Lorne Basskin, PharmD

						Pharmacists have specialized clinical and advanced training that enables them to monitor and adjust drug therapy while working in collaborative relationships with physicians and patients.

						Although hospitals have used clinical pharmacists for years, recent legislation enacted in several states now enables community pharmacists to provide clinical services through collaborative protocols with physicians.

						Most community collaborative protocols between a physician and a pharmacist are narrow in scope, limiting pharmacist clinical services to specified patients and disease states.

						Clinical services include some or all medication monitoring, adjusting doses and regimens, and possibly changing prescriptions.

						Most collaborative protocols require specific record keeping and communication between physician and pharmacist.

						Legislation differs in each state regarding the disease states that may be managed, the nature of the services to be provided, the wording of the protocol and need for registration, and the setting in which the services can be provided (ie, retail, hospital, clinic, etc.).

						Opportunities exist for enhancing customer relations and increasing revenue by billing for services through Medicaid or medication therapy management (MTM) through Medicare Part D in some state programs.

						Adequate training, a private area for patient consultations, a system for documenting patient and physician interactions, and extra support to cover drug distribution activities while the pharmacist is counseling are all necessary for pharmacist clinical services to be successful.

Thirty years ago, a pharmacist’s primary role was safeguarding and distributing medications. The term “clinical pharmacy” first entered the hospital pharmacist lexicon in the mid-1980s; pharmacists, typically in the hospital setting, would interact with physicians and make recommendations on drug regimens to avoid adverse drug reactions or interactions, in addition to enforcing the formulary. Other services included making recommendations to the physician regarding: 

		Dose adjustments for patients with renal or hepatic failure

		Monitoring patients for adverse drug events

		Converting intravenous doses to equivalent oral doses

		Recommending therapeutic substitutions from nonformulary orders to formulary medications

Over time, the job description of the clinical pharmacist was differentiated from that of the staff pharmacist, and as the relationship between pharmacist and physician developed, doctors began asking the pharmacist to go beyond offering advice and to initiate changes to the drug and dosage regimens in response to patients’ signs and symptoms. Although hospitals had the legal flexibility to allow such practices, initially a physician was generally required to countersign each order. In time, hospitals approved blanket protocols that gave pharmacists direct authority to make these changes without prior or subsequent approval by a doctor. In the retail or community setting, however, pharmacists were not allowed to modify any regimen without a prescription by a physician. Prior to making a change, pharmacists were required to call the physician to ask for a new prescription.

Today, as collaboration between pharmacists and physicians has grown and education and training of pharmacists has improved, a new model is being explored throughout the country: the community pharmacist as clinical provider of services in the community setting. This article explores the trends, nature of such services, and opportunities available as more states are enacting legislation to facilitate greater collaboration between pharmacists and physicians than took place in the past.

The education and experience a pharmacist gains during school and after graduation includes much more clinical training than it did 30 years ago. Accredited pharmacy schools in the United States have gradually phased out the bachelor of science (BS) degree in pharmacy and now only offer a doctor of pharmacy (PharmD) degree. Hospitals now also offer 1- and 2-year general and specialized post-PharmD residencies in various areas of hospital pharmacy clinical programs. In addition, some hospitals have started placing pharmacists in outpatient clinics where they can assist in managing a patient's chronic disease, such as asthma, hypertension, and hypercholesterolemia. These outpatient services are provided as either part of a multidisciplinary term including pharmacists, physicians, and nurses, or under the supervision of a physician.

In 1990, some states passed legislation under which pharmacists could provide clinical services in the hospital setting under specific protocols considered to be prescribing but that were “dependent” on a licensed prescriber. Washington, one of the first states to offer such a protocol, allowed pharmacists to provide defined services to patients of specified physicians. Although the protocols sound restrictive, specific services could be anything the physician wanted done by a qualified and trained pharmacist. Examples of more unusual services include monitoring and modifying parenteral nutrition, adjusting doses of lidocaine for patients receiving lidocaine infusions, and initiating and modifying opioid regimens for patients needing patient-controlled analgesia after surgery.

