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FDA Safe Use Initiative

Joshua Sharfstein, M.D.

U.S. Food and Drug Administration


The Food and Drug Administration (FDA) is a public health agency committed to protecting lives wherever possible, said Joshua Sharfstein. Since its inception, people have looked to the FDA to take action when there are deaths related to medications. Now the FDA is looking to the roundtable and other stakeholders for critically important input on its Safe Use Initiative, which is in its formative stages.

Launched in November 2009, the Safe Use Initiative (FDA, 2009b) aims to minimize the risk of medications to improve their ratio of benefits to risks. If successful, many lives will be saved, injuries and suffering will be prevented, and health care costs will decrease.

The Safe Use Initiative is not a broad, amorphous concept. The idea is to tackle one drug at a time in order to make progress, working within structures and systems to achieve the goal of patient safety. Janet Woodcock and her team at the FDA Center for Drug Evaluation and Research (CDER) unveiled the approach in a concept paper, FDA’s Safe Use Initiative, Collaborating to Reduce Preventable Harm from Medications (FDA, 2009a). The aim of the Safe Use Initiative is to identify specific preventable problems related to medication use and identify specific metrics that can measure progress; and to do it all by developing collaborations.

Partnerships will be important in the Safe Use Initiative. Clearly the clinical community is an important partner. Pharmacies have a very important role. Insurers should be engaged as they have the access to patients and know what is being prescribed. Pharmaceutical companies also have a vested interest in seeing their medicines used well. Patient groups and



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2 FDA Safe Use Initiative Joshua Sharfstein, M.D. U.S. Food and Drug Administration The Food and Drug Administration (FDA) is a public health agency committed to protecting lives wherever possible, said Joshua Sharfstein. Since its inception, people have looked to the FDA to take action when there are deaths related to medications. Now the FDA is looking to the roundtable and other stakeholders for critically important input on its Safe Use Initiative, which is in its formative stages. Launched in November 2009, the Safe Use Initiative (FDA, 2009b) aims to minimize the risk of medications to improve their ratio of benefits to risks. If successful, many lives will be saved, injuries and suffering will be prevented, and health care costs will decrease. The Safe Use Initiative is not a broad, amorphous concept. The idea is to tackle one drug at a time in order to make progress, working within structures and systems to achieve the goal of patient safety. Janet Wood - cock and her team at the FDA Center for Drug Evaluation and Research (CDER) unveiled the approach in a concept paper, FDA’s Safe Use Ini- tiative, Collaborating to Reduce Preventable Harm from Medications (FDA, 2009a). The aim of the Safe Use Initiative is to identify specific preventable problems related to medication use and identify specific metrics that can measure progress; and to do it all by developing collaborations. Partnerships will be important in the Safe Use Initiative. Clearly the clinical community is an important partner. Pharmacies have a very impor- tant role. Insurers should be engaged as they have the access to patients and know what is being prescribed. Pharmaceutical companies also have a vested interest in seeing their medicines used well. Patient groups and 

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 THe SAFe USe InITIATIVe AnD HeALTH LITeRACY consumer groups must also be engaged, as should those involved in the development and deployment of electronic medical records (EMRs). It is the FDA’s job to propel this initiative forward, highlighting the issue of safe medication use because there are lives to be saved. Medica- tion injuries can occur in two scenarios. In the first scenario, questions are raised about the benefit/risk balance for a particular drug. In the second scenario, medication-related injuries include cases where the benefit does exceed the risk, but unnecessary injuries still occur. In the benefit/risk balance scenario, the FDA has the authority to pull the drug from the market or to put new safety restrictions on the use of the drug. Restrictions are deemed necessary for making the benefits exceed the risks. The FDA can restrict sales to a single pharmacy, or make sure doctors obtain training, and make sure patients are informed. Restrictions have been put in place for several medications because the FDA believes those drugs cannot be safely marketed without these restrictions. The second scenario for medication injuries where benefit does exceed the risk has been overlooked historically, but there are lives that can be saved by reducing the risks of these medicines. Examples of this category include children who ingest adult medicines or patients who experience difficulties with warfarin or insulin. This second category has been over- looked historically, but we know that there are lives that can be saved by reducing the risks with these medications. If systems can be put in place to make medication use safer in practice, then regulation is less necessary. The FDA prefers not to have to employ burdensome restrictions on use to keep medications safe. The Safe Use Initiative is about identify - ing areas where there are unnecessary, preventable medication adverse events, developing a coalition, setting clear goals, and accomplishing real things so people are not injured. The agency wants to be able to measure progress. One way to do this is to monitor what happens in emergency rooms in the United States before an action is taken and how those mea - sured events change after the action. Since launching the Safe Use Initiative in late 2009, the FDA has been holding listening sessions to obtain input from the public and professional groups about the kinds of issues the initiative should address. The FDA also wants to know about existing efforts and ongoing activities. Having FDA become part of the process may be just the extra push that is needed to move the activities forward. For example, professional societies, phar- macies, insurers, and many others have guidelines for making decisions based on evidence. How can such existing knowledge and energy be channeled to reduce medication harms? Karen Weiss, director of the Safe Use Initiative, is here to participate

