I recently attended a conference entitled "What Evidence is Essential for New Medical Products?" held at the American Association for the Advancement of Science in Washington, D.C., that probed the vexing dilemma of balancing the duty of the Food and Drug Administration (FDA) to assure the safety and efficacy of new drugs and medical devices versus the demand of patients for faster access to potentially life-saving cures.

The dilemma is best illustrated by the following "Tale of Two Drugs." When the AIDS epidemic first swept the country and the world in the 1980s, AIDS activists successfully pressured the government to speed up the drug approval process for AZT (zidovudine), a process that usually took many years before drugs came to market. Those suffering with AIDS were literally dying in the streets. Robert Yarochoan, an FDA official at that time, told the conference audience of the dramatic response of both the FDA and the pharmaceutical company Burroughs-Wellcome to expedite the approval of AZT. Countless lives were saved as a result.

The other tale also deals with a deadly disease that is ravaging the world—tuberculosis (TB) that is resistant to the usual antibiotics used to treat it. Jerry Avorn spoke about what appeared to be a promising drug called bedaquiline. In the laboratory, bedaquiline appeared to kill multi-drug resistant TB germs. Usually, the next step in the drug-approval process would be small-scale clinical trials where the drug is tested in actual patients suffering from the disease. This vital step in the drug approval process was skipped in order to bring a potentially life-saving drug to market quickly. Tragically, when the drug was used for the first time by real patients, it ended up having a fatality rate five times that of more standard drugs for multi-drug resistant TB.

Similar problems exist for medical devices. Devices, such as pens to inject insulin, that are very similar to devices that are already approved and on the market, only have to pass a cursory review to assure that they are essentially equivalent. Truly new devices or ones that are substantially different from existing ones must go through an intensive approval process, more like new drugs, in which they must prove both their safety and efficacy. Astonishingly, only 1 percent of devices go through this intensive review process according to medical journal editor Dr. Rita Redberg.

Dr. Redberg informed the conference about WINGSPAN, an implantable filter that was supposed to protect against second strokes. FDA did not require the device to go through the intensive review and the device turned out to increase strokes and death. Even worse, it remains on the market and more than 11,000 devices are still being implanted every year.

Stories like this illustrate the dangers of rushing drugs and devices to market. Pharmaceutical and medical device manufacturers continue to ratchet up the pressure on the FDA to expedite the approval process, both by directly lobbying FDA, and by enlisting the support of many patient advocacy groups and lawmakers. At present, more than half (56%) of newly approved drugs used an expedited-approval pathway. (Darrow JJ, Avorn J, Kesselheim AS. New FDA breakthrough-drug category—implications for patients. N Engl J Med 2014;370:1252–8)
Congresswoman Rosa DeLauro (D-CT) spoke passionately about the dangers to patients in the present system, and has submitted a letter to FDA about her grave concerns about the proposed new accelerated drug approval guidelines. At the conference she also pledged to support giving the FDA the resources it needed to safeguard the nation's drug supply.
Dr. Peter Lurie, associate commissioner of FDA, defended the agency, noting that just because FDA approved a drug didn't mean doctors needed to prescribe it. But we know that drug company marketing strategies go into high gear once a drug is approved. Doctors then face intense pressures from industry, as well as pressure from patients who have been urged to "ask their doctors" on television ads about the latest drugs.

A number of other ideas emerged from the conference that offered more promise as a means of dealing with these dilemmas. In Europe, drugs that have been approved but where there is more uncertainty about their safety and effectiveness are labeled with a black triangle. Such a system could be used by FDA, which already mandates black box warnings to alert doctors to particularly dangerous side effects.

The FDA gives some new drugs a “conditional approval” and then requires the company to provide more data, but many companies fail to provide the data and there is no penalty. In some countries, however, where the government is the major purchaser of drugs, it pays the manufacturer a lower rate for the drug until it can produce convincing data about the safety of the drug. This provides a powerful financial incentive for drug companies to make good on their promise of more data. Public programs like Medicare and Medicaid could do this same in this country. The FDA should also consider including “sunset provisions” along with any conditional approval—the approval would end unless the additional data on safety and efficacy were provided in a specific timeframe.