The newest term used when discussing collaboration between pharmacists and physicians in the retail arena is “community clinical pharmacist (CCP).” Not that the idea of a pharmacist working in collaboration with a physician for retail patients is new; pharmacists have often called physicians to discuss medications, doses, and regimens when they encountered potential drug interactions or intolerable adverse effects. What makes the role of CCPs different is their ability to directly adjust a patient’s medication regimen without contacting the physician. The CCP service allows pharmacists to assist in the care of patients/customers in the community and prescribe therapies for patients in acute situations to reduce urgent care and emergency department visits.

The rules regarding the role and responsibilities of a CCP differ in each state, depending on specific legislation. The list below was collected from the allowable protocols and regulations of numerous states.

 Depending on the state, a pharmacist can do some or all of the following: 

		Specify or limit the disease states the pharmacist can assist in managing

		Determine whether the pharmacist can administer medications

		Determine the setting in which the pharmacist may render clinical services (ie, community, hospital, or clinic)

		Specify additional training needed beyond the degree, both initially and as ongoing continuing education

		Require communication with a physician and documentation of their services

		Specify whether the physician needs to countersign after each medication adjustment.

		Determine whether a pharmacist may do each of the following: (a) initiate, (b) modify regimen, and/or (c) discontinue a medication

		Require ordering and interpreting tests to monitor drug safety and efficacy

		Indicate whether the protocol is limited to specifically identified patients of the physician

Most states require that a majority of services be rendered pursuant to a collaborative relationship with a physician who is also seeing that patient. Some states, such as California, make exceptions for services such as administration of vaccinations, prescription of nicotine replacements, and initiation or furnishing of hormonal replacements.

At least 36 states authorize pharmacist clinical services in any setting as set out in a written protocol. Some protocols are specific to certain disease states, while others limit services to vaccinations, smoking -cessation counseling, and provision of emergency contraception. Twenty-one states require pharmacists, and in some cases, physicians, to submit the written protocol to the Board of Pharmacy or Medicine.

As a result of the wide variance between states regarding the nature of services allowed, the nature of the protocol, and the disease states covered, each setting needs to refer to the specific rules in the state in which the pharmacist will practice to determine the extent to which clinical pharmacy services can be provided under protocol. For example, in North Carolina, clinical pharmacist practitioners (CPPs) provide drug therapy management (including controlled substances) under the direction or supervision of a licensed physician who has provided written instructions for a patient and disease-specific drug therapy. The instructions may include ordering, changing, or substituting therapies or ordering tests. Any changes to the protocol must be approved by the pharmacy and medical boards. The supervising physician must be readily available for consultation with the CPP and will review and countersign each order written by the CPP within 7 days. The written CPP agreement must be specific with regard to the physician, the pharmacist, the patient, and the disease; specify the predetermined drug therapy, which shall include the diagnosis and product selection by the patient's physician; and include any modifications which may be permitted and the dosage forms, dosage schedules, and tests which may be ordered.

In Washington state, pharmacists who wish to be certified as CPPs must complete 20 hours of self-paced training to learn how to identify illness, rule out complications, and prescribe therapies. CPP training also includes instruction on developing a business and marketing plan, documentation, ethical considerations, and practical points. Each of the 14 clinical modules provides a comprehensive review of a disease state, the differential diagnosis to rule out other causes of symptoms, and treatment recommendations. The certified CPP can complete one or all the clinical modules, or pick the ones that best fit their practice setting.

Studies have shown that drug therapy monitoring, counseling, and educational services provided by community pharmacists contribute to improved health outcomes for patients with chronic conditions such as hypertension, diabetes, and hyperlipidemia.

 One of the more famous studies showing the success of the clinical community pharmacist is The Asheville Project.

The Asheville Project was a North Carolina state-sponsored project that allowed pharmacists to provide education and counseling to state employees with specific chronic health problems After the patients were provided with intensive education through the Mission-St. Joseph’s Diabetes and Health Education Center, they were teamed with community pharmacists for a medication review and patient education. There were numerous positive outcomes from this project, including fewer sick days, improvement in diabetes monitoring parameters, and lower health care costs.