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 FDA SAFe USe InITIATIVe in the conversation, Sharfstein said. Others from the FDA are here as well. There is also an open docket for comments on the Safe Use Initiative. 1 DISCUSSION Isham started the discussion, saying that the Safe Use Initiative is about identifying specific preventable problems related to medication use, developing cross-sectoral interventions to reduce harm, identifying specific metrics that can measure progress against actions, and doing all of this through collaborations. Sharfstein added that preventable injuries include such things as a patient being on a medicine that his or her doctor does not know about, or a doctor incorrectly prescribing a medicine, or children taking medicines they should not be taking. All of these scenarios are preventable. Will Ross from the Washington University School of Medicine asked for greater involvement from developers of EMRs. For example, there needs to be a linkage of EMRs and appropriate dispensing and accurate recording of medications. Sharfstein responded that there is a great deal of evidence that EMRs can be used to avoid specific medical errors. Deter- mining how the tools of EMR could help with educating physicians is difficult but important. Cindy Brach of the Agency for Healthcare Research and Quality (AHRQ) said the IOM Roundtable on Health Literacy has engaged in a number of activities focused on communications between clinicians and patients around the problems of prescription medication labeling, areas in which health literacy plays a major role. How, she asked, does health literacy specifically fit in with the Safe Use Initiative? Sharfstein responded that it is unlikely anyone would defend the kind of confusing information patients currently receive, and the FDA is pursuing some regulatory initiatives with respect to labeling. The FDA is open to input from the roundtable. But once labels have appropriate information, what other tools are needed to make the information meaningful and useful to people? Are there electronic ways to make it useful? How can the useful - ness of the information be measured? Are there targets to focus on to see if health literacy efforts can prevent adverse events? For example, if the FDA changes the information that is disseminated on warfarin and that change helps people really understand the need to come back to get their levels checked, then they avoid the bleeding risk and trips to the emer- gency room; that is the Safe Use Initiative. Isham asked how the FDA will create the coalitions. How can inter- ested people get involved and stay informed about what is happening 1 See http:/ www.regulations.gov/search/Regs/home.html#documentDetail?R=0900006480a517a4. /

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 THe SAFe USe InITIATIVe AnD HeALTH LITeRACY with the initiative? Sharfstein expects that information about the different efforts is going to be available on the FDA Safe Use Initiative website. 2 However, the FDA does not envision convening the different collabora - tions that are needed. It does not want the Safe Use Initiative to be consid- ered a regulatory initiative. The FDA would like to see so much interest and activity that it can tag along and ask how it can help. It is the FDA’s job to highlight the need for the Safe Use Initiative, to put the initiative before the public, and to emphasize that lives can be saved through work in this area. But then the agency wants potential collaborators to come forward to identify how the FDA can help support the efforts of others in different ways. The FDA wants to know if there are particular issues it should address, what outcomes need to be tracked, and which partners should be involved. For populations with mental illness in residential outpatient rehabili - tation, their mental health care needs to be coordinated with their medi - cal care, said Carolyn Cocotas, Quality Corporate Compliance at F.E.G.S. Health and Human Services System, a nonprofit health, education, and human services organization. It is a challenge. Clients are often on psy - chotropic drugs and dealing with treatments for chronic conditions such as heart disease or diabetes. She asked if the FDA has given thought to coordination around medical and behavioral health care. Sharfstein said the question raises two important issues. First, it is not sufficient to think about safe use related to specific diseases or medications alone without also thinking about different populations of patients. The issues may be different for different ethnic populations, or, as was mentioned, for those with both medical and behavioral health issues. Methods that work for one group might not be effective for another. When developing interven- tions, it is important to engage people who really understand the needs of different groups. The other issue is the idea of combination medicines. Combinations medicines are a challenge for the FDA because these medicines must be regulated one at a time. This is an issue that the medical community can weigh in on because they have the necessary tools to educate physicians in this area. The FDA wants to address both physical and mental health issues, which is challenging because the usual methods of communication may not be useful, Sharfstein noted. And the issues of drug combinations are also important. What kinds of systems should be in place for patients on multiple medicines to alert their doctors to problems, he asked. Rather than thinking about the whole challenging system, Sharfstein said he would, for example, choose to begin by identifying particular types of 2 See http://www.fda.gov/Drugs/DrugSafety/ucm187806.htm.