Dr. Bernie Good from the Veterans Administration reported that the VA protects veterans by taking into account safety data when it decides whether to place a drug on the VA formulary. New drugs that have not undergone the full rigorous testing are less likely to be placed on the formulary. Medicaid programs also consider safety when deciding what to include in their list of approved drugs. This is not true for the Medicare program for the elderly, but is an idea worth pursuing.

Most importantly, the FDA must be given the financial resources it needs to do its job without depending on industry fees, which now pay for a large percentage of the costs of the drug approval process. The temptation is just too great for the agency to cut corners to avoid biting the hand that feeds it. While the FDA needs to have a good working relationship with industry, its mission is to serve the public's best interests. That mission must never be compromised.

Stephen R. Smith, M.D., M.P.H., Community Catalyst physician consultant

Two patients recently saw me within a two-week span asking for a prescription for Belviq (lorcaserin), a weight-loss drug. Both patients told me they had seen the drug advertised on television. Like most direct-to-consumer (DTC) ads, the one for Belviq ended with the advice to "ask your doctor about" it.

Based on my knowledge of weight loss drugs, my initial reaction was a negative one. The last weight-loss rage for phentermine+fenfluramine ("phen–fen") resulted in patients with damaged heart valves—an adverse effect that became evident only after millions of patients used the drug combination for many years. And with all the weight-loss drugs I have seen, the benefits in terms of weight loss were neither dramatic in magnitude nor long-lasting in duration.

That gut reaction notwithstanding, I recognized the patients' sincere desire to lose weight. Both were middle-aged women whose body-mass index (BMI) scores were above 30, thus putting them in the category of "obese." Belviq is approved by the Food and Drug Administration (FDA) for patients with a BMI of 30 or higher.

The first patient said she had been trying to lose weight on her own but had been unsuccessful. I thought this was a good opportunity for the patient and me to balance the pros and cons of taking the drug. I opened a recent issue of The Medical Letter that reviewed Belviq. Together, we read the indications for its use, how effective it was likely to be, and its side effects. After reading this information, I remained skeptical of her use of this drug, but she reiterated her fervent desire to try this drug as a means to lose weight. So I agreed to allow her to start the drug on a trial basis, so long as she agreed to stop taking it if was unable to lose 5 percent of her weight within three months. She agreed to that stipulation, which is included in the drug labeling.

A second patient of mine who asked for Belviq was struggling with weight loss, but she had already lost 5 pounds. I praised her successful efforts thus far with diet and exercise, noting that this was the best way to adopt a sustainable approach to weight control. When she reiterated her request for Belviq, I told her about the phen–fen disaster and the possibility that there could be hidden dangers with Belviq, as well. Though she still said she wanted the prescription, she agreed to continue on her own program of weight loss without drugs, with the understanding that we could revisit the question in a few months, depending on her continued progress.

These two cases highlight the controversy surrounding DTC advertising. Clearly, the TV ads for Belviq had motivated these patients to ask me for a prescription for the drug. In an ideal world, primary care providers would proactively engage their obese patients in discussions about weight loss without DTC advertising. Indeed, DTC advertising is not permitted by governments in the rest of the world other than in the United States and New Zealand. But here in the U.S. the FDA allows such advertising and physicians must be prepared to deal with DTC ad-driven patient demand.

Drug companies will do everything they can to maximize that demand and the sales that advertising promises. This can include promising consumers better outcomes, while downplaying the risks of taking a drug.

My patients were a perfect example of the outcome drug companies hope for, except for one thing. Both of my patients were covered by Medicaid, and in Connecticut where I practice, the state Medicaid program does not cover drugs for weight-loss, even though they cover other weight-loss treatments.

My patients were not the kind of patient that these DTC ads were intended to reach. The drug company wants to motivate patients with commercial insurance to go ask their doctors about Belviq. But commercial insurance companies should be just as careful as Medicaid in evaluating whether specific drugs with limited benefits should be covered. Our nation's ability to achieve sustainable, affordable health care for all depends on avoiding expensive treatments that are likely to be wasteful (because less expensive equally good alternatives are available),unnecessary or even counterproductive, because of risky side effects. 

For the time being, DTC advertising appears to be here to stay in the U.S., but that doesn't preclude steps that can be taken to mitigate its most worrisome features.

First, all DTC ads should be approved by the FDA before they are released to the public. This will assure that an accurate presentation of the risks and benefits is achieved.

