BILL ANALYSIS                                                                                                                                                                                                    






                                 SENATE HEALTH
                               COMMITTEE ANALYSIS
                        Senator Elaine K. Alquist, Chair


          BILL NO:       SB 971                                       
          S
          AUTHOR:        Pavley                                       
          B
          AMENDED:       April 7, 2010                               
          HEARING DATE:  April 14, 2010                               
          9
          REFERRAL       Business, Professions and Economic  
          Development    7
          CONSULTANT:                                                 
          1
          Orr/                                                       
                                     SUBJECT
                                         
                  Bleeding disorders: blood clotting products

                                     SUMMARY  

          Establishes standards of service and storage for entities  
          that deliver blood clotting products and related equipment  
          and supplies for home usage to people with hemophilia and  
          other bleeding disorders.  Requires the Board of Pharmacy  
          to administer and enforce these standards. 

                             CHANGES TO EXISTING LAW  

          Existing federal law:
          Requires the U.S. Food and Drug Administration (FDA) to  
          regulate the manufacture of pharmaceuticals derived from  
          blood and blood components, such as blood clotting  
          products, including establishing standards for those  
          products for shipping, storage, and delivery, inspecting  
          the products, approving them for use by patients, and  
          recalling products that may be defective or potentially  
          harmful.
          
          Existing state law:
          Establishes various state programs, administered by the  
          Department of Health Care Services (DHCS), that provide  
          medical care for persons with certain genetically based  
          diseases, such as the Genetically Handicapped Person's  
                                                         Continued---



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          Program (GHPP). GHPP provides medical care for qualified  
          adults with genetically handicapping conditions, including  
          hemophilia. Another such program, the California Children's  
          Services Program (CCS), provides health care for qualified  
          children with various diseases, including hemophilia or  
          other bleeding disorders. 

          Requires pharmacies to be licensed by the California Board  
          of Pharmacy.  California pharmacies must meet specified  
          statutory and regulatory requirements, including those  
          relating to proper storage, handling, dispensing, and  
          disposal of drugs, staff training protocols, drug and  
          supply inventory, labeling, and maintenance of patient  
          confidentiality.  Existing law authorizes pharmacists to  
          perform specified procedures, in accordance with procedures  
          developed by physicians and health facility administrators,  
          including ordering or performing routine drug  
          therapy-related patient assessments, ordering laboratory  
          tests, initiating or adjusting a patient's drug regimen,  
          and administering drugs and biologics, such as blood  
          clotting products, by injection, pursuant to a prescriber's  
          order.  

          Existing state regulation: 
          Prohibits pharmacies from leaving, picking up, accepting,  
          or delivering prescriptions to or from any place not  
          licensed as a retail pharmacy, with certain exceptions,  
          including a patient's residence or workplace, or a licensed  
          health facility, as specified.
          
          This bill:
          Establishes the Standards of Service for Providers of Blood  
          Clotting Products for Home Use Act. Institutes the  
          following requirements for providers of blood clotting  
          products for home use:
               1.     Have sufficient knowledge and understanding of  
                 bleeding disorders, have sufficient clinical  
                 expertise, and ensure the same of customer service  
                 staff.
               2.     Have an on-call pharmacist or staff available  
                 24 hours a day to handle emergency requests for  
                 clotting factor, maintain on-call services all day,  
                 every day of the year, screen calls for emergencies,  
                 and respond to all calls within one hour.  Provide  
                 customers with a contact number for reporting  
                 problems with their delivery, and respond to  




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                 problems immediately. 
               3.     Maintain adequate stocks of clotting products  
                 and ancillary infusion equipment, such as syringes  
                 and tourniquets, and must supply all brands of  
                 approved clotting products in multiple sizes and  
                 amounts, provided manufacturer supply exists and  
                 payer authorization is approved. Providers are  
                 excluded from making substitutions of clotting  
                 products or amounts. 
               4.     Store, ship, and deliver products in accordance  
                 with the product's approved package insert and state  
                 and federally mandated standards, and ensure  
                 nonemergency prescriptions are sent within two  
                 business days of the approved authorization for the  
                 request.  Ensure that emergency prescriptions are  
                 delivered to patients within 12 hours if the patient  
                 lives within 100 miles of a major metropolitan  
                 airport, and within one day for patients living  
                 outside that radius. 
               5.     Ensure the qualifications of the prescribed  
                 home nursing services rendered or arranged by the  
                 provider.
               6.     Alert patients to recalls and withdrawals of  
                 blood clotting products and ancillary equipment, and  
                 participate in the National Patient Notification  
                 System for product recalls. 
               7.     Provide language translation services as needed  
                 by the customer.
               8.     Have a detailed plan for meeting these  
                 requirements in the event of a natural disaster or  
                 other disruption of normal business operations. 
               9.     Provide for collection, removal and disposal of  
                 hazardous waste.
               10.    Inform customers of their payment  
                 responsibilities, including copays, deductibles and  
                 coinsurance payments, and provide customers with  
                 copies of billing invoices, and retain copies of  
                 their prescriptions. 
               11.    Comply with federal and state recordkeeping and  
                 documentation requirements, and federal privacy and  
                 confidentiality requirements. 