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 FDA SAFe USe InITIATIVe drug combinations used by people who are severely mentally ill that cause trouble for them. A structure for addressing that particular problem could be identified and then, perhaps, be used to address other problems. Of major importance is determining who needs to come to the table to move such a project forward. Ruth Parker, at the Emory University School of Medicine, said that the first IOM report on health literacy (IOM, 2004) stated there are a large number of people who can not do what is necessary to use medications safely and effectively for a variety of reasons. Another way of saying this is, it is hard to be a patient and it is easy to mess up. Now, however, it is being realized there is more to health literacy than a focus on the indi - vidual and his or her capabilities. This other focus is the demands and complexities of the system and what the system is asking consumers to do. How can the FDA help shine a light on the need for a health literate system for safe use? Sharfstein agreed that critically important information must reach patients in a way that they can use, and that it needs to be made relevant for different groups of patients. He said this is an area in which the FDA could seek guidance through collaborations with others. Sharon Barrett of the Association of Clinicians with the Underserved suggested that when talking about different population groups and dif - ferent cultures, limited English proficiency is an important issue. Safety net providers are often the ones who see individuals with low literacy and limited English proficiency. They need to participate in efforts aimed at safe use. They are the ones who deal with populations who may have to use medications that could potentially harm them if they don’t under- stand how to use them. Sharfstein suggested that members of the round - table could provide input on who should be at the table so that no one is left behind. The FDA needs help with coalition building, since that is not its historical strength. The U.S. Pharmacopeia Health Literacy Advisory Committee has developed recommendations for patient-centered labels for prescription drugs (USP, 2010), noted Cindy Brach. The recommendations are specific as to what a patient-centered label should look like and what it should and should not include. How, she asked, could the FDA use that standard? Currently the information on the label serves the pharmacy more than the patient. The labels are not regulated, and the FDA has said it is not in its bailiwick to regulate prescription labels as opposed to OTC labels. Sharf - stein replied that the FDA has made recommendations to state pharmacy boards on labels, and if there are things that mislead patients they need to be fixed. What productive role could the FDA play? Even if the FDA role is not regulatory in this case, it should be able to bring people together

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 THe SAFe USe InITIATIVe AnD HeALTH LITeRACY and catalyze action. Isham offered that a small group from the roundtable could meet with relevant FDA staff to explore this further. Sue Johnson from the FDA added that the agency has regulatory authority with regard to properly labeling prescription medications. A strong coalition around this issue makes sense. Isham reminded partici - pants that the roundtable does not make recommendations, but it can cre- ate conversations, stimulate interest, and influence people to take action. Winston Wong, Kaiser Permanente, asked if there are timelines or milestones for building coalitions and stakeholder involvement in the Safe Use Initiative. What are some of the sentinel things to look for regarding progress? Sharfstein said he envisions a collection of discreet, focused efforts around different medication issues. Some are moving forward now. Sharfstein will determine success by counting how many of the initiatives are going on, whether they have clear goals and are making changes happen, and assessing whether the goals are being met. This is a big umbrella project, and progress will only occur one drug at a time and one set of adverse reactions at a time. Whether people are healthier at the end of the initiative will be the measure of success. The FDA’s Weiss added that because the agency’s main role is regula- tory oversight and enforcement, it is not in the exam room when a physi- cian decides to prescribe a long-acting opioid when a less-potent medi - cation would be better. And it is not at the pharmacy when the patient is looking at an acetaminophen-containing medication and is confused about whether he should take two every 4 hours or four every 2 hours. The FDA wants to work in partnership with the greater community of health-care providers, practitioners, and consumers who are in the room when the medications are being prescribed, paid for, and used. These groups need to identify the big safety issues. The FDA is asking for input at Let’s Move sessions with various health care communities. It started with some professional societies, pharmacy groups, and nursing com- munities. More will come this fall, and the FDA will learn about things already going on that FDA can help move forward. Specific things the FDA is working on through regulatory activities include misprescribing long-acting beta-agonists for children with asthma (Chowdhury and Pan, 2010). Safe prescribing of long-acting, sustained- release opioids is another area. There are risk evaluation and mitigation strategies (REMS) required of manufacturers to ensure that the benefits of a drug or biological product outweigh its risks being developed as an educational component for health-care providers. What else can the FDA do to compliment REMS, which only go so far? The FDA is working with the Acetaminophen Awareness Coalition to disseminate and communi- cate information about safe use of acetaminophen and acetaminophen- containing products. These are just some examples.