Second, DTC advertising should be prohibited for at least two years after a drug is first released. This can help limit the extent of its use during the critical period of time when unknown rare but serious and potentially lethal side effects may become evident.

Third, the revenue generated from financial penalties levied against companies for false and misleading advertising should be used for public service advertising and educational activities that warn patients about the risks of prescription drugs and promote healthier alternatives.

Finally, medical students, residents, and practicing physicians must learn skills in communication to more effectively interact with patients who, having just seen an ad on TV, come in and “ask their doctor about….”


 Stephen R. Smith, M.D., M.P.H., Community Catalyst physician consultant

In June, I had the great honor of presenting on a panel at the Practice Change Leaders for Aging and Health (PCL) meeting in Chicago. For the uninitiated, the PCL is a gem of a program through which ten leaders work to develop their leadership skills by undertaking a one-year project aimed at improving care for older adults. The PCL chose a panel topic near and dear to our hearts here at Community Catalyst: Engaging Older Consumers in Health Care Design and Delivery.

The panel began with a poignant and heartbreakingly honest presentation by a wonderful woman named Darlene. For the last several years, Darlene has been a caregiver for her husband, who has advanced dementia. As she described her journey, she talked about meeting with providers who gave her medical diagnoses, but when asked about help in caring for her husband on a day-to-day basis, essentially said “good luck with that!” After years of struggling to keep up with the daily challenges of caregiving and her husband’s deteriorating condition, it was only by pure chance that Darlene discovered AgeOptions, an Area Agency on Aging that provided her with a broad range of supportive services, including respite care.  

Throughout Darlene’s talk, I was struck by the failure of the health system to help her readily connect to the long-term services and supports (LTSS) she and her husband needed. This failure is both harmful to patients and their families, and is a major contributor to skyrocketing health care costs. It was also one of the driving forces behind provisions included in the Affordable Care Act that aim to provide better care at lower costs for our highest-need, highest-cost patients. One of the solutions to this problem can be found in better LTSS coordination, which is meant to be a feature of the dual eligible demonstration projects rolling out in over a dozen states.

Voices for Better Health recently released a video, Olivia’s story, that demonstrates the power of LTSS coordination to help people live with dignity and as much independence as possible. But, as Olivia’s Story emphasizes, getting LTSS right requires:

  • a comprehensive in-home assessment that takes into account a person’s needs, preferences and goals, not just a checklist assessment
  • a consumer and an informed and independent advocate working together to develop an effective, integrated plan for LTSS
  • a role for the coordinator on the care team

We hope Olivia’s Story inspires you to work for improved LTSS coordination. Because when it comes to caring for older adults and people with disabilities, getting the support you need shouldn’t be a matter of “luck.”

If you’ve been following children’s health policy lately, you have probably been hearing the term MACPAC bandied about. You might have thought, “What is MACPAC? Is this some new version of Pac-Man?” Those are excellent questions, which we will answer here.

First, the easy one – we love Pac-Man, but aside from his very healthy fruit diet, he has little to do with children’s health.

So secondly, what is MACPAC? It’s the Medicaid and CHIP Payment and Access Commission created as part of the Children’s Health Insurance Program Reauthorization Act (CHIPRA) in 2009. The authorizing language for MACPAC was added to the Social Security Act. The statute called for the creation of a non-partisan federal agency to conduct policy and data analysis relevant to Medicaid and CHIP, and make recommendations to Congress and the Secretary of Health and Human Services about the programs. Broadly, this means that MACPAC is a trusted source for in-depth analysis of Medicaid and CHIP issues and an apolitical voice in health policy debates.

MACPAC is made up of 17 commissioners; these commissioners serve three-year terms and bring an array of diverse backgrounds and experience to their work. The agency also has a full-time staff that supports its work.

MACPAC’s policy and data analysis takes the form of reports issued to Congress by March 15 and June 15 each year (the Commission may make additional reports). In its most recent report published earlier this month, the Commission made recommendations about the future of CHIP, supporting extension of the program. This recommendation represents an important affirmation of CHIP’s ongoing importance in the coverage landscape. Additionally, the June report includes recommendations about the role of Medicaid in paying for long-term services and supports and building Medicaid’s administrative capacity on the state and federal levels. You can read the full report, including MACPAC’s thoughts on the future of CHIP, here. For Pac-Man’s thoughts on the future of CHIP, you’ll have to ask him—watch out for the ghosts.

