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This book is being updated at a time of financial crisis leading to downward pressure on healthcare budgets. This means that pharmacists have to be even smarter at demonstrating their value for money and providing the evidence to underpin that. The value of clinical pharmacy services in terms of saving lives and saving money has been demonstrated, yet such services remain patchy in parts of central and eastern Europe. By contrast, many developing countries, particularly in Asia, are rapidly developing their clinical pharmacy with strong support from their health ministries.
The increased pressure of limited resources will continue to shape the way pharmacy is practised. Innovation continues to run ahead of our ability to fund new developments, and demands from an increasingly informed public who have ready access to medical information add to this pressure. Add to this the pressure to reduce our carbon footprint, and we have some challenges ahead.
These realities have created a fertile environment for the growth of evidence-based practice. Developments offering to identify effective interventions and providing the possibility of eliminating those activities which are ineffective or even harmful is clearly attractive to many, particularly those who purchase healthcare in some way or another. There is a further attraction in the belief that eliminating useless interventions will somehow free up resources for new developments. This is probably wishful thinking. However, other professionals regard the whole evidence-based movement as a threat to their right to practice.
The rapid unstoppable development of information technology has greatly facilitated the evidence-based healthcare movement. The rise of apps for tablets and smartphones makes information available at the fingertips. In 2012, some 250 000 medically related apps were downloaded worldwide. The internet provides worldwide and almost instant access across continents to high-quality evidence, good quality guidelines and protocols for treatment at little or no cost informing both professional and patient alike. Of course there is much that is bad or even downright dangerous. Who will help patients tell the difference?
Drivers for evidence-based healthcare
Consumerism has arrived in healthcare, almost certainly aided by the concept of an internal market where money was supposed to follow patients. The age of blindly following doctors’ orders is rapidly disappearing in many countries, and the relationship between doctor and patient is seen as a partnership with the patient often setting the agenda. Many users are demanding access to services at times that will not interfere with busy work schedules. Day surgery units are increasingly providing evening sessions, and private healthcare facilities are offering surgical procedures at a discount during times of lower demand, such as the Christmas break. Patients are becoming increasingly knowledgeable about their conditions. After receiving a diagnosis they may use the internet to gain further insight and to identify treatment options, often using the information as a bargaining tool with their clinician. New developments are featured on primetime news, on consumer programmes and in popular magazines leading to an increased demand for the latest and the best treatments as described by the media.
All these developments increase the pressure on healthcare services so that consultations need more time to deal with more evidence and the ensuing discussions (box 1).
Drivers for healthcare demand
Advances in health technology
Advances in other technologies including smartphones
Population ageing is another key factor increasing the need for healthcare. As the number of older people increases, the demand for healthcare increases simply because old people require more care. Expectations are changing. The generation of elderly who were mesmerised by the white coat is now largely replaced with those who have higher expectations and will not settle for poor or inadequate service. An older population will challenge some of our current practices; for example, we will need to offer renal transplants to an older age group than is often the case at present, and insert artificial hips not once but twice. There is on-going pressure to develop new effective treatments for some of the diseases of old age, such as Alzheimer's.
Advances in healthcare technology
Recent years have seen a number of developments targeted at diseases for which, in the past, there were either poor treatment opportunities or no treatment at all. Procedures that used to require long inpatient stays are now undertaken on a day care unit. New treatments for schizophrenia have improved the quality of life for many sufferers, and there are now treatments emerging for motor neuron disease, multiple sclerosis and Alzheimer's. New pharmacological interventions tend to be expensive, and some recent developments have not been as effective as had been hoped. It is likely that developments in the pipeline will be increasingly effective and costly.
While the high cost of interventions benefiting relatively small numbers of patients can have a major impact on budgets, equally difficult to manage are the ‘medium cost, medium volume’ developments. One current example is due to the explosion in diabetes and the development of Type 2 diabetes. In 2012, the cost of treating diabetes in the UK was €0.9 billion.1 This is from a total budget for that year of €140 billion. Many of these developments can, or have the potential to save costs elsewhere, such as reducing the need for hospital admissions, but certainly in the short term the impact is seen as an increased demand for resources.
There is a strategy to use new drugs—for example, direct thrombin inhibitor (rivaroxaban/dabigatran) instead of warfarin/low molecular weight heparin for deep vein thrombosis prevention after hip replacement. The drug is more costly but, overall, it saves money by prevention of deep vein thrombosis and monitoring. However, if the dosage/renal function is not assessed accordingly, the patient has a higher risk of a gastrointestinal bleed with potential harm to patients and additional costs to the healthcare budget.
