Life Science Compliance Update

October 08, 2013

Health IT: White House Office of National Coordinator Issues Surveillance Guidance for Meaningful Use

According to a report at FierceEMR, The Office of the National Coordinator of Health IT has issued its first surveillance guidance to assist its ONC-Authorized Certification Bodies (ONC-ACBs), which certify which electronic health record products qualify for participation in the Meaningful Use Incentive program.

The ONC-ACBs are required by the permanent health IT certification program to conduct periodic surveillance of the EHR products they certify. The new guidance, issued July 2, outlines ONC's surveillance requirements, such as annual submission of a surveillance plan and surveillance approach. It flags four surveillance plan elements that ONC considers priorities for the ONC-ABCs to monitor:

  • Exchange capabilities
  • Safety-related capabilities
  • Security capabilities
  • Population-management capabilities

The guidance also states that the assessment of EHR developers' complaint processes is a priority of ONC, and that the ONC-ACBs' surveillance plans need to address how they will assess vendors' handling of complaints, especially safety-related ones.

And according to InformationWeek: The Certification Commission for Health IT, or CCHIT, is among the half-dozen ONC-ACBs that have been testing and certifying EHRs products under the temporary program. CCHIT will seek to be authorized under the permanent program as well, said CCHIT chair Karen Bell.

"The good news is that ONC has given us a year for the transition before the permanent program goes live," she said in an interview with InformationWeek. In the meantime, CCHIT will continue to test and certify products for ONC meaningful use certification.

The rules of the permanent certification program "also open the door" for organizations in the future to be authorized to test and certify other health IT products that aren't currently identified in the ONC's meaningful use programs, such as remote monitoring and other devices, said CCHIT's Bell.


As for CCHIT, Bell expects 2011 to be a busy year, not only as other vendors seek meaningful use certification, but also as many hospitals seek certification for their mix of various vendor systems and/or home-grown EHR systems.


"So far the biggest demand [for testing and certification] has been from vendors, but we expect demand will burst from hospitals," Bell said. CCHIT is piloting a testing and certification program for hospitals.

February 14, 2013

White House PCAST: Rival Countries Gaining on US Medical Research Spending

The Nation once led the world in investments in research and development (R&D) as a share of gross domestic product (GDP), but more recently, the United States has been investing less in R&D than other leading and emerging nations invest.  Moreover, U.S. industry has been shifting its investments toward applied R&D, narrowing the support for basic and early-stage applied research, which is crucial to transforming innovation.  

Without adequate support for such research, the United States risks losing its leadership in invention and discovery—the driving force behind the new industries and jobs that have propelled the U.S. economy over the past century.  

President Obama’s Council of Advisors on Science and Technology (PCAST) recently released a report maintaining that if the United States wants to maintain “its lead in technology and innovation, then the federal government must be willing to spend more on basic research and encourage industry investments.” 

PCAST said that U.S. investment in research and development as a fraction of gross domestic product “has dropped from first in the world to eighth, and is fourth in the world among large economies.  The report warns that this is a significant threat to long-term research if qualified researchers left for other countries couple with a private sector that is focused primarily on near-term results. 

At the report’s announcement, John Holdren, director of the White House Office of Science and Technology Policy and PCAST co-chair, said that the United States, for now, has no equal in terms of capabilities.  “Note that I said unmatched and not unrivalled,” said Holdren, “we do have rivals.  More and more of them all the time.” 

The report argues that losing a high ranking in science and innovation would cost the US jobs in science and supporting industries as well as benefits to safety, medicine and national security.  “More than half, and perhaps as much as 85 percent of productivity growth in the United States in the first half of the 20th century” can be attributed to technical advances, says the report.  It also describes the National Science Foundation, National Institutes of Health (NIH) and the Energy Department as the “primary stewards of basic research in the United States.” 

To promote domestic research, PCAST urges the government to increase research and development expenditures from the current level of 2.9 percent of GDP to 3 percent as well as create policy incentives that will increase the private sector's investment in long-term research.  Private industry currently accounts for some two-thirds of the total R&D expenses in the U.S. 

The report also warns against instabilities in funding, such as the looming sequestration, and says the government should develop a multiyear funding approach to prevent research gaps.  It recommends using the multiyear financial plan similar to the Defense Department's future years defense program as a possible blueprint.

Taxes also come into play, with PCAST suggesting that the research and experimentation tax credit be made permanent and rise from a rate of 14 percent to 20 percent.  It says this will make it more useful to small and medium-sized enterprises that are R&D intensive. 

