Life Science Compliance Update

March 12, 2018

US Health IT Policy Lags Behind

Health Data

As described in a recent opinion article, Health IT Now and the Bipartisan Policy Center convened a work group of organizations representing clinicians, patients, hospitals, and technology companies to assess the current regulatory landscape, identify the most pressing needs of users, and develop consensus on the ideal future role of government in a post-meaningful use era, and a rapidly evolving delivery system and technology environment. Their report can be accessed and read here.

“For too long, federal regulation of health IT favored reporting and process over care and treatment. HHS must create a new era in which government rules provide tangible benefit to consumers; doctors see patients, not computer screens; and the private sector propels our health care system into the future without fear of an outdated regulatory framework holding us in the past. We have envisioned a system that is suited for the health IT challenges and opportunities of tomorrow. We want to make sure Washington sees that vision, too,” writes Janet Marchibroda, director of the Health Innovation Initiative for the Bipartisan Policy Center, and Joel White, executive director of Health IT Now.

Report and Recommendations

The work group members came together with the common understanding that while robust specifications were helpful in the early stages of HITECH implementation, over time, the level of prescriptiveness regarding health information technology (IT) contained within the Centers for Medicare and Medicaid Services (CMS) Electronic Health Record (EHR) Incentive Programs, CMS’ Merit-based Incentive Payment System (MIPS), and the Office of the National Coordinator for Health Information Technology (ONC) Health IT Certification Program, have contributed to dissatisfaction and increased burden among technology users and developers.

As a result, the work group’s report made several recommendations. Broadly, they encourage the federal government to provide assurances that protect consumer protections while leaving the evolution of products to the private sector. Additionally, the federal government’s role should continue playing a role in non-regulatory ways, like funding research to identify successful practices and adopting standards within health IT to signal government support.

The work group agreed on the following key principles for an ideal oversight framework for health IT and digital health:

  1. Encourage innovation by being flexible, technologically neutral, and not overly prescriptive; encouraging good development processes, rather than specific features and functions; supporting minimally necessary standards and baseline protections; and avoiding creation of unreasonable barriers to entry.
  2. Be risk-based, assuring that the level of oversight is based on the risk of harm to patients.
  3. Be stable and predictable, meaning that any changes must be implemented with sufficient notice and not create or add to uncertainty.
  4. Be accountable to the public and enforceable, by gaining considerable input, making performance transparent, and assuring enforcement, as applicable.
  5. Reflect the principles of a learning health system, by undergoing continuous improvement and innovation and embedding best practices as new knowledge is captured through experience.

The oversight framework should address the following six technology outcomes:

  1. Interoperability. Technology should facilitate interoperability and information sharing, which play a critical role—along with other technology outcomes—in advancing higher quality, more cost-effective, patient-centered care.
  2. Usability. Technology should reflect evidence-based, user-centered design principles; human factors science; and best practices. It should not create unnecessary burden on end users. It should be culturally competent, enabling access by users with diverse languages and abilities.
  3. Safety. Technology should not create patient harm. Instead, it should help reduce patient harm by supporting the delivery of safer care.
  4. Security. Technology should assure that information is available and accessible only to authorized individuals and processes and also provide assurance that information is not altered or destroyed in an unauthorized manner.
  5. Patient Access to Information. Technology should enable and not create barriers to patients’ access to their own health information.
  6. Support for an Evolving Health Care System. Technology should be adaptable and flexible enough to meet the changing needs of users and an evolving health care system.



April 21, 2017

Dr. Stossel Corrects a Common Misconception


Thomas P. Stossel, MD, MD (Hon), is a visiting scholar at the American Enterprise Institute and professor emeritus at Harvard Medical School, who has recently published several articles on how to remove barriers to medical innovation, and how medical innovation actually happens. This article highlights the impressive research by Dr. Stossel, supporting his position that private investment does much more to push the progress of medicine along than people think.

One article, published in the Wall Street Journal on January 5, 2017, addressed the assumption that “the root of all medical innovation is university research, primarily funded by federal grants.” He noted that the assumption is incorrect and that it is the “private economy, not the government,” that “actually discovers and develops most of the insights and products that advance health.”

The article opens with complimenting Congress for passing the 21st Century Cures Act, claiming that it “will promote medical innovation,” while at the same time telling readers to be “wary, however, of the $4 billion budget boost that the law gives to the National Institutes of Health.” In addition to his Wall Street Journal article, Dr. Stossel wrote a more in-depth article in National Affairs, arguing the same points, with more research and information embedded into the article. 

There were few findings in medical science that could significantly improve health until the late 19th and early 20th centuries, with innovation primarily coming from “physicians in universities and research institutes that were supported by philanthropy.” Dr. Stossel notes, however, that things changed after World War II when the National Institutes of Health became the major backer of medical research, changing incentives. Universities that previously lacked research operations started to develop them, and existing programs were largely expanded. As noted in Dr. Stossel’s article in National Affairs, “for decades, Congress allocated generous and growing funds to the NIH that enabled it to provide many research grants to universities. As a result, universities expanded their laboratory facilities and research faculties — and the government-academic biomedical complex, or GABC, was born.”

Since that time, improvements in health have rapidly occurred. Also during that period, funding for the National Institutes of Health has lagged behind the growth of an aging population in need of medical innovation while private investment in medicine has largely kept pace with the aging population and “is the principal engine for advancement.”

