Edmund J. Pezalla, MD, MPH

In late 2010, the Pharmacy & Therapeutics Society, with significant contribution from the National Hemophilia Foundation (NHF), convened a series of 6 educational webinars for payer executives. The primary objective of the webinars was to explore how health plans and hemophilia treatment centers (HTCs) can better work together to deliver high-quality care and manage costs associated with hemophilia. Each webinar’s faculty comprised an HTC hematologist, an HTC nurse, and a managed care executive. Each webinar was part of an accredited continuing education program. These webinars were made possible by an unrestricted educational grant from Novo Nordisk.

Most of the 66 webinar attendees represented the medical and pharmacy departments from commercial, Medicaid, and Medicare health plans located across the country. The health plans were diverse, ranging from national plans with multiple lines of business to small regional plans and state Medicaid agencies. A few attendees represented pharmacy benefit managers (PBMs) and specialty pharmacies. This article is based on the educational materials presented and the discussions that transpired during the webinars.

Hemophilia is a chronic, rare disease with significant individual patient cost. Over the last few decades, with the introduction of new treatment modalities, the management of pediatric and adult hemophilia patients has progressively improved outcomes and resulted in increased longevity and decreased morbidity of disease. However, the transmission of viral blood-borne infections via replacement products (largely prior to 1990), clotting factor deficiency—associated arthropathy, and spontaneous and traumatic bleeding episodes have affected a significant segment of the hemophilic population. Overall care for bleeding-disorder patients is complex and requires an expert, dedicated multidisciplinary team. As hemophilia is a rare disorder, health plans often do not invest as many resources to evaluate formulary and coverage issues as they might for higher prevalence diseases with greater associated patient numbers and total cost.

Many webinar attendees suggested that their health plans do not fully understand the comprehensive medical and support services that HTCs provide and the positive impact those services have on patient quality of life and cost of care. Also, many webinar attendees reported that they have not fully evaluated the complexities of hemophilia and their impact on medical and pharmacy benefit design, formulary development, therapy acquisition, and patient management. Many webinar attendees believed that payers and HTCs might not have fully explored appropriate avenues to productively work together.

As this article demonstrates, a closer working relationship and better mutual understanding between health plans and HTCs could improve outcomes for hemophilia patients while providing a more favorable cost-to-benefit ratio. Treatment of rare diseases like hemophilia is unlikely to result in cost offsets that are greater than or equal to the drug costs. Managed care’s goal should be to optimize care and outcomes in a way that minimizes the cost of treating the rare condition. Managed care also should keep in mind that the model of care for hemophilia, as described below, can be a model of care for many other congenital diseases with costly treatments, such as those treated with enzyme-replacement therapies.

Hemophilia is an X-linked recessive bleeding disorder that affects approximately 1 of 5000 live male births.

 Currently in the United States, approximately 400 to 500 babies are born with hemophilia each year, and there are a total of approximately 30,000 affected individuals. Males are almost exclusively affected, yet females who carry the genetic mutation may be symptomatic, depending on the clotting factor level.

Hemophilia A, characterized by decreased activity and/or levels of Factor VIII, affects 80% to 85% of hemophilia patients. Hemophilia B, characterized by decreased activity or levels of Factor IX, affects 15% to 20% of hemophilia patients.

, most hemophilia patients are classified as having severe disease and have levels of Factor VIII or IX of less than 1%. They experience approximately 2 to 4 bleeding episodes per month, many of which are spontaneous (ie, there is no apparent inciting cause). The remaining affected patients have a less severe form of the disease, with Factor VIII or IX activity levels between 1% and 40%.

Hemarthrosis: A Common Complication of Hemophilia

Patients with severe hemophilia frequently experience spontaneous intra-articular hemorrhages, mainly in the ankles, knees, and elbows. Over the long term, repeated episodes of hemarthrosis may cause irreversible damage to the joint, leading to hemophilic arthropathy, which is characterized by joint stiffness, chronic pain, and a severely limited range of motion.

