Pharmaceuticals: The Good and the Bad

Every drug is a triangle with three faces, representing the healing it can bring, the hazards it can inflict and the economic impact of each. All of us—doctors, patients, regulators, taxpayers, insurers and policy makers—must learn how to balance these three dimensions better if we are to get the maximum benefit from this most common and powerful of all health care interventions.

—Jerry Avorn, Powerful Medicines: The Benefits, Risks, and Costs of Prescription Drugs


The pharmaceutical industry highlights the complexity of the health care system and its interactions. Academics, manufacturers, research scientists, patient advocates, and customers all play a role, and the issues surrounding the development, regulation, marketing, and use of pharmaceuticals are often contentious.


Pharmaceuticals are the most common medical intervention, and their potential for both help and harm is enormous. Ensuring that the American people get the most benefit from advances in pharmacology is a critical component of improving the national health care system.

BAD DRUGS OR A BAD SYSTEM?

Pharmaceuticals are so prevalent in modern medicine that they are colloquially called just that: medicine. In any given wefi, four out of five U.S. adults will use prescription medicines, over-the-counter drugs, or dietary supplements. Nearly one-third of adults will take five or more different medications. Most of the time, medications are beneficial—or at least benign—but this isn’t always the case. Injuries to the human body due to medication—or adverse drug events (ADEs)—are inevitable. Not surprisingly, the most powerful medications, such as chemotherapy treatments, are also the most likely to have significant harmful side effects. Equally troubling is the harm that results from human errors in prescribing or taking a broad range of medications. These mistakes can, and should, be minimized.


In hospitals, medication errors are more common than they should be at every step: they occur when procuring the drug, prescribing it, dispensing it, administering it, and monitoring its impact. Estimates of preventable hospital-based ADEs run from 380,000 to 450,000 a year, and they skyrocket to 800,000 a year



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PharMaCeutICals: the Good and the Bad Pharmaceuticals: The Good and the Bad Every drug is a triangle with three faces, representing the healing it can bring, the haz- ards it can inflict and the economic impact of each. All of us—doctors, patients, regu- lators, taxpayers, insurers and policy makers—must learn how to balance these three dimensions better if we are to get the maximum benefit from this most common and powerful of all health care interventions. —Jerry Avorn, Powerful Medicines: The Benefits, Risks, and Costs of Prescription Drugs The pharmaceutical industry highlights the complexity of the health care sys- tem and its interactions. Academics, manufacturers, research scientists, patient advocates, and customers all play a role, and the issues surrounding the develop- ment, regulation, marketing, and use of pharmaceuticals are often contentious. Pharmaceuticals are the most common medical intervention, and their po- tential for both help and harm is enormous. Ensuring that the American people get the most benefit from advances in pharmacology is a critical component of improving the national health care system. Bad dRUgS oR a Bad SyStem? Pharmaceuticals are so prevalent in modern medicine that they are collo- quially called just that: medicine. In any given week, four out of five U.S. adults will use prescription medicines, over-the-counter drugs, or dietary supplements. Nearly one-third of adults will take five or more different medications. Most of the time, medications are beneficial—or at least benign—but this isn’t always the case. Injuries to the human body due to medication—or adverse drug events (ADEs)—are inevitable. Not surprisingly, the most powerful medications, such as chemotherapy treatments, are also the most likely to have significant harmful side effects. Equally troubling is the harm that results from human errors in pre- scribing or taking a broad range of medications. These mistakes can, and should, be minimized. In hospitals, medication errors are more common than they should be at every step: they occur when procuring the drug, prescribing it, dispensing it, ad- ministering it, and monitoring its impact. Estimates of preventable hospital-based ADEs run from 380,000 to 450,000 a year, and they skyrocket to 800,000 a year 

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Informing the Future: Critical Issues in Health in any gien week, four out of fie in long-term care facilities. Medicare outpatients U.S. adults will use prescription experience an estimated 530,000 ADEs each year. medicines, oer-the-counter drugs, Not only are these mistakes expensive, but they or dietary supplements. nearly one- also have a serious impact on the health of pa- third of adults will take fie or more tients and the health care system as a whole. different medications. UndeRStanding the PRoBlem Recognizing this problem of medical errors, Congress urged the Centers for Medicare and Medicaid Services (CMS) to contract with the Institute of Medi- cine (IOM) to explore its extent and analyze possible solutions. Preventing Medica- tion Errors: Quality Chasm Series (2007) documented the surprising frequency of such errors and their cost to the nation. The report estimated that at least 1.5 million preventable ADEs occur in the United States each year. When all types of errors are taken into ac- count, the average hospital patient can expect to be subject- ed to at least one medication error per day. While only a frac- tion of these errors are significantly harmful to the patient, the report estimated that the costs of treating drug-related injuries in hospitals alone were $3.5 billion a year, not includ- ing lost wages, lost productivity, or additional health care costs. These dramatic facts resulted in media attention from National Public Radio to Rush Limbaugh and government at- tention from the halls of Congress to those of the Food and Drug Administration (FDA). The report also led to the IOM’s recognition by national pharmacist groups with their “Safe-Rx Evangelist Award.” The report outlined a comprehensive strategy to minimize this problem. Be- cause medication errors are the result of multiple points of system failures, effec- tive prevention will require changes on the part of the pharmaceutical industry, doctors, nurses, and pharmacists, as well as the organizations that support them, including hospitals, nursing homes, the FDA, and other government agencies. These stakeholders must become partners with the patients themselves to develop a solution. Reducing errors will come from fundamental changes in patients’ at- titudes and expectations. Clear, complete, and . . . the aerage hospital patient can continuing communication between patients expect to be subjected to at least one and their health care providers is central to drug medication error per day. safety. 