The previous three drivers on demand for healthcare have an impact on professional expectations. Many professionals want to be at the leading edge in their own fields and are keen to gain experience of the latest developments. An anaesthetist is often keen to gain experience of the latest inhalation anaesthetic, and the oncologist wants to try out the latest chemotherapy. This desire to gain experience of use is not always rational or firmly based on good evidence.
Figure 1 illustrates what often happens. The enthusiast ‘evaluates’ a new intervention and quickly becomes the local expert on its use. The intervention is then incorporated into clinical practice, often quite quickly without the necessary evaluation.
More demanding patients’ expectations will inevitably lead to a rise in professional expectations. For example, clinical pharmacists in the course of their rounds are increasingly asked to provide more information on the potentially harmful effects of medication. The depth of answers required often goes beyond a cursory scan of the standard pharmacy reference books or even familiarity with one or two published papers on the subject.
What is the purpose of this book?
Professional practice has to be about doing the right things right. It seeks to answer the questions: ‘How do we know what the right things are and how do we know when we are doing them right?’ Take the example of blood level monitoring for a drug with a narrow therapeutic window such as an aminoglycoside. We need to know whether blood level monitoring is of any value, and if it is proven to be of value we need to know when the levels should be measured in the treatment plan and at what time blood should be taken in relation to dosing. Failure to take the blood at the correct time can result in doing the right thing (ie, monitoring blood levels) wrongly (because the action renders the result meaningless). Take another example of a 50-year-old patient with high blood pressure and several risk factors for cardiovascular mortality. According to National Institute for Health and Care Excellence (NICE) recommendations, an ACE-inhibitor or a low-cost angiotensin II receptor blocker is first-line treatment. But, if the patient has increased sympathetic drives, and is a woman of child-bearing potential, a β-blocker is probably a better choice; and if the patient is black, of African family origin, then a calcium-channel blocker is more appropriate. However, if the patient does not take the medicine or does not tolerate the treatment, there is a problem. Therefore, the current NICE guideline has also incorporated ‘patient education on medication adherence’ as part of hypertension management pathway. Results from clinical trials, systematic reviews and guidelines are based on population mean values, but not all patients will fit the population approach. Evidence-based medicine and pharmacy can help by using the evidence from systematic research to adapt care for an individual patient.
Evidence-based practice is all about first looking at the evidence based on populations and adapting the knowledge to practice and to the care of an individual patient or customer, while using the clinicians’ clinical expertise in the specialty.
This book seeks to guide the pharmacist to effectively work with physicians, patients and customers to improve the outcome of drug therapy. Additionally, tools and wisdom are presented to facilitate this.
Doing the right things
While the objective in healthcare is to do more good than harm, there are many examples of treatment making patients worse than before. Pharmacists are very aware of the need to balance benefit of an improvement with the risk of causing harm either temporarily or permanently. Interventions can have three possible outcomes (box 2).
The three possible outcomes for an intervention
Doing more good than harm
Doing more harm than good
Unknown, whether beneficial or not
Good implies effective, but it must also be safe and acceptable
Harm is self-evident but often overlooked in the intensity of treatment. The search for a cure can lead to long-term damage, which may not be reversible. There are many examples of new pharmacological developments where the benefits are actively promoted but the adverse effect profile only emerges over a considerable period of time. These effects may be caused by the active agent or by additional ingredients or vehicle used. One example was the use of a polyethoxylated castor oil vehicle for the intravenous anaesthetic agent alphaxalone (althesin). This vehicle was later associated with severe anaphylactic reactions, hyperlipidaemias and alterations in blood viscosity.
It is apparent that good and harm have to be weighed for each situation. A toxic treatment may be more acceptable in a life-threatening illness though patients may not always agree, and they need to be given the choice.
Underpinning all this should be an evaluation of the strength of the evidence, which informs the evaluation of benefit and risk. As will be shown in Chapter 2, if the expression of good or harm is only based on a personal opinion then it should be discarded. It is important to look for evidence, which eliminates the possibility of the result being chance, or of bias.
Table 1 outlines the action that needs to be taken when benefit and harm have been assessed.
Strategies to increase the good to harm ratio
The time delay from publication to acceptance for a new intervention may be many years. Antman et al3 compared the results of meta-analyses of randomised controlled trials with clinical expert recommendations for the use of thrombolytic therapy in the treatment of myocardial infarction in a paper which has become a classic. Figure 2 lists the publication dates of the trials, the numbers of published trials and the overall OR showing benefit. The figure shows that in 1975 there was clear evidence of effect. This evidence becomes increasingly stronger as more studies are published.