In a longitudinal 6-year study performed during the 1980s and 1990s, 90% of people with severe hemophilia experienced chronic degenerative changes in 1 to 6 joints by the age of 25 years.

 In another study, 42% of patients with severe hemophilia were restricted in their physical activities because of arthropathy.

A critical factor for avoiding the occurrence of hemophilic arthropathy is the prevention of articular hemorrhages.

 However, despite regular infusions of antihemophilic concentrate at an early age, recurrent joint bleeding and the possibility of hemophilic arthropathy still persist in some patients.

 Innovative strategies for the preventionand treatment of this common and serious complication are therefore required.

Several treatment regimens are utilized for the care of patients with hemophilia. One regimen, on-demand therapy, is defined as the administration of clotting factor concentrates at the time of an acute bleed. Another regimen, prophylaxis, consists of the administration of clotting factor concentrate in advance of active bleeding with the intention of preventing bleeding events and associated subsequent damage to the affected area. Prophylaxis can be primary (ie, prevention of bleeding events starting early in childhood before the development of a hemarthrosis) or secondary (ie, prevention of bleeding events in those patients with a history of previous bleeding events). Prophylaxis has been documented to improve the joint health and quality of life for many patients with severe hemophilia. Pain also may be averted or mitigated by prophylaxis strategies.

Available data demonstrate that prevention of bleeding episodes from an early age enables hemophilia patients to avoid or reduce the clinical impact of joint disease and improve psychosocial development and quality of life.

 For example, young patients on prophylaxis are better able to participate in physical activities, regularly attend school, and achieve their academic and personal goals.

One of the most serious and costly complications of hemophilia is inhibitor formation.

 As many as 20% to 30% of severe hemophilia A patients

 and approximately 1.5% to 3% of those with severe hemophilia B

 develop a neutralizing antibody—called an alloantibody inhibitor&mdash;directed against the exogenously administered factor replacement therapy required to treat or prevent bleeding episodes. Most inhibitors develop early in life at a median age of 1.7 to 3.3 years and after a median of ~9 exposure days (ie, the number of days the patient was exposed to any clotting factor replacement therapy).

When inhibitors develop, the administration of normal replacement therapy to achieve hemostasis may fail, requiring the use of “bypassing therapies.” For patients with high titer inhibitors, examples of bypassing therapies include rFVIIa and an activated prothrombin complex concentrate. When inhibitors develop, there is a decreased ability to perform needed or elective surgery and an associated increase in morbidity and mortality.

While hemophilia patients with inhibitors comprise a minority of an HTC’s patient population, they often require many times the hours of care and volume of services utilized by their counterparts without inhibitors. Patients with inhibitors require intensive treatment interventions with frequent telephone interaction, education, emotional support, and appropriate plans for pain management.

The total cost of care for patients with alloantibody inhibitors is much greater than that for patients without inhibitors. In addition to requiring more costly therapies, hemophilia patients with inhibitors are often hospitalized at a higher rate. Also, hospitalizations for inhibitor patients may be more prolonged or complicated.

Regardless of the severity of disease and presence of inhibitors, the treatment of hemophilia is complex and requires expert evaluation and knowledge of the disease and individual patient. Therapies are costly and dosing varies from patient to patient. An experienced physician’s medical direction is required for appropriate care.

Hemophilia treatment centers have demonstrated their ability to deliver high-quality, cost-effective care for patients with hemophilia. Patients who received care directly from, or coordinated through, an HTC experienced a 40% reduction in mortality.

 Patients treated as part of an HTC program experienced better outcomes than patients cared for outside of the HTC network, even though the population treated within an HTC is composed of a higher percentage of patients with severe disease and those with blood-borne viral infections and/or inhibitors. Also, patients treated within the HTC network experienced a significant reduction in bleeding-related hospitalizations, especially those on home therapy, compared with patients without an HTC relationship.

As of January 2011, there were 141 federally funded HTCs across the country.

