Life Science Compliance Update

February 16, 2016

Senate HELP Committee Moves Closer to Creating Corollary to 21st Century Cures

Last week, the Senate Health, Education, Labor, and Pensions (HELP) Committee approved a series of seven bills to work up medical innovation legislation that will eventually likely become the Committee's response to the 21st Century Cures Act passed by the House of Representatives.

Last month, HELP Committee Chairman Lamar Alexander of Tennessee estimated that the Senate's version will not mirror the House bill, and that the Senate's effort will focus on priorities that are identified by the Committee's members, such as improving federal electronic health record programs.

Last week's productive meeting was not the last meeting for the Committee on this topic, either. The panel is set to consider approximately twenty pieces of innovation-related legislation, and there are meetings set for March 9, 2016, and April 6, 2016.

During Chairman Alexander's opening statement last week, we saw a preview of his expectations for the process of how to move the innovation bills through the panel all the way to the Senate floor. He emphasized the bipartisan nature of the fifty proposals included in the twenty bills, and that he would personally ensure that consideration of each proposal would include an opportunity for debate and open amendment. Chairman Alexander is hoping that he can work to create a consensus on the measures while they are in the Senate panel, holding off the contentious debates until they reach the Senate floor.

The Advancing Targeted Therapies for Rare Diseases Act of 2015

This bill aims to speed up the approval process of genetically targeted drugs by allowing companies to use evidence from already-approved drugs. As per the text of the bill, this legislation would "facilitate the development, review, and approval of generically targeted drugs to address an unmet medical need in one or more patient sub-groups (or gene variant subpopulations) with respect to rare diseases or conditions that are serious or life threatening; and maximize the use of scientific tools or methods, including surrogate endpoints and other biomarkers for such purposes." This approach is different from the 21st Century Cures Act in that the 21st Century Cures bill would provide a six-month extension of exclusivity periods and patent protection for an already-approved drug if the sponsor works to obtain approval for a new indication for either a rare disease or rare condition.

The FDA Device Accountability Act of 2015

This bill is similar to the 21st Century Cures Act and attempts to eliminate "burdens" that are slowing down the FDA's consideration of new, innovative, medical devices. This bill asks for FDA reviewers to use least burdensome requirements in their review, permits centralized, non-local institutional review boards for device trials, and also requires the FDA to update their guidance on Clinical Laboratory Improvements Act (CLIA) waivers. The Senate goes further than the House, however, by allowing reliance on post-market information for premarket approval devices as part of the least burdensome requirements.

The Next Generation Researchers Act

This bill works with the NIH and establishes a Next Generation of Researchers Initiative, where policies and programs would be coordinated to promote and provide research opportunities for new researchers and work to give researchers earlier independence. The legislation also calls for the National Academy of Sciences to study and report on the barriers that are likely to be faced by the next generation of researchers, the impact of sequestration and federal budget constraints, and recommendations for ways to improve entry into, and sustain careers in, research.

The Enhancing the Stature and Visibility of Medical Rehabilitation Research at NIH Act

This bill calls for the National Center for Medical Rehabilitation Research (NCMRR) to develop and update a research plan for medical rehabilitation research. Under this bill, the NCMRR is also required to annually report on the progress of achieving the objectives, benchmarks, and guiding principals described in the research plan. The bill mandates HHS enter into interagency agreements to better coordinate medical rehabilitation research.

The Advancing Research for Neurological Diseases Act of 2015

This bill amends the Public Health Service Act to require the Centers for Disease Control and Prevention to enhance and expand infrastructure and activities to track neurological diseases. The bill also mandates that CDC incorporate that information into a National Neurological Diseases Surveillance System, and requires that HHS provide for the collection and storage of information on neurological diseases, including incidence and prevalence rates, to the extent practicable.