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 FDA SAFe USe InITIATIVe Lee M. Sanders, Associate Professor of Pediatrics at the University of Miami, runs a federally funded (Title V) program that coordinates care for more than 10,000 children with special health-care needs, all of whom have parents with low literacy or limited English proficiency. Can the FDA reduce confusion by serving as a warehouse for low literacy point- of-care tools for parents—such as an easy-to-use personal health record and easy-to-understand health information for special needs children, Sanders asked? Sharfstein replied that under existing authority, the FDA is very inter- ested in this. The agency has a pilot program on sending the MedWatch bulletin3 to mobile devices for clinicians. There is an effort to make sure the information the FDA has is available for use by clinicians and others. One approach would be to make sure the National Library of Medicine has the information. For patients, the FDA would like guidance from the group as to what the FDA’s role could be. There are many ways to inter- act with patients other than at the point of care. For example, cell phones might be a potential tool for interaction. It would be very helpful for the roundtable to identify partners and convene discussions about where and how patients get into trouble with medications. Margaret Loveland, a pulmonologist now working for Merck & Com- pany, stated that clinicians are often reluctant to report adverse events because they believe such events indicate they did something wrong. Also, sometimes physicians stop prescribing a drug or patients stop tak - ing a drug, with disastrous consequences, because of publicity about adverse events. Loveland said that the FDA and health literacy advocates need to make sure clinicians and the public understand that there are many causes of preventable adverse events and that reducing such events requires the collaboration of many types of people. Sharfstein responded that it is important to make clear that one of the goals in reducing the risk is not reducing the benefit at the same time. The FDA does not want to cut out appropriate use of medications; rather it is attempting to decrease inappropriate use. Isham said that it might be useful for the roundtable members to iden- tify one or two issues and the kinds of individuals who could be brought together to stimulate discussion of those issues in a way that includes health literacy as one aspect. It could be that bringing individuals together for discussion might lead to those individuals deciding to form a coalition to pursue the issue further. Sharfstein responded that the FDA does wish to facilitate that kind of activity. Arthur Culbert from Health Literacy Missouri described a coalition of states that is organizing around the issue of health literacy. The coalition 3 See http://www.fda.gov/Safety/MedWatch/.

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0 THe SAFe USe InITIATIVe AnD HeALTH LITeRACY will include as many as 20 states and provides an excellent opportunity for partnering with the FDA at the state level. Sharfstein encouraged con - tact with Weiss to discuss this further. Bernard Dreyer from the New York University School of Medicine said that he is concerned that, other than the issue of vaccines in the United States, children are viewed as basically healthy with very few problems. We know in pediatrics that medication errors are generally the result of parent confusion about how to administer over-the-counter and prescription medications. Most of these errors do not lead to mortality, even though they do result in adverse events. When you speak of the Safe Use Initiative, he said to Sharfstein, you mention saving lives, and if that is the criteria by which the FDA chooses issues of importance, then medi- cation errors in children will not rise to the top as a key issue. Yet those children do end up in emergency rooms and require medical treatment. Sharfstein responded that while he emphasized saving lives in his presentation, other outcomes are extremely important. In fact, there is a tremendous amount of effort being expended on children’s use of OTC medicines. The FDA very much supports the efforts of the American Academy of Pediatrics (AAP) and encourages AAP to bring together individuals to address the issue of safe use of medicines in children.