 (The HTCs are partially funded by federal support from the Health Resources and Services Administration [HRSA] and the Centers for Disease Control and Prevention. As of 2009, government funding for HTCs was approximately $11 million.) Hemophilia treatment centers are a model of comprehensive care for a chronic, rare disease. As such, HTCs provide multidisciplinary programs including direct medical care, patient support, patient education, genetic and mental health counseling, and physical therapy. In addition, HTCs may offer an integrated pharmacy program. Through this coordinated approach to care, HTCs are able to cost-effectively accomplish their primary goal of achieving optimal patient outcomes.

Each HTC has a core team of providers that includes hematologists, nurses, social workers, and physical therapists. Patients and their families are integrated into and considered key members of the care team. The HTC coordinates care of its patients across a variety of medical disciplines and specialties, including but not limited to primary care, orthopedics, genetics, pharmacy, dentistry, and educational/vocational counseling.

Providers at HTCs evaluate most individuals with severe hemophilia at least annually. The frequency of visits may depend on age, therapeutic regimen of care, and associated morbidities or conditions. The number of visits also depends on the patient’s geographic access to the HTC or an HTC-based outreach program.

For each patient, the HTC team determines the optimum care regimen, which may range from primary prophylaxis to secondary prophylaxis to on-demand therapy. The regimen is based on each patient’s diagnosis and associated clinical course. In addition, HTC team members are experts in the management of bleeding events and are critical for the planning of elective diagnostic or therapeutic invasive procedures.

Hemophilia treatment centers provide many important services in addition to direct medical care. These include, but are not limited to, patient/family education, research, outreach, emotional support, and oversight of home care services. HTC team members are available by phone 24 hours per day, 7 days per week, for intervention and consultation. HTCs provide these services to enhance the quality of their patients’ lives, improve outcomes, and decrease disease-associated morbidity and mortality.

The HTC staff also develops annual care plans for those patients who are located miles away from the HTC and who may receive their daily and routine care from their local hematologists or other medical care providers. The medical community frequently considers the hematologists within the HTC to be the local and regional experts in bleeding and clotting disorders. The HTC hematologists also provide the specialized laboratory expertise necessary to diagnose these rare bleeding and clotting disorders in a timely and accurate manner.

Physicians and nurses at HTCs teach patients and their families how to quickly and effectively manage bleeding episodes in the home and when to contact the HTC for advice and intervention. Also, HTC staff may educate primary care and hospital providers, dentists and other specialists, schools, and employers about hemophilia. Many of the people and organizations involved in a hemophilia patient’s life do not completely understand the disease, or have misconceptions. The HTC staff ensures that these groups understand the patient’s disorder, the need for safety measures and emergency protocols, and the treatment regimen and activity restrictions for individual patients.

Many HTCs have an integrated pharmacy program, which offers access to therapy and allows close medical involvement for bleeding events. More than half of the HTCs are qualified entities under HRSA’s 340B Drug Pricing Program. Section 340B limits the cost of covered outpatient drugs to certain federal grantees (including HTCs), federally qualified health center look-alikes, and qualified disproportionate-share hospitals.

 The main purpose of the 340B program is to stretch federal resources provided for hemophilia care as far as possible, thereby reaching more eligible patients.

 As 340B entities, HTCs realize significant savings on the cost of factor products.

Hemophilia is a rare disease with a well-established network of comprehensive centers of excellence and expertise. Translation of advances in hemophilia care is carried out in these centers of excellence in a cost-effective fashion, and these efforts have led to improved survival and quality of life. The ability to maintain, advance, and promote excellent care for individuals with hemophilia and other rare bleeding disorders depends on the preservation of the HTC network despite the present environment of reduced federal funding support and truncated reimbursement for time-intensive comprehensive care. The federal government has enabled HTCs to survive through purchasing and selling replacement therapy products through the 340B mechanism while providing the important benefit of passing along a reduced cost of replacement therapies to participating patients.

In summary, through state-of-the-art medical care and comprehensive support in addition to integrated pharmacy programs, the HTC team strives to decrease morbidity and mortality and decrease costs, such as those associated with unnecessary emergency department visits or inappropriate use of expensive replacement therapy.