Among the actions that PCAST recommends, three stand out in scope and importance: 

(1)  that the President reaffirm his stated goal that U.S. total R&D expenditures (across the public and private sectors) should achieve and sustain a level of 3 percent of GDP;

(2)  that actions be taken, some achievable entirely by Executive decision, to increase the stability and predictability of Federal research funding; and

(3)  that Congress not only make the R&D tax credit permanent, but increase it to at least 17 percent, as you have already advocated. 

While the benefits from scientific advancements are evident in virtually every aspect of modern life, they are perhaps most immediate in the area of human health.  Biological discoveries have lengthened our lives; in the mid-2000s, the life expectancy in the United States reached an all-time high of 77.5 years, up from 49.2 years at the beginning of the twentieth century. 

 Advances in treatment rooted in improved understanding of biological function contribute to continual improvements in the quality of life in later years. HIV-AIDS, whose diagnosis was once a death sentence; is now treatable: In areas where HAART (highly active anti-retroviral therapy) became available, deaths related to HIV have decreased by 94 percent and transmission rates by 96 percent. 

Thanks to the development of vaccines, global incidences of diseases, including diphtheria, polio, yellow fever, and tetanus, have plummeted, and smallpox has been eradicat-ed. Sequencing of the human genome has led to the identification of genes associated with diseases such as multiple sclerosis and cystic fibrosis, bringing the promise of improved treat-ment and ultimately a cure. 

A healthier population is also an economically more productive population.  That is not, however, the only reason that Americans value the benefits of biomedical research.  We want longer, healthier lives for our elder parents, ourselves, and our children.  Further, it is an American social value to be generous in bringing healthier lives to our own neediest and to the world.  Today, Americans look to science and technology for health care that is not only more effective, but also more affordable, so that all may benefit fully.

October 01, 2012

President’s Council of Advisors on Science and Technology: Propelling Innovation in Drug Discovery, Development, and Evaluation

National Center for Translational Sciences
Due to biomedical innovations—including advances in medicines, medical procedures, and public health, life expectancy at birth has risen from around 47 years at the turn of the 20th century to 78 years today, and many diseases that were once fatal or debilitating can now be prevented, delayed, or ameliorated.   

In fact, pharmaceutical therapies have helped lead to vaccines that have eradicated or controlled many devastating infectious diseases including polio, small pox, and measles.  HIV is now treated with over 20 Food and Drug Administration (FDA)-approved drugs.  Multi-drug regimens effectively control HIV infection, preventing the development of AIDS.  Pharmaceutical therapies have led to cures for multiple malignancies that were once universally fatal; for example, childhood leukemia is now cured in 80 percent of cases, testicular cancer in over 90 percent of cases, and Hodgkin’s lymphoma in over 90 percent of cases.  Along with a reduction in smoking and better medical care, cholesterol-lowering therapy, blood-pressure-lowering drugs, anti-platelet agents, and diabetes treatment have contributed to a substantial decrease in death from heart attacks (70 percent decline over the past 60 years). 

A recent report recognized that in addition to academic researchers who “have unlocked secrets of basic biology and revealed mechanisms that underlie disease,” these innovations have been brought forth by a “a robust bio-pharmaceutical industry, which has developed molecules to treat disease and conducted clinical trials to demonstrate their efficacy.”  Despite this progress, challenges remain.   

To address these challenges, “The United States should set a goal of doubling the output of innovative new medicines that meet critical public health needs over the next 10 to 15 years, while continuing to increase drug safety,” a presidentially appointed council of experts advised in a report released in late September.  The council recommends a number of actions involving industry, academia, and the Federal Government.  

“While basic biomedical sciences have seen stunning progress in past decades, challenges remain in translating those scientific advances into practical solutions,” according to the report—Propelling Innovation in Drug Discovery, Development, and Evaluation—produced by the President’s Council of Advisors on Science and Technology (PCAST).  The report assesses the reasons for that long-term trend.  

To support innovation and accelerate the development of new therapies, the report makes a number of detailed recommendations aimed at  

  • bolstering the discovery and development of new therapeutic compounds;
  • optimizing processes used by the Food and Drug Administration (FDA) to evaluate the safety and efficacy of candidate drugs;
  • enhancing long-term monitoring of approved medicines; and
  • enhancing public understanding about the benefits and risks of medicines.  

“With improved collaboration among all the participants in the drug development ecosystem and optimization of drug-evaluation pathways, American researchers and companies should be able to accelerate the development of safe and effective drugs while also strengthening the U.S. economy,” said Eric Lander, who co-chairs PCAST.  

The report notes that heart disease and stroke remain leading causes of mortality, many common cancers are still incurable unless they are caught in the earliest stages, and the vast majority of rare diseases lack effective therapies altogether.  Infectious diseases, including those caused by antibiotic-resistant bacteria and viruses with pandemic potential, pose a constant threat of large-scale mortality. And treatments for psychiatric diseases, which impose a tremendous burden on society, are frustratingly limited in their efficacy, as are treatments for neurodegenerative diseases such as Alzheimer’s.  

