIG Advisory Opinions

Risk-Factor Based-Medicine and Its Discontents

The Healthcare Blog - Tue, 07/12/2016 - 14:16

If concepts could get awards, then “risk factor” would surely be a Nobel prize winner.  Barely over 50 years of age, it enjoys such an important place in medicine that I suspect most of us doctors could hardly imagine practicing without it.  Yet, clearly, the concept is not native to our profession nor is its success entirely justified.

A few years ago, on the occasion of the risk factor’s fiftieth anniversary, my colleague Herb Fred and I published an editorial highlighting some of the problem with the use of this concept.  I will summarize here some of those points.

The risk factor concept was developed in the first decades of the twentieth century from within the life insurance industry as it began to systematically apply statistical methods in order to optimize actuarial predictions.  The idea was to identify which baseline characteristics held by individuals would correlate with future risk of death.

The Framingham investigators imported this idea into the public health sphere and introduced the term risk factor in the medical literature in 1961.  From then on, the concept and term have enjoyed an unmitigated success.

Originally confined to the cardiovascular arena, the concept is now thriving in every single medical discipline.  The diagram below is from a 2005 paper by Brotman et al.   The authors found that close to 1,200 papers per year claim new knowledge about “independent” risk factors.  In the field of cardiovascular medicine alone, 105 such risk factors have been identified.


–Risk factors, which can only make statistical claims, have morphed into full-fledged diseases.  For example, hypercholesterolemia is not a naturally identifiable entity but a construct established by fiat on the basis of arbitrarily chosen cut-off numbers.  Yet its disease status is widely embraced and it is sanctioned by an ICD code.

–The “diseasification” of risk factors has potential to cause harm by virtue of the so-called labeling effect.  For example, it is well established that the incidence of headaches can be increased simply by affixing a label of hypertension to a person.  Work absenteeism is also higher after patients are told they have high cholesterol levels.

–Risk factors garner plenty of attention even if their potential contribution to clinical outcomes is minuscule.  One example is hypertriglyceridemia, whose independent contribution to the risk of cardiac disease is still debated.  Obesity also barely increases cardiovascular risk when considered in isolation, and it may even be preventive in certain situations.  Such attention to trivial risk factors may benefit the public health sector and the pharmaceutical industry, but whether patients are actually served remains to be seen.

–The incidence of risk factors is further increased by the establishment of “pre” conditions such as pre-hypertension and pre-diabetes.  New risk factors can also be concocted by combining other risk factors, e.g., the so-called metabolic syndrome.  Many newcomers are “proto risk factors,” i.e., risk factors for risk factors. One example is “normal weight obesity,” which is a risk factor for the metabolic syndrome.

–Frequently, the relationship between risk factor and clinical outcome is not as straightforward as claimed.  For example, fatality rates for stroke have been in steady decline since the beginning of the twentieth century, long before any mechanism was available to identify or reverse its related risk factors.  Likewise, careful analysis of life insurance data shows that coronary mortality has been declining since the mid 1950’s, before risk factor prevalence started to fall.  Some risk factors, like hypertension, may have a “U-shaped” relationship to outcomes.

–Finally, as we now know from randomized clinical trials, risk factor modification is itself risky, as demonstrated by the trials of clofibrate, hormone replacement therapy, torcetrapib, rosiglitazone, to name but a few examples.

Risk factors have become the principal justification for turning medical care into a public health activity.  Using risk factor modification as a surrogate for quality of care, doctors are given incentives to treat patients according to statistical norms.   As we have shown in the paper, there is sufficient ambiguity to warrant caution against the widespread use of such a powerful concept.

Categories: OIG Advisory Opinions