Hemophilia is a chronic condition that affects a limited population. Therefore, health plans may not devote as much attention to the management of hemophilia as they do to more prevalent diseases with a greater budgetary impact.

Current medical and pharmacy policies related to hemophilia often are limited to prior authorization or precertification for clotting= factor replacement therapy. The primary goal is to ensure that products and technologies are used within labeling and according to best clinical practices. A secondary objective can be to identify hemophilia patients who experience a significant bleeding event requiring hospitalization and costly therapy management. For these patients, health plans may make case management services available to minimize barriers to care and improve its quality.

Given the complexity and cost of care for individual hemophilia patients, there may be opportunities for health plans to play a more active and effective role in managing these disorders.

To successfully improve a health plan’s approach to hemophilia, pharmacy & therapeutics (P&T) committees might seek to address the issues described below. Once these issues have been considered for hemophilia, there may be an opportunity for health plans to apply their experience and insights to other chronic, rare diseases that have a significant per-patient cost.

As reflected by webinar participants, many pharmacy and medical directors and case managers are not familiar with the complexities of hemophilia care and the HTCs’ approach to improving the quality of care while managing costs. Many of these health plan executives said they would benefit from education addressing the following topics: (1) a better understanding of hemophilia treatment and potential complications associated with hemophilia, (2) approaches for improving outcomes, (3) the nature and benefit of prophylaxis, (4) the nature of inhibitors and how they are managed, (5) genetic counseling, (6) management of pregnancies and deliveries of hemophilia carriers, and (7) new hemophilia therapies in the pipeline.

Education on these topics would help payers implement policies that foster improved quality of care. For example, education about prophylaxis and its benefits could help payers better understand the value of a significant near-term investment that could yield long-term benefits. A better understanding of inhibitors and the associated cost of care might encourage health plans to more closely monitor these hemophilia patients and encourage them to seek care from HTCs.

Partnering Between Health Plans and HTCs: A Key Activity

In 1954, NHF formed a Medical and Scientific Advisory Council (MASAC) to advance clinical care and promote hemophilia research. This council establishes quality-of-care guidelines for the treatment of hemophilia and other bleeding disorders. Issued in the form of recommendations, MASAC guidelines set the standard of care for HTCs and other providers around the world. The guidelines are referred to by an international array of physicians, medical schools, pharmacists, emergency department personnel, insurance companies, and others.

 The scientists, physicians, and other treatment professionals who comprise MASAC are internationally regarded as experts in the broad field of bleeding disorders research and care, AIDS, hepatitis, other infectious diseases, and blood safety.

All MASAC guidelines are published on the NHF Web site.

 MASAC has published 77 guidelines covering such topics as standards of care (MASAC #132), prophylaxis (MASAC #179), and preferred drug lists (MASAC #166). HTCs and specialty pharmacies that provide care for hemophilia patients are encouraged to comply with these guidelines.

Given the quality of care HTCs provide based on MASAC guidelines, there are opportunities for health plans and HTCs to partner more effectively. For example, HTCbased providers offer expertise that could assist health plans with the development of evidence-based policies and guidelines for hemophilia management.

Health plans’ case managers coordinate care and provide support for patients with a range of morbidities. As previously described, HTCs also coordinate care and provide support for hemophilia patients. Therefore, a closer working relationship between the health plans and HTCs could enhance care and limit duplication of effort. One option is for the HTC to act as the lead medical manager for a health plan’s hemophilia patients. Health plans could directly or through other available resources provide case management services for members without access to HTCs while encouraging these patients to connect to the HTC network.

Webinar participants were generally unaware of HTC efforts to manage the cost of care. For example, HTCs typically do not authorize automatic medication refills or autoshipment of expensive pharmaceuticals. HTCs work with patients to verify the number of bleeding episodes and the quantity of medications administered since the previous refill. HTCs closely monitor for appropriate dosing. HTCs are cognizant of cost issues and proactively manage them. Working more closely together could enable payers and HTCs to identify new avenues to better manage costs while enhancing the quality of care.