One of the more complex and difficult aspects of providing clinical services is whether the pharmacy can be paid for the services provided. There are many examples of pharmacists using physician codes to bill commercial insurance companies and having varying degrees of success at getting reimbursement. Some states have conducted major projects evaluating whether outcomes improve when pharmacists are reimbursed. Mississippi was the first state to seek and gain Medicaid approval to pay pharmacists for their clinical services in 1998. A similar waiver and plan was implemented in Ohio in 2012.

Payments for pharmacy services provided through state Medicaid programs vary from state to state. The most commonly reimbursed services in the 15 states that provide Medicaid compensation for direct patient care include smoking cessation, counseling, and other preventive services.

 Under Medicare Part B, pharmacists are not included in the statutory definition of “providers” and cannot directly bill for patient care services. In contrast, Medicare Part D does reimburse for MTM services when provided under contract with the sponsor of a prescription drug plan. Qualified MTM services include medication review but not chronic disease management. MTM does not require a formal collaborative agreement between a pharmacist and a physician, and it can be provided without dispensing a medication.

To make clinical services a success, community pharmacies need to ensure that pharmacists are afforded the time to provide patient care services, not just dispense products to patients. This may require additional staff and training of existing staff. Community pharmacists should move to a technician-driven, pharmacist-managed dispensing process. Investment in technology may also be required, such as purchasing a state-of-the-art pill counter or a robot for automated dispensing. Community pharmacies should also look into what is allowed under state board of pharmacy rules with regard to technicians being able to verify the work of another technician without requiring a pharmacist verification.

Community pharmacists also need to think about where they will counsel patients during the dispensing process and if that space provides some level of privacy. The space should allow for an intimate discussion where the patient does not feel rushed or concerned about privacy. The CCP will need to document any drug therapy problems found during the dispensing process when pharmacists are performing clinical services, along with an action taken. Electronic or hard-copy records or charts should be kept for all patients and should include all interventions and recommendations the pharmacists make.

The nature of pharmacy services has been steadily changing as new models of care and new reimbursement programs have emerged. New legislation in many states provides opportunities for personal and professional growth for individual pharmacists and pharmacies that want to offer more to their customers than simply filling prescriptions. Pharmacists need to grow their collaborative practice in the community setting by partnering with care groups and physicians, and savvy pharmacy owners will see this as an opportunity for both customer and revenue growth, while improving the quality of patient care.

Lorne Basskin, PharmD, is a consultant for hospitals and hospital systems in formulary development and management and drug use policy. He has written and presented numerous research projects on clinical pharmacy services, pharmacoeconomics, and outcomes research. After serving as director of clinical services and director of pharmacy services at a Florida hospital, he developed and served in a national pharmacist position and as director, clinical and information services, both at a large for-profit hospital corporation. He is an adjunct professor at Brown University School of Public Health in Providence, Rhode Island.

		 Centers for Disease Control and Prevention: Division for Heart Disease and Stroke Prevention. Select features of state pharmacist collaborative practice laws. cdc.gov/dhdsp/pubs/policy_resources.htm. Published December 2013. Updated July 16, 2015. Accessed October 6, 2015.

		Clinical community pharmacist. Washington State Pharmacist Association website. wsparx.org/?page=CCP. Accessed October 10, 2015.

		North Carolina Administrative Code. Board of Pharmacy—Pharmacy Rules. 21 NCAC 46. 3101. Section .3100&mdash;Clinical Pharmacist Practitioner. North Carolina Board of Pharmacy website. www.ncbop.org/LawsRules/Rules.pdf. Updated November 2015.

		McBane SE, Dopp AL, Abe A, et al; American College of Clinical Pharmacy. Collaborative drug therapy management and comprehensive medication management - 2015. 

. 2015;35(4):e39-e50. doi: 10.1002/phar.1563.