Even though it was shown that thrombolytic therapy was beneficial in the mid 1970s, these results were not routinely used until the late eighties—a delay of around 10 years. The tragedy is that knowledge has not been disseminated or incorporated into the textbooks in a timely fashion. This phenomenon, clearly illustrated by Antman et al3 is displayed in figure 3.
The key lesson in this is not the indiscriminate use of new interventions but the careful appraisal of evidence and decision making based on this appraisal.
The strategy discussed above is based on a population approach. As a practitioner, it is important to take account of the individual patient or customer. The principles of pharmaceutical care4 focus on the pharmacist's responsibility to improve the patients’ quality of life. Even if the best and most safe medication is selected, there is a potential for either lack of efficacy or adverse events in an individual patient. A medicine for a patient should be considered as a ‘trial’. Once the risks and benefits have been identified it should be possible to identify treatments where the benefits outweigh the risks.
At the same time, proven clinical expertise and specialised pharmaceutical aspects put pharmacists in a strong position to provide evidence-based healthcare. Clinical expertise in each field is important to appraise the evidence in the context of each specialty. Physicians are frequently the first-line clinical staff who may see the ‘apparent’ harm done to a patient. In the example of the thrombolytic therapy, bleeding is an adverse effect.
Three battles that pharmacists should need to note
Eddy,5 writing primarily about health reforms in the USA, describes three battles that he saw emerging, and even though the arguments were made some time ago, they are still worth considering. He argued that the three main goals of healthcare reform are access, quality and cost, but that cost, particularly the rate of increase of cost, is the driving force. The fact that budgets are consistently overspent suggests that healthcare is becoming an uncontrollable item. Eddy further points out that cost will never be controlled unless the decisions made by physicians about treatments, in the widest sense, are challenged and controlled. This will lead to battles over what practitioners do and how they do it. He sees the three battles in this area focusing on the three topics of evidence, costs and physician autonomy.
Three battles in healthcare reforms (Eddy)
Eddy argues evidence is the first battle that is made up of three components.
How much and what type of evidence is sufficient to justify a treatments use? This covers issues such as what type of evidence is required and how much. If enough experts declare a treatment valuable, is this sufficient?
The second component surrounds the burden of proof. Should a treatment be considered ‘on trial’ until there is sufficient proof or should it be considered effective until proven otherwise?
The third component is about what we should do with old treatments. The weight of research funding is being applied to new and innovative treatments but there are hundreds of treatments, which have gained acceptance in days when the burden of proof required was far less. This may account for 80–90% of available treatments according to Eddy. Because we do not have the evidence, we do not even know what we do not know.
The cost of healthcare is a major problem for any government. The battle takes place on several fronts as we saw with evidence. The most familiar is knowing where to draw the line for the intensity of treatment or for groups of patients. New drug developments in the UK together with somewhat equivocal supporting evidence have led to the so-called ‘postcode prescribing’ phenomena where access to treatment largely depends on which health authority catchment area a patient happens to reside in. This occurs where at least in theory patients who live in an area that happens to be on a health authority border can have different treatments or even no treatment because of a decision by their health authority. A similar debate is currently taking place over the use of statins. Many would argue that they should be kept for secondary prevention of patients with hyperlipidaemia who have already suffered a myocardial infarct, but others argue that it is cost effective to treat hyperlipidaemic patients with a number of risk factors before the myocardial infarct occurs. If one subscribes to the second argument then a decision has to be made about the cholesterol level or combination of risk factors that determine should lipids be started. There are no easy answers.
The second cost issue dilemma exists when there are several treatments for the same problem. This occurs where benefits may differ slightly but costs differ a great deal. Staying with the statins, some argue that their action is a class effect, so it does not matter which one is used, while others argue that only the more expensive brand leaders should be used, because they are supported by large randomised controlled trial evidence. Somehow, Eddy argues, there has to be a trade-off between quality and cost.
The third area Eddy calls is ‘It's not much but it's all we've got’. This is where there is only one treatment, but the benefits are small even when compared with no treatment. The most difficult of this type are the last-hope treatments for cancer. These are the ones that can be very expensive, are extremely emotive, rarely produce benefit and can hit the headlines!