The Preventing Superbugs and Protecting Patients Act

This bill mandates that the FDA publish a list within six months following enactment of a reusable device that would need to provide proposed cleaning instructions as part of a 510(k) premarket submission. The devices on the list would also be required to provide the FDA with the validation data that shows that the proposed cleaning instructions are sufficient for safe cleaning, disinfection, and sterilization of the devices between patient use.

The Improving Health Information Technology Act

This bill scrapped an earlier plan of setting up a new health IT committee to replace current committees. This proposal eases requirements on physicians who enter data into electronic health systems by permitting nurses and other healthcare provider teams to enter and document the information as well. The bill also establishes a rating system for health information technology to help professionals in choosing products and facilitating information sharing between providers. This most recent draft also strikes fines and compensation fund found in earlier proposals.

These bills are a start for the Senate to work towards a companion bill to the House 21st Century Cures Act. We will monitor the Senate's progress and try our best to keep our readers abreast of any developments.

July 22, 2015

FDA: "More Collaboration, Research Needed to Develop Cures"


The Food and Drug Administration's Deputy Commissioner for Medical Products and Tobacco, Robert Califf, recently offered his take on what is holding back medical progress. While a common critique is that the Agency's red tape is holding up innovation, Califf and an accompanying FDA report note that a "lack of understanding of the biology of the diseases" is to blame for the lack of treatment for Alzheimer's and many rare diseases.

"In these cases, the scientific community still lacks basic information about what causes these diseases and how they can be slowed and treated," states Califf. "When research does not offer answers to important scientific questions, cures cannot be developed. And when viable cures are not in the pipeline, focusing on regulation will not improve the situation, since FDA can only approve therapies with evidence for safety and effectiveness."

Califf writes:

Once key scientific questions are answered, we can use a variety of tools to reduce the length and cost of initial clinical trials for drug approval for these disease areas, and we can provide guidance to industry including advice on how to develop additional reliable biomarkers. For instance, we’ve improved the efficiency and predictability of clinical drug development by developing tools such as biomarkers and surrogate endpoints—markers of drug effect that do not directly represent an improvement in how a patient feels or functions, but are reasonably likely to predict a clinical benefit. Thus, for example, lowering a patient’s blood pressure can be used as a surrogate for the clinical benefit of preventing heart attack. Such tools have modernized clinical trial designs and may dramatically reduce the length and cost of drug development. They also can help target drugs to specific patients who can benefit most, thereby limiting the number and size of clinical trials.

An accompanying FDA report goes into detail about the state of drug discovery and development. for a number of diseases, starting with Alzheimer's (where "scientific discovery is in its infancy"), to diabetes (which is understood better than Alzheimer's, though scientific understanding is still lacking), to Hepatitis C (which now has a targeted therapy to prevent and treat the virus in specific patients). 

"While FDA has worked to transform the landscape for the final stage of drug development, progress in the discovery and testing stages of drug development has not kept pace," the report states. "As a result, too many diseases are still awaiting treatments and cures." View the report here.

Califf adds:

These are exciting times as we experience simultaneous revolutions in the biological and information sciences. We expect that the astounding increase in knowledge of biological systems enabled by whole genome sequencing, cloud computing, social media, and wearable devices to monitor physiology will create challenges to traditional thinking. And we are confident that this increased knowledge will continue to expand the pipeline of new therapies. This report emphasizes that we are prepared to deal with the product of this scientific investment by using regulatory paradigms that match the state of the science and by supporting dissemination of the latest knowledge applied to drug development.

The Agency will "continue to work to speed patient access to therapies shown to be safe and effective through our existing programs that allow for expedited review, development, and approval of certain medical products." In conclusion, he adds: "To encourage innovation, we also will continue to work with other government agencies and the healthcare community, including members of patient groups, academia, and industry. It will take a collaborative effort to improve our nation’s understanding of certain diseases and to translate any resulting scientific discoveries into cures."



June 29, 2015

The Importance of Private Investment into Research and Development


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.


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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