		The Asheville Project. theashevilleproject.net

		Isasi F, Krofah E. The expanding role of pharmacists in a transformed health care system. National Governor’s Association website. nga.org/files/live/sites/NGA/files/pdf/2015/1501TheExpandingRoleOfPharmacists.pdf. Published January 13, 2015. Accessed October 7, 2015.

		Pai AK. Integration of a clinical community pharmacist position: emphasis on workflow design. 

Articles

As the relationship between pharmacist and physician developed, doctors began asking the pharmacist to go beyond offering advice and to initiate changes to the drug and dosage regimens in response to patients' signs and symptoms.

Opportunities for Collaboration in Clinical Pharmacy Services

						Biologics are large, complex molecules manufactured from living organisms. Cell lines are programmed to produce a specific therapeutic substance (a “biosimilar”) with a goal of replicating the safety and efficacy of an existing “reference” biologic drug.

						Biosimilars require a 2-step approval process by the FDA: the first step is to show “similarity” in safety and efficacy, and the second step is to show “interchangeability” with the reference drug.

						Biosimilars are approved for specific treatment indications, but the FDA may allow extrapolation to other disease states, depending on clinical evidence submitted.

						State laws permitting interchange at the pharmacy will likely have strict rules about record keeping and communication between prescriber and patient. More than half of states have yet to pass rules regarding substitution.

						The primary safety concern of patient advocate groups is that antigenicity, or immune response, for any one patient, will differ unpredictably for different biosimilars for the same reference drug.

						Expected savings to health care systems will depend on how quickly prices change in response to competition, and how many biosimilars are produced for any one reference drug.

In 2015, we saw the first FDA approval of a biosimilar of a biologic drug that lost patent protection, but many questions remain. What exactly is a biosimilar, and what are the repercussions for patients, payers, and manufacturers, if multiple biosimilars are approved for the same drug? Are they as safe and effective as the original drug (also known as “reference drug”)? What is the pricing and competition experience of other countries that have had biosimilars for several years? To answer these questions, we must first understand what a biologic is.

Biological products are used to treat rheumatoid arthritis, anemia, low white blood cell counts, inflammatory bowel disease, psoriasis, and various forms of cancer.

Two equivalent biologics (biosimilars) made using different cell lines and different manufacturing processes will rarely, if ever, be exactly the same. Notwithstanding that, a biosimilar needs to work in the same way, and with the same mechanism of action, as its reference product.

Unlike a generic approval, in which the substitution just needs to show same active ingredient and bioavailability, approval for a biosimilar is a 2-step process. First, the drug needs to be shown as “highly similar” to an already-approved biological product. It must have the same safety and efficacy profile as the reference drug, but does not need to have an identical active ingredient to the reference drug.

Second, under the Patient Protection and Affordable Care Act (Affordable Care Act), the FDA requires additional evidence for the biosimilar to be “interchangeable” (ie, it may be dispensed to a patient when a reference product is prescribed). According to the FDA, there must be no risk in switching from reference to biosimilar, or between multiple biosimilars for the same reference product. Head-to-head clinical studies (without placebo arms) comparing the biosimilar with the reference drug are required. While approvals for interchange are for a specific combination of drug and indication (ie, disease state being treated), the FDA may allow “extrapolation” between some closely related diseases, though it has yet to give any examples and has not stated if it will require clinical studies for approval for each indication in which the innovator product is licensed. 

Why would a biosimilar not be interchangeable for all indications? It may be that the biosimilar manufacturer never bothered to submit data for all indications, or the reference drug gained a new FDA-approved indication after the biosimilar’s approval. Though drug companies will be prohibited from promoting a biosimilar for an indication “not interchangeable,” past experience has shown that labeling for specific indications never stopped physicians from prescribing agents missing the FDA-approved indication. The difference this time is that the pharmacist will probably be prohibited from switching to a less expensive biosimilar if it does not have the interchangeable feature for all indications. One can see a complicated substitution list in the works in which each prescription refill will need to be judged for interchangeability based on its indication.

It is unlikely that a manufacturer in the United States will produce a biosimilar that is not ruled interchangeable, since it could only be dispensed if the doctor wrote a new prescription for that specific biosimilar, and the patient could not then be switched to any other biosimilar for a refill.