As much of our energy is focused the “big” policy issues of coverage and the implementation of the Affordable Care Act, an often overlooked health issue for children is vision care. Vision issues impact 5 percent to 10 percent of the nation’s preschool aged children and disproportionately impact children of color and children with special health care needs. This issue may seem small in number, but children with preventable vision issues (the majority of which can be corrected if identified by the age of 3) deserves “all eyes on them.”

So why do so many children go undetected? Kira Baldonado, Director, National Center for Children's Vision & Eye Health at Prevent Blindness, attributes the problem broadly to inconsistent vision screenings, lack of coordination and a need for national guidelines.

Vision screenings vary widely

There are varying rates of vision screening state to state. According to the National Survey for Children’s Health, approximately 74.5 percent of 4 to 5 year olds in the New England region had their vision tested in 2011-2012 – this is the nation’s highest screening rate while other regions fall well below ranging from 36.3 to 44.3 percent in the Great Lakes, Midwest, and South-Central regions. In addition, the tools that providers use to screen children vary within and across states and depend upon the screening setting. To add complexity, vision screening is more than identifying shapes at the end of a hallway. Additional tests such as stereopsis screening are important for detecting amblyopia (lazy eye) or strabismus (crossed eyes)—conditions that can lead to blindness if not detected early.

Lack of systems of coordination and communication

Vision testing can occur in multiple settings: a pediatrician’s office, a school, a childcare center or as a part of a community-based effort. This can result in multiple communications to parents and differing results. Most states lack a systematic approach to vision screening surveillance where data is captured in a single format and is accessible across multiple settings (Ohio stands out as a leader). There is also a lack of communication with the public about the importance of vision screenings, comprehensive exams and treatment. Children and their families must be at the center of care, but also must be partners in elevating the issue of vision health. (We illustrate one way to do this below.)

Evidenced-based practice

Maternal and Children’s Health Bureau and its partner Prevent Blindness America are spearheading a National Center for Children’s Vision and Eye Health that will provide recommendations for establishing national guidelines for children’s eye health.

Moving Vision Issues on the Ground

Massachusetts is a leader in elevating the importance of vision screening and treatment. Children’s Vision Massachusetts seeks to advance the practice of appropriate vision screening and treatment for children and insure that all children have the opportunity to develop and retain their best possible vision to support healthy development and academic success. The coalition works through multiple approaches including public education, workforce training, and facilitating greater coordination and communication across care settings.

Cognizant that pediatricians have the greatest access to children ages zero to five, the Pediatric Physicians’ Organization at Children’s (PPOC) and Children’s Vision Massachusetts decided to better understand what was happening in the pediatric office setting. Through a survey and chart review of their pediatric practices, they found that approximately 63 percent of 3, 4, and 5 year old children were vision acuity screened during well visits. Armed with this information, PPOC and Children’s Vision Massachusetts embarked on a massive education effort to train pediatric practices on proper vision screening techniques and better understand the implementation barriers. PPOC continues to monitor the success of their efforts and proactively engage their pediatric practices to ensure continued support for vision screenings for our youngest children. We applaud the transparency of PPOC in its efforts to improve the quality of pediatric care delivery.

This past month, Children’s Vision Massachusetts began a campaign to tell their story. We are proud to share their efforts! It teaches us not just the value of children’s vision but also the value of working with a diverse group of stakeholders: early childhood educators, school nurses, pediatricians, optometrists, ophthalmologists and other vision providers, AND, most importantly, the children and families impacted.

Children’s Vision Massachusetts documented the value of vision screening for the public as a part of their work for the American Academy of Pediatrics. Leveraging their pediatrician supporters and optometrist and ophthalmologist partners, they began to tell the narrative of child vision. Advocates developed a series of stories of children and families and ultimately, developed this public education video that highlights vision health through the eyes of children. We are very proud to support Children’s Vision Massachusetts as they begin this important work. They are at the initial stages of educating the public, bringing diverse stakeholders to the table, and developing a stronger uniform system that ensures that every child has a vision screen before entry to school (the US Preventative Task Force (USPTF) now recommends age 3). We invite you to spread the word that children’s vision matters: It opens eyes and opens doors.