All three major components of the drug development ecosystem—basic biomedical research in universities and research institutes, clinical research in hospitals, and drug discovery and development in the biopharmaceutical industry—are facing growing challenges as the time, complexity, and cost of developing drugs have gone up, the report states.  The rate of new-drug applications submitted by industry to the FDA, as well as new drug approvals, has remained relatively constant for 20 years.  In an encouraging sign, however, the FDA approved 35 new medicines in the past year—among the highest totals in the past decade. 

The report concludes there are two critical needs related to drug discovery and development that must be addressed to advance innovation:  

(1)  Scientists need better methodologies and tools for translating basic biological insights into validated therapeutic targets and leads—a gap in the drug discovery and development pipeline that academic scientists often view as “too applied” and pharmaceutical companies often eschew as “too basic” to justify private investment.  

(2) Pharmaceutical developers and regulators need to incorporate new efficiencies into clinical trials of candidate medicines—complex and costly human studies that today constitute fully 40 percent of the biopharmaceutical industry’s R&D budget.  

To achieve some of the report’s broader goals, PCAST recommends the creation of a public-private “Partnership to Accelerate Therapeutics,” involving representatives from the bio-pharmaceutical industry; the academic biomedical research and ethics community; physician societies and pharmacists; patient-focused research foundations and advocacy groups; healthcare providers and insurers; and the Federal Government. The Partnership would help identify and plan collaborative actions to speed drug development while balancing competing stakeholder interests and minimizing duplication of efforts.  

This report should be applauded for such a recommendation.  We have long praised industry collaboration, and with such a large and diverse group of stakeholders, we are hopeful that this report will lead to more progress and innovation.  The report recognized that “development of innovative medicines … remains essential for progress in the prevention and treatment of human disease.” 

In addition, the report concludes that the return on investment in certain disease domains may be too low to justify their pursuit by companies, even though the potential benefits for public health in these domains may be large.  It recommends that the Department of Health and Human Services (HHS) commission a study to assess potential mechanisms to encourage companies to tackle important medical challenges that may be financially unattractive.  

“There is a tremendous need for new antibiotics, for example, but the potential market share for such medicines is typically small and their duration of use is typically short,” said PCAST member Christine Cassel, a physician who—with Lander and PCAST members Ed Penhoet and Rick Levin—oversaw a group of 30 outside experts who helped inform PCAST in its work.

R&D Under Stress

The report recognized, contrary to some critics assertions, that there is evidence that industry R&D investment, a major component of this innovation ecosystem, is under significant stress: 

  • The pharmaceutical industry is facing the largest “patent cliff” in its history: drugs with annual sales exceeding $200 billion will come off patent in the period 2010-2014, resulting in a loss of more than $100 billion in sales to generic substitutions; only a small fraction is expected to be replaced by new product revenues.
  • Venture capital to start new biotechnology firms and fund innovative drug development activities appears to be declining, due not only to general economic conditions but to what are cited as concerns about unfavorable returns in the drug-innovation sector.
  • Many companies are exiting important fields of critical public health need. For example, despite the growing health care and economic burden of neurodegenerative diseases, such as Alzheimer’s disease and psychiatric diseases, many major pharmaceutical companies are closing down or severely curtailing drug discovery programs. 

The report identified two critical areas related to drug discovery and development that must be addressed to advance innovation: 

  1. 1.    Scientific knowledge gaps between basic research and commercial projects.
  2. 2.    Inefficiency in clinical trials.  

The report also highlighted multiple issues and opportunities that relate to the evaluation of new drugs for approval:   

  1. There are opportunities to accelerate the approval of a broader range of truly innovative drugs for patients who need them. Such acceleration should be supported by stronger tools for and enforcement of post-approval study 
  2. The FDA requires methods to rapidly approve drugs for narrow populations for which there is a favorable benefit-risk balance, while protecting the broader population from drugs that have an unknown or unfavorable benefit-risk balance 
  3. Stronger post-marketing surveillance and communication tools are needed to generate evidence on the benefits and risks of drugs and to communicate those benefits and risks to the public 
  4. Innovators require greater clarity about general regulatory pathways for innovative products and approaches 
  5. Innovators require greater consistency, efficiency, and communication with respect to their individual drug applications 

These recommendations are encouraging.  We have seen the pharmaceutical and medical device industry significantly maligned for their attempts to create wealth from medical products.  Reports like these go a long way to dispelling the myth that creating new products is easy and can cost as little as a million dollars.  The truth drug development is around $3 billion per new product which in the end may not be sustainable.  A little encouragement from the Whitehouse on the need for reduced regulations will go a long way to encouraging future investment.


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