While the FDA rules whether biosimilars are interchangeable, it is the states that determine whether a pharmacist can, must, or cannot legally substitute a biosimilar for the reference drug. Individual states either have enacted or are enacting their own regulations on biosimilars. Eight states have already enacted legislation, 10 states are deliberating legislation that has been introduced, and in 8 other states, steps are being taken to introduce legislation. The provisions of state legislation vary, but several features and requirements that have been supported by both advocate groups and biopharmaceutical groups are frequently included:

		Any biological product under consideration for substitution must first be approved as “interchangeable” by the FDA.

		The prescriber (eg, physician, oncologist, physician assistant, etc) would be able to prevent substitution by stating “dispense as written” or “brand medically necessary.”

		The prescriber must be notified of any allowable substitution made at a pharmacy.

		The individual patient must be notified that a substitute or switch has been made. In some cases, state law would require patient consent before any such switch is made.

		The pharmacist and the physician must retain records of substituted biologic medications.

Only one drug has been approved in the United States to date: Zarxio, a biosimilar of the white-cell growth factor filgrastim, a granulocyte-colony stimulating factor used to stimulate bone marrow to produce more white blood cells. Five other biosimilars are currently under review by the FDA: Apotex’s biosimilar versions of Amgen’s Neupogen (filgrastim) and Amgen’s Neulasta (pegfilgrastim); Celltrion’s infliximab biosimilar; Hospira’s epoetin alfa biosimilar; and Sandoz’s version of Amgen’s Embrel.

There are over 700 biosimilars in development worldwide for 44 reference product applications.

Table 1: Countries with Reference Product Applications

Number of Reference Products with Biosimilars

What exactly is a biosimilar, and what are the repercussions for patients, payers, and manufacturers, if multiple biosimilars are approved for the same drug?

Biosimilars-Substitution is Not Just for Your Grandfather's Generics Anymore

Health care data streams offer great promise, but pharmacy stakeholders must beware of improper data mining and false conclusions. 

“Big data” has been described by various futurists and health industry pundits as the source of the answers to all problems in health care. The promise of vast amounts of data about patients, doctors, and hospitals that can be sorted and summarized numerous ways has led some stakeholders to conclude that the reasons for any problem can be determined and the solution predicted with great certainty. The reality: although it is true that the data streams are now quite voluminous and opportunities abound, there are plenty of caveats to the notion of elegant and accurate data sorting and analyzing, as well as the prediction of outcomes.

Big data describes any large data set that has the potential to be mined for information. It is high volume, has a wide variety, can be quickly generated and aggregated, and is often (regrettably) incompatible with different databases. Big data allows for faster identification of high-risk patients, more effective interventions, and closer monitoring. It has earned the label of “big” because it comes from so many more sources than in the past. Everything everyone does can now be stored capable of being stored in, and potentially recalled, from a computer, cell phone, tablet, etc. Every clinical outcome or lab, charge, cost, and provider identity can be both stored and combined with another database for any patient, disease, or doctor. Detailed reports can be prepared for any category and drilled down to any population subset within seconds rather than days.

Big data is data now coming from many diverse corners of the health care system: research from drug manufacturers, digitized patient records, clinical trial information, and claims databases from public payers such as Medicare and Medicaid. In addition, an individual patient’s clinical data now come from a variety of sources, as well: payers, hospitals, outpatient clinics, doctors’ offices, and the patient themselves. Electronic medical records (EMRs) have become a major source of data thanks to federal incentives. With EMRs, every lab, drug, intervention, order sheet, physician order, progress note, and (potential) clinical outcome is available for aggregation by population and identification for future trends.

		Big data streams are high volume and quickly generated and aggregated.

		Big data allows for faster identification of high-risk and high-cost patients.

		Self-reported data by patients are particularly powerful in predictive terms and useful for patient satisfaction, quality of life, and correlation with clinical data; they come via cellphone, social media, or online surveys.

