Life Science Compliance Update

April 21, 2017

Dr. Stossel Corrects a Common Misconception

Research_Hero

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 12, 2016

JAMA: Research Parasites Wanted

As reported, earlier this year, a top medical journal ignited a backlash by referring to "research parasites", also known as people who did not personally run a clinical trial or collect data but "use another group's data for their own ends, possibly stealing from the research productivity planned by the data gatherers." The New England Journal of Medicine quickly backtracked, as we noted, and clarified its support of data sharing.

Four days after jointly submitting the editorial piece with Dan Longo, M.D., Jeffrey Drazen, M.D., stated the Journal's support and noted it is "committed to data sharing in the setting of clinical trials." He went on to comment that he believes "there is a moral obligation to the people who volunteer to participate in these trials to ensure that their data are widely and responsibly used" and that "researchers who analyze data collected by others can substantially improve human health." In the walk back, he concludes by bringing up data sharing through collaboration, signaling that such form of data sharing may be palatable to more than just the "data socialists" who want to take your hard-researched data a mere six months after the publication of your findings.

Now, research published in the Journal of the American Medical Association (JAMA) suggests there are not enough of these so-called data parasites. The study found that although more than 3000 trials are available to investigators through open data platforms, only 15.5% had been requested by a limited number of researchers. Most proposals focused on nonprespecified subgroups or predictors of response rather than validation of study results. The reasons speculated for underutilization of clinical trials data include lack of knowledge about these resources, possibly due to lack of publication of results from proposals, or lack of funding to support analyses.

In an accompanying editorial, the editors of JAMA argue that sharing data has two principle purposes: to verify the original analysis and hypothesis generation. "It has the potential to advance scientific discovery, improve clinical care, and increase knowledge gained from data collected in these trials. As such, data sharing has become an ethical and scientific imperative," they wrote.

They also outline that researchers intentionally or inadvertently do not always report important findings from their investigations, citing a report involving clinical data. However, offering all possible data available for reexamination and replication of analyses can help ensure that the publications have fidelity to the trial plan. Perhaps more important, they write, are the study participants. The article cites the International Committee of Medical Journal Editor's position that there is an ethical obligation to responsibly share data generated by interventional clinical trials because participants have put themselves at risk.

Increasing, data sharing is actually being mandated by trial sponsors and has been supported by numerous groups like the Institute of Medicine/National Academy of Medicine, European Medicines Agency, and ICMJE. Journals and funders, they write, must find common ground to ensure data sharing occurs, but note it is easier to call for data sharing than to actually create a system that protects the privacy of patients and is also efficient, effective, and fair to the investigators who collected the data.

The editorial explains that three issues must be addressed for such a system to be successful. First, the shared data must be deidentified for the protection of individual study participants. Second, a system of sharing data must be efficient. Finally, the system must be fair and respect the investment and contributions of the trial investigators.

June 29, 2015

The Importance of Private Investment into Research and Development

NIH

Recently, we covered Thomas Stossel, M.D.’s new book, Pharmaphobia: How the Conflict of Interest Myth Undermines American Medical Innovation. The distinguished Harvard hematologist and research fights back against the “conflict of interest movement” as one that chills medical progress and hurts the people who benefit most from industry research collaboration: patients. “Physician-industry interactions have been critical to the development of a large percentage of the medical products that allow physicians to prevent heart attacks, cure cancers, and restore mobility to the elderly,” he writes. Despite their importance to modern research and development programs, critics remain undeterred, but a recent report suggests private research—which often relies upon physician-industry relationships—is critically important to the United States’ economy.

R&D Growth and Hope for the Economy

Bloomberg Business reports that amidst concerns of a permanently slowing United States economy, private investment into research and development remains “hope on the horizon,” growing at the fastest pace in 50 years. Specifically, from November 2014 through March 2015, American companies funded R&D to a tune of $316 billion, around 1.8 percent of gross domestic product. This represents the largest share ever for the private sector.

One economist quoted believes additional R&D spending will in turn spark the creation of new technologies, encouraging businesses to invest in new equipment. This domino effect by boosting worker productivity may then create higher growth rates. If funding breakthrough technologies does not bring the United States out of its economic rut, the article asserts there simply may be no other way out.

But it takes decades for research spending and subsequent development to translate into new products, if it ever happens at all. This is compounded by the fact that private partnerships with government are diminishing, despite their historic success after World War II when industry turned the most promising advances from government labs into critical products today like GPS and the Internet. As Bloomberg notes, pharmaceutical companies are one example of an industry where a substantial share of revenue is invested back into R&D. Most companies have no choice but to keep spending or risk getting left behind from important breakthroughs.

The article recommends making the R&D tax credit permanent. Since its introduction in 1981, the credit has been modeled internationally. Now, over three decades later, the United States ranks 27th in terms of the generosity of its R&D tax credit. It is one reason domestic companies have boosted overseas R&D 2.7 times faster than what companies have spent inside the United States. This corresponds with the biopharmaceutical industry which saw its largest 12 companies achieve a 4.8 percent return on R&D investments, compared to 10.5 only five years prior in 2010.

Many Other Challenges Facing R&D

The importance of private-sector R&D cannot be understated. It is critical as both public funding declines and critics continue to villainize industry-researcher collaborations, making R&D even more difficult. As we previously noted, a recent study found that it costs drugmakers $2.6 billion dollars to develop a new prescription medicine that gains marketing approval. This is up from $802 million in 2003, equal to approximately $1 billion in 2013 dollars. While the average time it takes to bring a drug through clinical trials has decreased, the rate of success has gone down by almost half, to just 12 percent.

Furthermore, the estimated cost of post-approval research and development of $312 million “boosts the full product lifecycle cost per approved drug” to close to $3 billion. R&D costs include studies to test new indications, new formulations, new dosage strength and regimens, and to monitor safety and long-term side effects in patients as required by the FDA as a condition of approval.

Conclusion

The importance of private investment into R&D is a key part of scientific discovery and spurs growth domestically and abroad. But the burdens upon researchers are vast, and only further complicated by additional costs related to the Open Payments program. Every dollar spent on a compliance manager is a dollar not spent on R&D or complementary departments. All of this makes it even more remarkable that despite these hurdles, industry R&D, especially in the pharmaceutical industry, remains a crucial component to jumpstart the stagnated American economy.

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