Articles

Exploring how health plans and hemophilia treatment centers can better work together to deliver high-quality care and manage costs associated with hemophilia.

Hemophilia and Managed Care: Partnering to Achieve Cost-Effective Care

There is little precedence for the changes we have seen in the healthcare marketplace in the past 36 months. From the passage of a historic healthcare reform bill to an economic slowdown that saw the first-ever decline in the number of prescriptions filled in a year, we have been presented with a number of challenges that will have long-lasting impacts on our industry. It is imperative that we continue to seek a clear method for defining value in healthcare delivery.

New developments in genetic testing and comparative effectiveness research make it possible to tailor therapies to specific situations and patients. This tailoring will make it possible to continue to promote the use of generics for many common conditions and, in turn, make it possible to pay for branded products and biopharmaceuticals when they are the best treatment choices.

The pharmaceutical marketplace continues to evolve, with an expanding array of generic medications for common serious diseases such as hypertension and diabetes. There also are increasing numbers of more expensive, specialized medications in both oral and injectable forms for a growing list of serious illnesses. Our challenge will be to improve the adherence to generics and to manage the more expensive, specialized products to be certain that they are both available for and received by those in need of treatment.

This dichotomy will lead to approaches that manage large populations via new technology and social media while providing personalized services to those with more difficult-to-manage diseases. Networking via social media with an appropriate moderator may allow patients with specific disorders to share concerns, ask and answer questions in their own way, and encourage each other to take a greater role in their own care. Such roles could include lifestyle changes, improved medication adherence, and advocacy for better treatments. What will come next? Will formularies tailor themselves to the genetic profiles of patients? Will consumer-directed plans expand to increase the incentives to patients for adherence and for using higher value drugs? Time will tell. What we do know is that the readers of this journal and members of the Pharmacy and Therapeutics Society will be at the forefront of these innovations. Keep it up.

As the number of both generic and specialized products continues to increase, we must seek a clear method for defining value in healthcare delivery.

The Evolving Pharmaceutical Marketplace: What Will Come Next?

The Pharmacy & Therapeutics Society (PTS) is pleased to announce that we have selected The American Journal of Pharmacy Benefi ts to serve as our offi cial journal. We believe that the Journal will quickly become a leader in the dissemination of information on the impact of formulary management strategies on utilization, cost, and quality of pharmacy services. Its mission is fully consistent with that of the PTS: “to foster interdisciplinary collaboration and information exchange and the P&T process; to support evidence-based formulary development and implementation across all practice settings as key elements in value-based benefit design; to evaluate best practices in P&T organization, process, and outcomes; and to share what we learn with all interested parties.”

The PTS leadership has been working to build an alliance of pharmacy and therapeutics (P&T) experts, corporate and healthcare organization medical directors, employers, and others to represent the offi cial “voice” of the P&T community. We want to develop new educational opportunities and policies that refl ect industry best practices and believe that the Journal is the best possible partner to help us achieve this goal.

The US healthcare system is confronting major change. Healthcare institutions, organizations, and managers of pharmacy benefits must integrate new approaches to evidence with efforts to maintain high-quality services and manage cost effectively. Healthcare providers and patients have an array of choices with respect to coverage and cost. It is important to ensure that patients and their families make the best possible decisions.

Generic products can be expected to change the formulary landscape in some major drug categories because some familiar branded products will soon be losing patent protection. New approaches to pharmaceutical care, biologics and “biosimilars,” vaccines, healthcare technology, specialty pharmacy, and healthcare procedures are on the doorstep. Comparative effectiveness research and the impact of biomarkers on the personalization of healthcare also are ahead. With all these changes come increasing demands from employers and government for evidence of quality healthcare and improved health outcomes commensurate with continued growth in healthcare spending.

The PTS will play a major role in this challenging environment. We will aid in the improvement of evidence-based benefi t design, drug formulary development, and implementation across all healthcare practice settings. The Journal will be our full partner in these efforts.