		Big data enables searching of data for relationships and trends between outcomes, costs, providers, hospitals, and certain disease states that be used to predict future behavior and performance or identify areas for improvement.

		Big data streams often reside in separate databases that may be incompatible with other databases.

		Missing, unverified, or incomplete data can limit usefulness. Different databases may lack standardization in definitions and of terminology.

		Big data doesn’t equal big evidence; well-designed research to build a case is still needed. Big data correlations do not necessarily establish cause and effect, and can result in ridiculous conclusions. Potential for serious sampling errors exist with retrospective analysis of big data as opposed to more rigorous (but also more expensive and time-consuming) clinical trials.

		Data mining (also known as data dredging) may enable business intelligence, but may be problematic when attempting to establish relationships between costs, outcomes, and providers. It is easy to get “accidental,” and incorrect conclusions, from this approach.

Examples of Contemporary Uses of Big Data 

		Prediction of patient behavior (adherence, emergency department utilization, and other outcomes and behaviors of interest).

		Establish cost-effectiveness and use patterns among competing hospitals, drugs, and providers by comparing costs and clinical outcomes with providers and facilities, but by grouping the patients by things they have in common (eg, location, disease state, age, gender, etc).

		Develop recommendations for clinical pathways, clinical guidelines, protocols, and formularies for better outcomes based on past experience.

		Enable the targeting of patient groups and focused interventions for patients who are the most expensive in a system (eg, high-cost patients, readmissions, patients whose condition worsens, adverse events, and patients with complicated, multiorgan diseases).

		Determine which drugs are associated with high rates of adverse events.

		Monitor patients and providers for compliance with treatment guidelines, and educate or penalize those who fail to comply (or reward the compliant ones).

		Pharmacoeconomic analysis to determine which drug, device, or service is the most cost-effective.

Big Data vs Big Evidence: What’s the Difference? 

Doing research is just like cooking food your guests will enjoy and want more of: you need the right ingredients (data) and a method of preparing and combining them (research and statistics) to create the end product (evidence). Until all the ingredients come together properly, you do not have something worthy of presentation. According to the International Society for Pharmacoeconomics and Outcomes Research Task Force Report on Real World Data, “Evidence is generated according to a research plan and interpreted accordingly, whereas data is but one component of the research plan. Evidence is shaped, while data simply are raw materials and alone are noninformative.”

Much has been written in recent years suggesting that medicine decision making should be evidence-based. Evidence-based medicine (EBM) has been defined as “the conscientious, explicit, and judicious use of current best evidence in making decisions about the care of individual patients.”

 Although EBM requires data, it also requires a rigor for both collection and analysis of the data so that the conclusions are not due to bias, statistical sampling error, or errors in data analysis. Simply copying data to a spreadsheet and looking at the average values may be quick, but it may also be inaccurate. No matter how much data exist, researchers still need to ask the right questions to create a hypothesis, design a test, and use the data to determine whether their hypothesis is true.

Big data includes data from patients or unverified sources: patient registries, social media, and government sites that allow users and providers to enter data directly. These data can be aggregated and sorted anonymously or, with patient permission, be tied directly to objective clinical data, charges, cost of care, their disease states, and the medications they are taking. They can be used to measure a patient’s quality of life; their experience with physicians, hospitals, or other providers; or even home monitoring. Using GPS-enabled devices and smartphone apps, it is possible to directly report heart rate, blood pressure, arrhythmias, medication use or refill information, and blood glucose levels.

A trend seen among less-experienced database users is the improper use of data mining. Individuals may search and re-sort the database until they find something that looks significant, even if it seems illogical. There are 3 problems with this approach:

		As you data mine, you tend to shrink the size of the sample because fewer and fewer people have all the characteristics you add. If you search long enough, you can find results that may not be statistically significant.

		It is easy to bias the data mining by first looking at which drugs had desirable outcomes and then choosing patients to match.

		It is possible to generate spurious correlations, or things that correlate with each other but in fact have no relationship with each other. There is an entire website (www.tylervigen.com) devoted to such correlations between unrelated findings, such as the number of films a particular actor appeared in compared with the number of drowning deaths during a given time period.

