Pharmacy Regulatory Updates and an Introduction to Specialty Pharmacy Mark Atalla, PharmD

Disclosure I have no relevant financial relationships with commercial interests pertaining to the content presented in this program

Objectives 3 Key Things to Walk Away With: Understand what Specialty Pharmacy means Understand Regulatory Landscape for Sterile Compounding Discuss Pedigree and FDA Track and Trace Requirements

Renal Specialty Pharmacy Regulatory Tie-in Renal Specialty Pharmacy Renal Therapy Management Integrating care among clinics and patients to deliver health outcomes Focus on value added pharmacy services Right dose, right time Counseling Adherence Adverse events Breakdown of the medicine value chain by stakeholder – IMS illustrative

The Importance of Phosphate Management Specialty Regulatory Tie-in The Importance of Phosphate Management *Urea Reduction Ratio Block GA et al. J Am Soc Nephrol 15: 2208-2218, 2004

Renal Specialty Pharmacy Regulatory Tie-in Renal Specialty Pharmacy Specific Goals of a Renal Specialty Pharmacy: Create alignment with prescribers to improve medication compliance Offer care and service to help our patients by providing convenient delivery of medications in the most cost effective way Be a valued resource to our staff to help improve patient outcomes Breakdown of the medicine value chain by stakeholder – IMS illustrative

Renal Specialty Pharmacy Regulatory Tie-in Renal Specialty Pharmacy How to get there: Clinical pharmacists that review lab values, compliance, and engage patients Pharmacists with certification in ESRD and co-morbidities Focus on execution, collaboration, and experience Breakdown of the medicine value chain by stakeholder – IMS illustrative

Specialty Pharmacy Overview Regulatory Tie-in Specialty Pharmacy Overview Specialty Medications are: CMS: medications that cost ≥$600 for a 30 day supply Represent a limited number of drugs that are used by a small proportion of beneficiaries Have specific processing, handling, and administration requirements May be a limited distribution network Potential narrow, and narrowing, commercial networks Alexion – Ultra-rare diseases

Renal Specialty Medications Regulatory Tie-in Renal Specialty Medications Indication Medication Comments Hyperphosphatemia Lanthanum carbonate (Fosrenol®) Chewable or Powder Sevelamer hydrochloride (Renagel®) - Sucroferric oxyhydroxide (Velphoro®) Chewable Ferric citrate (Auryxia®) Potential increase in iron (ferritin) Sevelamer carbonate (Renvela®) Available in powder or tablet

Renal Specialty Medications Regulatory Tie-in Renal Specialty Medications Indication Medication Comments Anemia Darbepoetin alfa (Aranesp®) Available in pre-filled syringes Epoetin alfa (Epogen®) Single and Multidose vials Secondary hyperparathyroidism Cinacalcet (Sensipar®) Daily dosing – compliance is important

Case Study Specialty Regulatory Tie-in ESRD patient with HIV Common therapy for HIV is tenofovir (Viread) Patient was taking Viread daily Data and recommendations from manufacturer: Intermittent hemodialysis Tenofovir 300 mg PO every 7 days following routine dialysis; administer following completion of dialysis. Adverse reactions: hypertriglyceridemia, increased cholesterol, decreased bone mineral density, GI side effects Key Take Away: Specialty pharmacies strive to deliver better outcomes through developing disease-state specific expertise

Regulatory Environment Specialty Regulatory Tie-in Regulatory Environment Specialty pharmacies build expertise in specific disease states and the medications that affect those disease states The regulatory environment heavily governs pharmacy practice State and Federal Regulations Three regulatory issues that affect practice and our patients: CMS consent Sterile Compounding Pedigree and Track and Trace Goal: Build awareness of the regulatory issues that impact pharmacy with leaders in ESRD care

Patient Consent Specialty Regulatory Tie-in Automatic refills -> potential for waste Patient complaints; consistent with decreasing fraud, waste, and abuse 2014 CMS – Part D sponsors to require pharmacies obtain patient consent prior to each delivery Goal: Decrease unintended waste, accumulation, costs What does that mean for you or our patients? Pharmacies must obtain patient consent prior to each delivery Patients have a right to put a prescription on hold or refuse treatment

Sterile Compounding Specialty Regulatory Tie-in September 2012 – multistate outbreak of fungal meningitis 751 patients affected in total, 25 in Florida (meningitis, stroke, spinal infections) 64 patients died, 7 in Florida Previous Background April 2002 – FDA issues Form 483 with findings February 2003 – FDA issues Form 483 with findings “…the potential for serious public health consequences if compounding practices are not improved…so long as a pharmacy’s operations fall within the scope of the practice of pharmacy, FDA will generally continue to defer to state authorities for regulatory oversight.” January 2007 – FDA Warning Letter October 2012 – FDA Form 483 FDA New England Compounding Center: http://www.fda.gov/AboutFDA/CentersOffices/OfficeofGlobalRegulatoryOperationsandPolicy/ORA/ORAElectronicReadingRoom/ucm325828.htm

FDA Guidance Specialty Regulatory Tie-in 2013 - Drug Quality and Security Act and Compounding Quality Act Establishes 503B outsourcing facilities Drugs must be compounded in compliance with cGMP 2013/2014 - Section 503A –Requirements for exemption for licensed pharmacists or licensed physicians compounding small quantities Section 503B – outsourcing facilities – must follow current good manufacturing practices “Inordinate amounts of compounded drug products interstate” Positive changes and guidance from the FDA to help guide sterile compounding

State of Florida Specialty Regulatory Tie-in 2007 - Standards of practice for compounding sterile preparations 2013/2014 – New Standards of Practice Adoption of USP Standards Sterile Compounding Permit Required – in- and out-of-state Pharmacy inspections – new inspectors; significantly different form Consultant pharmacist: provide guidance for Class IIb permit (eg. immediate use, beyond-use dating) Key Take Away: Significant Federal and State changes require that all pharmacies that supply CSPs must be permitted by the State of Florida and follow USP or cGMP guidelines for sterile compounding

Pedigree Specialty Regulatory Tie-in Counterfeit medications: Less than 1% in the US (WHO; FDA) Proactive FDA approach – as specialty items increase, greater economic incentives for counterfeiting FDA - Counterfeit Version of Botox Found in the United States. http://www.fda.gov/Drugs/DrugSafety/ucm443217.htm

Pedigree Specialty Regulatory Tie-in 2013 - Drug Supply Chain Security Act – Track and trace Goal is building an electronic, interoperable system over next 10 years Trading partners (manufacturers, wholesale distributors, and re-packagers) provide transaction documentation to dispensers Transaction information, transaction history, and transaction statements Trading partners maintain for ≥ 6 years after transaction Key Take Away: The pharmaceutical supply chain is becoming more regulated with eventual greater transparency and tracking of medications

Conclusions Specialty Regulatory Tie-in Pharmacies should be seen as a partner that can offer value added services for the patients we all treat Specialty Pharmacy refers to the clinical and practical expertise needed to dispense the range of medications that fit specific criteria Significant Federal and State changes require that all pharmacies that supply CSPs must be permitted by the State of Florida and follow USP or cGMP guidelines for sterile compounding The pharmaceutical supply chain is becoming more regulated with Track and Trace.

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