In addition to our work with the Journal, I would like to inform you of 2 other PTS developments. The first relates to a new feature of the PTS Web site. The second relates to an extremely important conference taking place in May 2009.

) now offers free webinars of selected sessions from our 2008 Annual Conference. You’ll be able to listen to the opening general session, “Clinical Value and Comparative Effectiveness: Generating Evidence,” with faculty Sean R. Tunis, MD, MSc, founder and director of the Center for Medical Technology Policy, and Steven D. Pearson, MD, MSc, FRCP, president of the Institute for Clinical and Economic Review, Harvard Medical School. You then will be able to hear an interactive panel discussion, “Healthcare Delivery Systems: Applying Evidence in Clinical Practice,” moderated by and featuring board of trustees members from 5 different healthcare practice settings. Finally, we will be presenting a workshop on “New Challenges in Vaccine Therapy Management.”

This is the first time we have used webinars to disseminate information from our annual conference. We hope that they help you in your own policy development activities and encourage you to attend our annual conference in Washington, DC, November 5-6, 2009, at the Gaylord National Hotel. We are developing the annual conference program now and welcome suggestions for topics to be covered, speakers to be invited, and potential corporate and organizational sponsors. Please feel free to contact our executive director, Joe Eichenholz, at 

The conference that will take place on May 18-19, 2009, at the Embassy Suites Airport Hotel in Philadelphia, Pennsylvania, is the first annual forum of P&T committees conducted by the Center for Business Intelligence in association with the PTS. This conference will help you understand the latest market dynamics impacting formulary development, management, evaluation, and the P&T process. The drug pipeline spotlight will review some of the most promising and challenging treatments for formulary consideration in the pharma and biotech areas. You will hear the latest P&T strategies from leading healthcare organizations and institutions. I am pleased to be chairing this conference on behalf of the PTS and know that you’ll find it a unique experience. Information on the conference is available on our Web site (

The year 2009 is one of unprecedented challenge to all Americans. The healthcare system must adapt to change and accommodate the needs of millions of Americans suffering from economic hardship. But we also have a tremendous opportunity to share knowledge, insights, and best practices to achieve the maximum value from our nation’s investment in healthcare services and products. 

The American Journal of Pharmacy Benefits 

and the Pharmacy & Therapeutics Society can responsibly address the healthcare needs of our country today and in the future. We welcome the 

 as our partner and as a positive force in the dissemination of new ideas in the healthcare arena.

The President of the Pharmacy & Therapeutics Society reviews the vision and goals for 2009 and announces its selection of The American Journal of Pharmacy Benefits as the Society's new official publication.

A New Alliance: Message From the PTS President

“The reform of healthcare delivery is but one challenge facing members of The Pharmacy & Therapeutics Society,” stated Ed Pezalla, MD, president of the organization. He explained that most of the debate on healthcare reform has centered on cost of insurance and payment reform, and the potential for a public insurance coverage option to compete with private insurers. “Eventually, something will come out of Congress, and the hard work of managing the cost and quality of care will get under way under this new framework,” said Dr. Pezalla.

Virtually any healthcare reform bill signed by President Obama will increase the number of insured Americans. As we cover more people, Dr. Pezalla believes generics will play “an increasingly important role. A lot of pent-up demand for access to medications will have to be addressed with generic medications.” Generics are available in most major therapeutic categories, and upcoming patent expirations will spur the introduction of more nonproprietary agents. “A larger number of people will be taking generics for a long time, because these medications are very effective,” he said, noting that generic dispensing rates, now in the 60% range, will rise over the next several years to 80%.

Generics alone, however, will not manage the coming wave of newly insured. “Increasing numbers of people will need very expensive [specialty] medications,” stated Dr. Pezalla. He is worried that providing affordable access to specialty medications for the population of presently uninsured who may benefit most from their use may be a costly endeavor.