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2. Regulatory Review 1. Research and Development Private-sector funding • ANDA or AADA (generics) Public-sector funding • NDA or BLA (new drugs Preclinical testing (animals) • and biologics) Accelerated track and • Marketing approval granted • IND application dietary supplements Phase I Trials (healthy humans) • • Postmarket surveillance Phase II Trials (small scale, humans w/ illness) • • Labeling and advertising Treatment IND review Phase III Trials (large scale, humans w/ illness) • Switch status to OTC • Generic bioequivalence Clinical trial ts en ev registry Product rse ve withdrawal ad g rtin po Re 4. Medication Use (hospital, ambulatory, home health, 3. Manufacturing, Marketing, and Distribution Reimportation and long term, self-care) supply shortages • FDA inspection • Physician prescribing and self- Documentation Production to economies of scale • prescribing Shipment to wholesalers • • Dispensing Shipment to pharmacies • • Administration and consumption Payer formulary and reimbursement and PBMs • • Monitoring for effects • Reporting errors and adverse effects Journal articles, academic investigators, accreditation organizations, media, patient safety organizations Marketing to doctors and public, Postmarket surveillance distributing product samples, studies of medication use DTC advertising Figure 2.1 Four stages of the drug system. SOURCE: Preventing Medication Errors, p. 52. Landscape view revised 4/27 moving towaRd a SolUtion 5/24 and The path to widespread use of a drug starts with FDA approval. As recent drug safety issues have made evident, the nation’s system for deciding what drugs are approved for marketing—and under what conditions—needs a major overhaul. The Future of Drug Safety: Promoting and Protecting the Health of the Public (2007) powerfully analyzed the current state of drug regulation and created a blueprint for change. The current problems stem partly from steps taken 15 years ago that were intended to speed up the FDA’s drug approval process. The Prescription Drug User Fee Act (PDUFA) imposed fees on drugs submitted to the FDA for review and approval; sponsoring companies have to pay the FDA to review their drugs. These increased funds from PDUFA fees were used to expand FDA staff, and they have succeeded in speeding up the process of drug approval. Unfortunately, the 

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Informing the Future: Critical Issues in Health PDUFA legislation was written so that very little the iom committee recommended of this new funding could be used for postmar- that the fda’s authority to ket drug safety surveillance. So while new drugs successfully monitor postmarket have been entering the market more quickly, safety be clarified and their resources the FDA’s capacity to provide adequate safety and expertise strengthened to enable monitoring and surveillance after the drugs be- effectie sureillance and rapid action come available for general prescription has been when problems are identified. under-resourced and overwhelmed. Because initial safety testing is limited to small groups of volunteers in order to minimize the risks of a new drug with unknown side effects, post-marketing surveillance is critical. Initial test groups are far too limited, and the study pe- riods far too brief, to understand the effects that a drug may have on a diverse population of users when taken over an extended period. Not surprisingly, safety problems have been discovered with some drugs long after marketing approval. These safety is- sues have had tremendous costs, both in dollars and, far more importantly, in lives lost or persons harmed by the drugs. The IOM committee recommended that the FDA’s au- thority to successfully monitor postmarket safety be clari- fied and their resources and expertise strengthened to en- able effective surveillance and rapid action when problems are identified. It noted that Congress should grant the FDA the ability to require postmarket risk assessment and risk management programs, so that the FDA can do more than just ask drug companies for help. The committee also rec- ommended the creation of regulations to limit direct-to-consumer advertising of new drugs until population-specific risks are understood as well as increased enforcement authority for the FDA, including better tools to ensure that inappro- priate promotions and advertising can be quickly stopped. Specifically, the committee recommended an array of actions, including • symbols to alert consumers to new products and denote heightened regulatory attention; • efforts to restore balance in the FDA between the resources necessary to approve new drugs and the resources necessary to monitor their safety once on the market; 

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Pharmaceuticals: The Good and the Bad • tools to hold industry and researchers accountable for making results of a drug safety study public, including mandatory registration of clinical trial results; • new rules to stabilize the leadership of the FDA by appointing commis- sioners for six-year terms; • an increased role and formal responsibilities for the FDA’s post-marketing drug safety staff; and • an increase in funding and staffing for the agency. The report provided the template for major reforms enacted by Congress. It continues today to inform the debate over post-approval drug surveillance in both the House and the Senate. In addition to these studies, the IOM has organized roundtables, forums, and public workshops that provide opportunities for government, academic, indus- try, and consumer experts to meet and share knowledge on critical issues (see Chapter 9). Although these activities do not yield reports, each does provide an invaluable opportunity to focus on important questions, explore new alterna- tives, and consider options that may be available to the government or private sector. For example, the Forum on Drug Development, which looks at ways to improve the pharmaceutical system, has convened workshops on • Emerging Safety Science (April 23, 2007); • The Future of Drug Safety: Challenges for the FDA (March 12, 2007); • Addressing the Barriers to Development in Pediatrics (June 13, 2006); • Understanding the Benefits and Risks of Pharmaceuticals (May 30, 2006); and • Adverse Drug Event Reporting (November 3, 2005). 

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