By MARGARET A. HAMBURG

Courtesy of the Wall Street Journal

There are encouraging signs that the pharmaceutical industry’s pipeline of new products is not as stalled as some say.  Already in 2011, the Food and Drug Administration (FDA) has approved 21 new, groundbreaking medicines—the same number as in all of 2010—including treatments for Hepatitis C, late-stage prostate cancer and lupus.

We hope this signals a larger trend in industry and at the FDA. Despite common criticisms that our agency impedes innovation by being slow and bureaucratic, we actually play a proactive role in promoting innovation by ushering new products through the approval process and to market—while making sure they meet the standards of safety and effectiveness that have served the American people well.

When presented with high-quality applications that are based on strong science, we work quickly and efficiently. And we must, because our mission is to promote and protect the health of the American people.

This includes promoting the development of innovative new diagnostics and treatments that will help solve the complex public-health challenges of the 21st century. This requires working closely with industry, academia, patient and consumer groups, health-care professionals and others. These include streamlining and improving our approval processes; increasing transparency and building new collaborations; and strengthening the field of regulatory science, at the FDA and beyond.

Today, products we regulate account for more than one in five of all dollars spent on consumer products in the U.S.—or more than $1 trillion a year. The industries we regulate lead the world in innovation and provide hundreds of thousands of high-paying jobs. The U.S. biopharmaceutical industry accounts for the largest share of global launches of new drugs and the medical-device industry has a positive trade balance, with the value of its exports growing annually.

The FDA does bear some responsibility for the criticism we receive, and we listen carefully to our colleagues and critics. In response, we are actively taking steps to improve the clarity, consistency and predictability of our regulatory systems. At the same time, we are working with the broader scientific community to address issues of scientific uncertainty that slow medical-product development and review.

But misconceptions about our effectiveness as an agency could derail these efforts. One prevailing misconception is that the FDA lags behind Europe in approving medical products. The truth is that the FDA consistently approves the vast majority of priority drugs and medical devices as fast as, or faster than, our European counterparts. Of the 35 new cancer drugs approved either by the FDA or the European Medicines Agency (EMA) from October 2003 to December 2010, the FDA approved 32—in an average time per drug of 261 days. The EMA approved only 26 of these same 35 products, in an average time of 373 days.

Furthermore, all 23 cancer drugs approved by both agencies during this period were marketed first in the U.S., according to a study published last month in Health Affairs by Friends of Cancer Research, a think tank in Washington, D.C. We see similar trends for critical drugs in other areas of medicine.

According to an industry-funded study by the California Healthcare Institute and the Boston Consulting Group, lower-risk devices, such as wheelchairs, CT scanners and knee-replacement implants—which make up 80% of all devices reviewed each year—come on the market first in the U.S. as often as or more often than they do in Europe. While higher risk devices, such as pacemakers and drug-eluting stents often do come on the market first in Europe, this is in large part due to the U.S. requirement, absent in the EU, that the device be shown to benefit patients.

Another misconception is that the demands of the FDA’s approval process cost jobs and undermine U.S. global competitiveness. The opposite is the case. By instilling confidence in American products and technologies, the FDA  stimulates economic growth, creating jobs at home and opening markets overseas.

Consider that the U.S. biopharmaceutical and medical-device sectors dominate their global industries. Even during the recent recession, from 2006 to 2009, the medical-device industry increased sales, boosted exports and expanded its work force. During the same period, major biopharmaceutical firms demonstrated strong financial performances. This could not have happened without widespread consumer confidence, at home and abroad.

Without question we face formidable competition overseas, especially from Europe and China, where significant investments are being made in regulatory science. But the FDA remains the regulatory gold standard throughout the  world.

President Obama has made it clear that this administration will do everything it takes to promote innovation in medicine and health while preserving the safety and effectiveness of critical medical products. The FDA, working with industry and other groups, is eager to help lead the way.

That is what it will take to protect the public health in the 21st century. And that is what the American people deserve.