He observed that better utilization of technology and data is necessary to manage the disorders of newly covered individuals. In terms of benefit design, he envisioned case management and intensive disease management as the best way to manage the newly covered Americans who can benefit from specialty medications. “Pharmaceuticals are a component of cost in the healthcare system,” he commented, “but they are the means to an end—to achieve good outcomes in the treatment of disease.”

A public option likely will place more decision making in the hands of the consumers. However, neither they nor providers are well prepared to enter this next stage in America’s healthcare evolution. Overall, Dr. Pezalla believes we need a better and faster national mechanism to bring evidence gained in clinical trials into practice. He pointed to medical and surgical procedures as a prime example: “We lack a great deal of evidence in terms of procedures. They are not regulated by the Food and Drug Administration or other bodies. Often, they are not subject to controlled trials.” Dr. Pezalla stated that comparative effectiveness research must play a prominent role, with evidence that can be translated into practice.

If private plans are to compete with a public option, they will need to aggressively manage cost through the use of narrower formularies and probably fewer 3-tier copayment plans. They will need to evaluate total care protocols and all aspects of medication therapy (eg, as in oncology care) rather than just 1 pharmaceutical. “Health plans will need more partnerships with the pharmaceutical companies to devise innovative contracting around guaranteed outcomes, appropriate use, and patient adherence,” he said.

Medications that have required Risk Evaluation and Mitigation Strategies (REMS) represent another challenge for P&T decision makers. Agents with associated REMS programs may be made preferred products because their increased level of scrutiny implies greater safety, Dr. Pezalla commented. “Or they may be avoided as having complex REMS requirements, especially if other medications are available.” 

Because not all comparative effectiveness research will be based on randomized controlled trials, P&T committee members will be required to understand complex statistical analysis, according to Dr. Pezalla, and “this skill is not currently in every P&T committee’s toolbox.”

“The pharmaceutical industry and P&T committees face an increasingly complex world,” concluded Dr. Pezalla. “One of our greatest challenges is to disseminate new tools for decision makers on the P&T committees. The Pharmacy & Therapeutics Society is a neutral conduit in providing these sorts of skills to P&T committee members.”

In the PresidentÃ¢ï¿½ï¿½s Address, Dr Pezalla observed that any healthcare reform will require the hard work of managing both the cost and quality of care.

Report on the 9th Annual Conference of The Pharmacy & Therapeutics Society

First and foremost, I want to commend and congratulate the editors and staff of 

for their outstanding efforts to launch this new journal. The concept is strong and the content thus far has been timely and actionable. We at The Pharmacy & Therapeutics Society are pleased to call the 

As we have begun to explore the ways in which the 

and the Society can collaborate to fulfill our respective missions, we have discovered a great deal of consistency in what we would like to accomplish, as well as efficient and cost-effective ways of reaching our audience. We invite all readers to raise issues that need to be addressed to improve best practices in these important areas: formulary structure, operation and review of emerging research, and new developments in patient care.

One opportunity to address P&T best practices and share emerging trends is participation in the Society’s 9th Annual Conference, 

, held November 5-6, 2009, at the Gaylord National Hotel in National Harbor, Maryland—on the Potomac River near Washington, DC. The opening general session will focus on the latest developments in comparative effectiveness research, both technically and from a policy perspective. That will be followed by a panel discussion on trends in healthcare information technology and its role in enhancing medical decisions. This topic is critical considering the challenges we face in identifying and measuring the metrics associated with evaluating the effectiveness of new and existing healthcare interventions. 

Our panel on contemporary issues in the P&T process, traditionally one of the most timely and highly regarded sessions, will focus on topics such as risk evaluation mitigation strategy initiatives to evaluate safety and barriers, and solutions to P&T committee collaboration within and across healthcare systems. I cannot stress strongly enough the importance of all these sessions to help guide us in developing our own business and formulary review processes for the future.

The annual conference also will include workshops to review the pharmaceutical pipeline and therapeutic areas where new product development can be expected to challenge the conventional P&T committee review process. 

