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106 PharmacyTimes.org December 2015 dosing strategies of ataluren therapy: low dose versus high dose, 3 times daily. 54 The low-dose group was dosed 3 times daily at 4 mg/kg, 4 mg/kg, and 8 mg/ kg. The high-dose group was dosed 3 times daily at 10 mg/kg, 10 mg/kg, and 20 mg/kg. Data indicated that treatment with ataluren produced improvements in CFTR activity, but additional stud- ies are warranted to evaluate the effect of ataluren on pulmonary function and pulmonary exacerbation rate. Following the dose-finding study described above, a phase 3 trial was initiated to further evaluate the high- dose regimen with the aim of evaluating improvement in pulmonary function. 55 Patients 6 years or older with a docu- mented nonsense mutation were included. At week 48, researchers found no statisti- cally significant difference in the relative changes from baseline of predicted FEV 1 between ataluren and placebo. The sec- ondary end point evaluating rate of pul- monary exacerbation was not statistically significant between the treatment groups. Of note, post hoc analysis revealed that patients not on long-term inhaled tobra- mycin showed a 5.7% change in FEV 1 from baseline and had 40% fewer pulmo- nary exacerbations. Understanding the underlying cause of CFTR dysfunction has led to incred- ible changes in the management of CF. These current therapies show a promis- ing paradigm shift and a brighter future for CF therapeutics. Role of Specialty and Community Pharmacists With their accessibility in communi- ties and extensive medication expertise, pharmacists are uniquely positioned to ensure continuity of care by educating patients on the current and emerging therapeutics, the importance of genetic testing, and importance of treatment adherence. CF is no longer known as a homogenous disease that claims the life of teenagers; it is now recognized as a disease with multiple gene expres- sion and emerging treatment options that require knowledge of gene-specific ther- apeutics approaches, as well as intense management of multiorgan involvement and lifelong drug therapy. In the management of CF, both com- munity-based and specialty pharmacists have a central role in managing polyphar- macy safely and effectively, with the goal of promoting appropriate use of all treat- ment modalities available. Specifically, specialty pharmacists housed within a CF clinic–based setting or intimately involved in the coordination and distri- bution of CF therapies play a vital role in CF medication–related care. With the approval of new therapies, such as iva- caftor and lumacaftor/ivacaftor, specialty pharmacists are pivotal in ensuring these therapies are prescribed and monitored appropriately. Screening patients for these therapies includes an initial evaluation of the patient's age and genetic mutations, allowing for the appropriate identification of candidates for the agents. A specialty pharmacist working inti- mately with the providers allows for a collaborative review of baseline char- acteristics such as pulmonary function tests and other laboratory markers, such as liver function testing, to ensure proper prescribing of these therapies. Specialty pharmacists are able to review all con- comitant therapies, being cognizant of the potential for drug interactions that may warrant dose adjustments. In addition, pharmacists are integral in the appropriate monitoring of all CF medi- cations and in recommending a monitor- ing plan to assess safety and efficacy of these specialty medications. The patient-focused care provided by specialty pharmacists can help maxi- mize the use of effective therapeutics while minimizing the risk of treatment- related AEs. Pharmacists can address treatment expectations and identify barri- ers to adherence with treatments, includ- ing AEs, dose scheduling, and perceived benefits. Given the heavy time burden of respiratory treatments, nebulized medi- cations, and long-term therapy, this is extremely important for patients with CF. Because pharmacists have the unique opportunity to communicate with physi- cians and patients, they can often be key players in the total care of the patient. Many patients with CF have local health care professionals to help manage other non-CF conditions, as well as their care at a regional CF care center. It is extremely important that medications prescribed be carefully reviewed to pre- vent potential interactions and duplicate therapy. In addition, coordination and facilitation of drug products from multi- ple sources can lead to decreased adher- ence. Pharmacists caring for patients with CF can help prevent this problem. Given the growing complexity of CF care, the cost of outpatient medications can be as much as $100,000 a year without the addition of one of the new disease-modifying agents. With that addition, the cost can exceed $300,000 a year. In addition, patients may be admitted to the hospital for intravenous antibiotics and other intense therapies during an acute pulmonary exacerba- tion. Pharmacists can play an integral role in helping coordinate care when patients are discharged from the hospital, help to ensure patients are getting their medications with various regimens of antibiotics, and carefully watch patient refill history for chronic medications. It is clear that a patient with CF has a tremendous amount of treatments for airway clearance as well as medications, and requires intensive coordination of services. Therefore, an important role of pharmacists is to maintain good com- munication regarding which medications may no longer be needed with hospital- ization and how regimens may change. In providing these services, commu- nity and specialty pharmacists have had a positive clinical impact on patient health care and play a key role in dis- ease management models for conditions such as CF, including providing patient education, increasing patient awareness, and improving patient adherence. To effectively provide these services, phar- macists need to stay abreast of chang- es in health care, including updates to clinical practice guidelines, availability of emerging therapeutics and their role in the treatment paradigm, and regula- tory guidance affecting dispensing and administration of medication. CONTINUING EDUCATION ADDITIONAL RESOURCES Some excellent resources to keep abreast of changes in therapy for CF are: • The Cystic Fibrosis Foundation website (cff.org): it is updated weekly with chang- es about CF care, research opportunities, and various educational tools that are extremely helpful for patients, families, and health care professionals. • The Cystic Fibrosis Research Institute (cfri.org): this organization of parents and health care professionals publishes a newsletter, funds research on CF, and conducts national and local meetings for parents, patients, and health care profes- sionals who care for patients with CF.