Eddy's fourth point on cost is ‘It's all we've got and it is a lot’. He mentions the case of Gaucher's disease a rare genetic disease (about four cases per million in the population) caused by a deficiency of glucosylceramidase. The results of the disease in children are severe neurological problems and early death. There is now a replacement enzyme available at a cost of up to US$200 000 per annum, possibly for the rest of a patient's life. A similar argument surrounds the use of total parenteral nutrition for the patient who has undergone a total gastrectomy. While not in the same cost league as Gaucher's disease, the costs involved are a significant sum for any health authority to bear. Remember, money in any budget can only be spent once.
Eddy's final point is on what he calls futile care, treatments that have no reasonable possibility of providing any satisfactory quality of life to the patient. These are almost invariably administered at the wish of a patient's relatives who want everything possible to be done, or for some sociological argument that it is unacceptable to give up. Eddy reckons that tens of billions of dollars are wasted that could be reallocated to provide greater benefit to a wider range of patients.
It is likely that treatment guidelines become increasingly prominent to govern some of these scenarios.
Eddy's third battle is physician autonomy. This is perceived by many clinicians to be one of the most desirable features of medical practice and is defended by the argument that physicians use their best judgement to provide the best possible care for an individual patient. The battles around evidence and cost challenge this area because judgement may not be evidence-based or even correct, and there may be pressure to get best value for money from limited resources with the strongest arguments coming from those who are not clinicians. Eddy believes that many practitioners will understand that it is no more insulting to use good quality guidelines than obtaining advice from a journal. However, pressure may come from administrators who have an eye on the bottom line, and medical colleagues who will wish to rein in those who take more than their fair share from the global pot.
So where are we going?
There is a choice, either to do nothing or to take action to shape the future. Pharmacy is well placed to be involved with the management of innovation and knowledge. There is no reason why pharmacists could not be leading the cultural transformation of the health service by influencing decisions based on current best knowledge on drugs both in terms of benefits and adverse events.
Pharmacists can add significant value to these processes. However, much of the arguments put forward by Eddy have direct relevance for pharmacists. Clinical pharmacists are already seeking to help clinicians juggle limited resources in secondary care, and physician autonomy has largely disappeared, but pharmacy will need to be sure that its own house is kept in order. For hospital pharmacists, the challenge of skill mix to ensure that pharmacists are put to the best use is uppermost as resources continue to be squeezed. One of the reasons that clinical pharmacy has been able to develop is because technicians have taken over many tasks that pharmacists used to perform.
There is still a need to be constantly evaluating what we do and why we do it in order to meet the demands of healthcare in the future. There is also a need to be aware of major new developments so we can change to keep in step. Some strong proponents of change, such as Gray,6 are already arguing that services will transform from an institution-based care to a population-based care. This would make care much more patient-centred, and a named pharmacist may well support a patient through their journey from primary care into secondary care and back into primary care—imagine what that would do to our pharmacy structures! There are further challenges to be faced, such as who will be the knowledge managers of the future. The challenge in community pharmacy is probably greater, the future is likely to belong to those who are willing to develop services, take risks and then seek funding rather than the common attitude of wanting money up front. Current funding mechanisms need to move away from the payment per dispensing item method; greater use will need to be made of technical support.
One thing is sure; whatever is developed will increasingly need to be supported by reliable evidence derived from top quality research.
Evidence-based Pharmacy was first published as a textbook by Phil Wiffen in 2001. Plans to bring it up to date have stalled on several occasions until recently a colleague suggested adding new authors and publishing in a serial form in EJHP. Both the EJHP editorial board and members of the EAHP board expressed enthusiasm. We present the first chapter here. Our aim is to present a chapter in each issue of EJHP. In addition to bringing the chapters up to date, we will add new material. There are plans to translate the final completed manuscript into Chinese to reach the rapidly developing clinical pharmacy initiatives in China.
Phil Wiffen is editor-in-chief of EJHP and also teaches methodology for EBM and systematic reviews.
Tommy Eriksson is Professor in Clinical Pharmacy and Program Director of the MSc pharmacy program at Lund University in Sweden
Hao Lu is a clinical pharmacist based at the Beijing United Family Hospital in China
Phil Wiffen is editor-in-chief of EJHP and also teaches methodology for EBM and systematic reviews.
Tommy Eriksson is Professor in Clinical Pharmacy and Program Director of the MSc pharmacy programme at Lund University in Sweden.
Hao Lu is a clinical pharmacist based at the Beijing United Family Hospital in China.
Competing interests None.
Provenance and peer review Commissioned; internally peer reviewed.
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