These workshops include avoiding and managing medication errors, with a focus on trends in e-prescribing and incorporating metrics into new healthcare information technology systems; and trends in the use of evidence and guidelines in oncology, with case studies on the evolution of the formulary review process as we examine increasingly complex therapies for smaller populations and “orphan” conditions.

Our final general session will focus on the policy implications of the healthcare reform discussions that have been taking place during the course of this year. We will focus on what has been accomplished, what remains to be done to fulfill the stated objectives of the major participants in the policy development process, and the likely implications for all of us in 2010 and beyond. Additional information about the annual conference is available on our Web site at 

The Pharmacy & Therapeutics Society also has made a commitment to provide information, training, perspective, and education to physicians and others who serve on formulary committees across all practice settings. In this way, the Society can help these individuals work more effectively in an increasingly complex P&T committee environment. To this end, we will need volunteers to serve on task forces. These task forces will provide education on best practices regarding committee organization and structure, and the review process. In addition, they will address challenges faced by generalists, who may not be as familiar with the clinical trials and research subtleties of particular areas; and subspecialists, who may be too close to those trials and research to effectively advocate for them and would benefit from a broadened perspective.

We also seek support from companies who want to partner with the Society as we create a new environment of data-informed P&T best practices. For further information, please contact the Society directly at 

The Society seeks input from both individuals and organizations as it creates a new environment of data-driven P&T best practices.

A Look Ahead: Future Direction and Collaboration

The past 3 decades have seen the development and application of increasingly powerful decision-making tools to formulary and utilization management programs. Beginning with the evaluation of the quality of medical evidence by Cochrane through the new emphasis on comparative clinical effectiveness, hospitals, group practices, and managed care companies have brought these sophisticated tools to bear on a variety of serious clinical and economic issues. But knowing what to do and getting it done are 2 different things.  This is where the science of technology transfer and translational research can help.

Ultimately, it will be up to physicians and other healthcare professionals to assimilate new findings into medical practice and translate research into real outcomes. Pharmacy and  therapeutics (P&T) committees can play a major role in achieving this goal. 

Physicians and patients often see pharmacy benefit structures and the accompanying utilization management programs as barriers. However, they can be seen not as barriers but as beacons providing guidance in an increasingly complicated world. Although providing this guidance via the formulary development process is an important function of P&T committees, committee members also should take an active role in promoting adherence to practice guidelines, development of practice quality measures, and increasing connectivity of physicians with benefits and claims data. By promoting attention and adherence to the medical evidence, P&T committee members will contribute significantly to improvements in medical outcomes.

I encourage physicians, pharmacists, and others involved in the P&T process to be champions within their organizations. In this role, we can stimulate and promote the discussion of medical evidence through conferences, seminars, and reading groups. We can promote the use of electronic medical records, especially electronic prescribing, as a means of reducing errors and becoming more connected to the pharmacy benefit. And we can discuss the value of working with—not against&mdash;the programs created to manage the cost and quality of care. 

Outside the P&T committee, we can work together by publishing articles on innovations in practice, communications, and clinical programs. We can encourage our colleagues and other medical practitioners to participate in the activities of professional societies and to remain engaged in lifelong learning.

A recent study by Cavazos et al found that 84% of physicians interviewed did not prescribe nonsteroidal anti-inflammatory drugs according to guidelines because they were unfamiliar with the guidelines.

 Other problems with use of guidelines included perceived limited clinical value, inertia, and medical heuristics. 

Many pharmacy benefit plans help to remind physicians of the use of guidelines through prior authorization, step therapy, and other programs. The members of P&T committees can help healthcare professionals to assimilate these and other guidelines by emphasizing the clinical basis for these utilization management tools. Other areas of change include earlier use of immune modulators for multiple sclerosis and early initiation of insulin for diabetes. These treatments have been shown to have benefits when instituted earlier in the disease process.

Members of P&T committees are a valuable source of clinical information and can be a resource for their colleagues in healthcare.

This commentary encourages P&T committee members to be champions for assimilating new findings into medical practice and translating research into real outcomes.