Dr. Hamburg is the Commissioner of the Food and Drug Administration.

New England Journal of Medicine

Auditing Access to Specialty Care for Children with Public Insurance

IOM has entered the next phase of this process and will be holding a serious of public meetings to hear from various stakeholders regarding essential benefits. The first meeting took place this week January 12 -14, 2011 and covered numerous topics within essential health benefits including:  the original legislative intent of health care reform regarding essential health benefits, stakeholder recommendations for defining health insurance packages, issues regarding medical necessity, how insurers determine benefit design and coverage and various perspectives from patients and health advocacy groups.

Some common themes emerged from the presentations:

Insurers argue that essential benefits should not be heavily prescribed through regulation to allow for the market to determine which benefits provide the best coverage options. They also cautioned against adopting state mandates, because of the tens of thousands already in existence across the country. They argue that adopting a multitude of mandates on the national level will bring a dramatic rise in health insurance premiums.

Patient and health advocacy groups argue that patients need access to treatment and providers of their choice especially those patients with chronic disease. Essential benefits must be designed so that this access is not denied. They urged the panel to ensure that the benefits reflect the diverse health care needs of the U.S. population and are based on appropriate standards of care and practice. According to the advocacy groups, the inclusion of some state mandates are necessary to maintain comprehensive care for those with complex health needs.

For more information on the Institute of Medicine and Essential Benefits please visit the study’s website.

Thank You!

Recent developments in Federal support for CER include:

• American Recovery and Reinvestment Act of 2009 provided $1.1 billion for CER
• Affordable Care Act
  • Created the Patience Centered Outcomes Research Trust Fund
  • Has mandatory appropriations funding
  • Sunsets September 2019
  • Ends the Federal Coordinating Council for CER and creates the Patient Centered Outcomes Research Institute (PCORI)
    • PCORI is a private nonprofit cooperation with a board of governors
    • The Board will have representation from AHRQ, NIH, and various stakeholder groups

For more information about Mathematica please visit their website: http://www.mathematica-mpr.com/.  For more information on the Center on Health Care Effectiveness and this issue forum click here.  To view HFA’s Issue Brief on CER click here.

The basis of the discussion was a newly released survey done by the Kaiser Family Foundation:  Health Care Among Non Elderly People with Disabilities and Seniors on Medicare, 2008. The survey results were reported in the September edition of the Health Affairs Journal: Medicare Doesn’t Work as Well for Younger, Disabled Beneficiaries as it Does for Older Enrollees.

The survey found:

• Overall non-elderly disabled beneficiaries facer greater financial and access barriers compared to elderly Medicare beneficiaries.
• More non-elderly Part D enrollees reported medication access problems compared to elderly Part D enrollees.
• Most of the survey respondents were satisfied with their Medicare coverage.
• Non-elderly disabled Medicare beneficiaries have more health problems/ co-existing conditions compared to elderly beneficiaries.
• Non-elderly beneficiaries with no supplemental coverage were more likely to report having difficulty paying for coverage.
• 1/3 of non elderly disabled Medicare beneficiaries reported being uninsured during the 24 month Medicare waiting period.  46% of all respondents states that the government should do away with the 24 month waiting period.

Below are some of the ways the Affordable Care Act will help Medicare beneficiaries.

• Health care reform will help some in Medicare’s waiting period access coverage through the new national high risk pools.
• Expansion of Medicaid eligibility up to 133% of the poverty level.
• Starting in 2014 insurance companies will be required to provide coverage and health insurance exchanges will be available to help individuals purchase health insurance. The government will also provide subsidies (if you qualify) to assist with the cost of health insurance.
• The new insurance program, Community Living Assistance Services and Supports (CLASS) Act will provide cash benefits for long terms services and supports for eligible participants.

For more information on the Kaiser Family Foundation and this forum please visit their website: www.kff.org or to view the presentation and background materials from the forum click here.