The biggest risk of error from these correlation conclusions is inferring cause and effect. When 2 things occur together (which is all that correlation confirms), the researcher has the chance to show bias by declaring which happened first, naming which is the cause and which is the effect.

Big data by itself has limited value. The usefulness lies in the ability of pharmacy stakeholders to determine the trends and relationships between data points for any single population member. There are always “hidden variables,” or confounders, that may not be seen in the data but could serve to be important predictors of the outcome. These confounders include other concurrent therapy, severity of an illness, standard of care, concurrent diseases, and a patient’s genetic makeup.

A field of science called “predictive analytics” is used to predict how a situation will play out in the future based on results from the past. A prediction can be used to treat an entire population similar to the one studied or can be used to tailor treatments for individual patients based on determining the provider, hospitals, or medications most likely to achieve a given outcome. In essence, big data serves to substitute the experience of thousands of similar patients who had a variety of outcomes for the clinical judgment of the treating physician.

Can Big Data Be Used to Tell Us Which Care Is Cost Effective?

To choose a cost-effective intervention (whether medications, clinical services, or devices), provider, or facility for a patient or group of patients, providers need to know the cost from the perspective of the user. Costs differ from the provider and payer perspectives, and a hospital's costs and its charges are not the same. We also need to know how effective an intervention is in achieving the primary clinical outcome, whatever that may be: a quicker cure, a longer life, a disability prevented, a successful surgery. Costs and efficacy may then be compared, and the following rules developed by the author of this article may be used to determine the most cost-effective treatment:

		If 2 drugs have the same cost, choose the more effective drug.

		If 2 drugs have equal efficacy, choose the less expensive drug.

		If 1 drug costs less and is more effective, choose it because it is dominant (a no-brainer).

		If 1 drug costs more and is more effective, the more expensive drug is considered cost effective if the extra benefits are worth the extra cost (ie, it has greater value).

Potential for errors in cost-effectiveness research include:

		Failure to capture costs outside the area served by the database, such as the cost of care of the home-care provider or a physician paid directly by the patient rather than the insurer.

		Insufficient clinical data or faulty assumptions for missing data.

		Cost figures that are an ill-defined mix of direct and indirect costs, and fixed and variable costs.

Big data seems to offer some real potential to improve the quality of care and related outcomes by trying to determine which procedures and providers offer cost-effective treatment. Database users need to know the information they will be accessing is complete and accurate. Big data needs to provide enough detail so that when users need to “drill down” to a specific treatment, patient category, or provider, the data can be accessed and summarized. Providers will, assuming that all the relevant databases can be tied together, have more information from multiples places where care has been provided: pharmacists, labs, physicians, hospitals, nursing homes, emergency departments, and outpatient surgery centers.

Because privacy and security will be concerns since significant database breaches are reported weekly among large companies, it will be important to ensure data do not fall into the wrong hands. Consumers will always be concerned that some of this data may fall into the hands of an employer, insurance company, or even an ex-spouse and be used in a prejudicial manner. How to collect and sort this data while keeping it away from the “wrong” people and getting to the “right” people will be a challenge for years to come.

The potential reward of big data is tremendous, but it coexists with the possibility of serious problems resulting from its misuse. Unverified data from patients; databases that cannot communicate and share information; the risk of missing, incomplete, or inaccurate information; and a lack of rigor in research, including false conclusions of cause and effect based on incorrect association or correlation, are all issues that must be addressed.

Lorne Basskin, PharmD, is a consultant on outcomes research, formulary decision making and pharmacoeconomics, and teaches in the School of Public Health at Brown University.

		Garrison LP Jr, Neumann PJ, Erickson P, Marshall D, Mullins CD. Using real-world data for coverage and payment decisions: the ISPOR Real-World Data Task Force report. 

		Sackett DL, Rosenberg WM, Gray JA, Haynes RB, Richardson WS. Evidence based medicine: what it is and what it isn’t. 

"Big data" has been described by various futurists and health industry pundits as the source of the answers to all problems in health care. 