In his National Affairs article, Dr. Stossel discussed research papers submitted for publication, noting:

Although revered by academics as a quality filter, “peer review” of research papers submitted for publication (and of grants for research funding) is a flawed enterprise. As scientific journals found success in providing researchers the priority and credit they were looking for, the volume of submissions began to exceed the supply of journals’ publication space. The practice of peer review — having selected experts render opinions regarding the quality of articles submitted to journals — was designed to solve that problem. Today, electronic publication has eliminated the space problem, but a prestige hierarchy of journals has replaced it with a false scarcity. Researchers covet attention in the most prestigious journals, and the high-profile journals sustain their elevated status by arbitrarily rejecting the majority of articles submitted to them. The monopoly power of these journals, fueled by researchers’ vanity, allows indifferent editors to delay decisions about whether to publish research articles until dueling authors and reviewers come to a resolution. The referees of these disputes provide a quality of service that would be expected from the nature of the reviewers: anonymous, unpaid cronies or competitors of a paper’s authors. As a result, research data can languish in obscurity for months or years while authors work their way down the prestige pecking order and finally obtain a place to publish.

According to Dr. Stossel, more than 80% of new drug approvals originate from work solely performed in private companies and such drug approvals come on average 16 years after the beginning of clinical trials, which typically cost $2.5 billion from start to finish. Therefore, it appears even if academics and NIH really wanted to create a new drug, economic reality would get in the way.

The National Affairs article notes that, “achieving innovation requires wanting to innovate more than trying to impress reviewers of research papers or grant applications. It involves trial-and-error efforts that academic-review committees dismiss as “fishing expeditions” and that violate the scholarly premium on ‘hypothesis-driven’ studies. Success in academe also demands sticking to one’s research ‘brand.’ By contrast, innovation usually requires shifting gears to employ different technologies and experimental approaches. Such inconsistency reliably leads grant-application reviewers to discount an applicant’s qualifications.”

Dr. Stossel closes his Wall Street Journal article by stating:

Despite its exaggerated role, basic research in universities does advance human knowledge, train scientists, and contribute to medical advances—albeit uncommonly and inefficiently. But the system is unsustainable. A better approach would be to encourage academics to join with industry, where the financial resources and drive to innovate reside. Unfortunately, the biomedical complex demonizes corporations. If academic institutions stopped demeaning the activities needed to develop medical products, industry might take a greater interest in supporting their research.

Great advances in health care have been made, but there are still important challenges, from obesity to dementia. One step toward addressing them would be for Washington to adopt the right approach to medical innovation—and to stop simply throwing money at the current inefficient system.

April 18, 2017

Will There Be an Uptick in FDA NDA Approvals This Year?


Late last year, John Jenkins, director of the FDA’s Office of New Drugs told attendees at an event that the decline in new drug approvals (NDAs) was not due to a shift in FDA standards or policies. The number in 2016 (22) is remarkably lower than the total in 2015 (45). Could there be changes in 2017?

Report from event: Uptick in 2017?

Regulatory Focus reported from the Prevision Policy conference, quoting Jenkins directly: “There are fewer applications in front of us to act upon,” Jenkins said, noting that although he cannot discuss individual applications, a handful of the complete response letters (CRLs) issued in 2016 were due to good manufacturing practice (GMP) deficiencies and the need for FDA to conduct inspections. But Jenkins did say that there has been an uptick recently in the number of applications received, meaning the number of approvals could increase in 2017.

Recent news of an FDA approved drug to treat Parkinson’s disease lends some evidence to that claim. The drug, Xadago (safinamide), is an add-on treatment for patients with Parkinson’s disease who are currently taking levodopa/carbidopa and experiencing “off” episodes. An “off” episode is a time when a patient’s medications are not working well, causing an increase in Parkinson’s symptoms, such as tremor and difficulty walking.


Introduced by Rep. Larry Loudermilk (R., Ga.), the Modern Employment Reform, Improvement, and Transformation (MERIT) Act, H.R. 559, would make it easier to “drain the swamp” by removing federal employees for poor performance or misconduct. The MERIT Act allows for due process: It requires notice in writing to the employee in question from the head of an agency and provides an opportunity to respond with an appeal. The Merit Systems Protection Board is required to issue a decision within 30 days of the appeal. 

The National Review suggests this Act could be quickly applied to the FDA. They argue: “In recent years, in both the formulation of policy and the evaluation of individual products, the FDA has made egregious errors and arbitrarily expanded its authority in extra-statutory ways that have had important consequences. Most of these missteps have been in the direction of excessive risk-aversion or heavy-handed regulation, although a few, such as oversight of herbal dietary supplements and compounding pharmacies, have been marked by laxity, timidity, or outright incompetence.”

It is unclear if this would increase the success rates of NDAs, but indicates the close intersection between the political environment, FDA policy, drug pricing, and even the regulators themselves.

Scott Gottlieb’s Impact?

President Trump’s nomination for FDA Commissioner, Scott Gottlieb, could result in faster approval of drugs as reported by the Hill. Gottlieb could introduce a new regulatory paradigm focused on competition to accelerate innovation, dramatically shorten the time from development to patient access, and sharply reduce the prices for new drugs. Rather than settling for the status quo that rewards delayed access and excessive caution, he can promote early access and fast learning.


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