          Defines provider of blood clotting product for home use as  
          a seller or provider of blood clotting products, ancillary  
          infusion equipment, home nursing services and patient  
          assistance for the management of bleeding disorders for  




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          home use. This would include specialty homecare care  
          pharmacies and hemophilia treatment centers that contract  
          with outside pharmacies. This also includes health system  
          pharmacies, hospital pharmacies, and retail pharmacies.  

          Requires prescribing physicians to name specific blood  
          clotting products when issuing prescriptions.

          Requires the state Board of Pharmacy to enforce these  
          provisions. 

          Establishes various other definitions in the context of the  
          bill, and makes findings and declarations regarding  
          hemophilia and other bleeding disorders. Establishes that  
          the purpose of the article is to establish standards of  
          service for entities that deliver blood clotting products  
          and related equipment, supplies and services for home use,  
          and promote access to equipment, supplies and services for  
          home use for persons with hemophilia and other bleeding  
          disorders. 

                                  FISCAL IMPACT  

          This bill has not been analyzed by a fiscal committee. 

                            BACKGROUND AND DISCUSSION  

          According to the author, there are currently no standards  
          in state law governing the proper storage and delivery of  
          blood clotting products and clotting factor, a prescribed  
          biologic that is infused several times per week by people  
          with hemophilia and other bleeding disorders. The intent of  
          this bill is to place standards in state law for the proper  
          storage and delivery of blood clotting factor and other  
          related equipment and supplies for home usage.  The author  
          believes it is critical to place life-saving standards in  
          law for the benefit of people with hemophilia and other  
          bleeding disorders, as well as to maintain the current  
          cost-effective model of care for future generations. 
          
          Hemophilia and other bleeding disorders
          Hemophilia is a genetically inherited bleeding disorder  
          that affects approximately 4,000 Californians, primarily  
          males.  Those with hemophilia are missing or have  
          dysfunctional proteins in their blood, called clotting  
          factors, which work to stop bleeding.  The lack of clotting  




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          factor causes hemophiliacs to bleed for longer periods of  
          time, and generally results in internal bleeding, primarily  
          into muscles and joints.  Without treatment, hemophilia can  
          cause pain, severe joint damage, disability, and early  
          death.  

          Bleeding disorders include von Willebrand disease, a blood  
          clotting disorder similar to hemophilia that is caused by a  
          deficiency or defect of a blood clotting protein called von  
          Willebrand factor which helps blood platelets to clot and  
          to seal off tears in injured blood vessels.  Both men and  
          women are affected by von Willebrand disease, which is  
          generally the least severe of the clotting disorders. 

          
          Blood clotting products 
          According to the National Hemophilia Council, bleeding  
          disorders may occur spontaneously or after mild to severe  
          trauma. These bleeding disorders are treated by replacing  
          the missing clotting factor in the blood by injecting  
          additional blood clotting factors into the bloodstream. The  
          timing and severity of bleeding episodes are unpredictable,  
          even for patients on regularly scheduled treatment; thus  
          providers of clotting factor concentrates must be able to  
          effectively respond to varying frequency and dosing needs. 

          Clotting factors used to treat hemophilia are FDA-approved,  
          and may only be used with a prescription.  Clotting factors  
          may be found in whole blood, plasma, and recombinant  
          factors, which are made in a laboratory and do not use  
          human blood products. A variety of blood clotting products,  
          produced by various pharmaceutical manufactures, can be  
          prescribed to temporarily replace the missing clotting  
          factors in order to prevent or correct bleeding episodes.   
          To properly administer blood clotting factors, patients  
          must often use ancillary supplies and equipment, such as  
          syringes, tourniquets, gauze, and alcohol swabs. 

          Blood clotting products are generally expensive, and  
          require special handling.  They should generally be stored  
          at certain temperatures, and are sensitive to extreme  
          exposure to light.

          Hemophilia treatment centers (HTCs)
          HTCs provide comprehensive diagnostic and treatment  
          services for individuals with hemophilia and other bleeding  




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          disorders, through interdisciplinary teams that include  
          hematologists, nurses, social workers, physical therapists,  
          and orthopedists.  HTCs provide case management services,  
          and often coordinate care with primary care providers and  
          specialists such as dentists, genetic counselors, and  
          obstetrician/gynecologists.  HTCs provide patient education  
          on self-care, and approximately 90 percent of severe  
          hemophilia patients seen at HTCs self-infuse their clotting  
          factors.

          HTCs are federally funded, and provide care in accordance  
          with goals established by the U.S. Department of Health and  
          Human Services Maternal and Child Health Bureau, and the  
          Centers for Disease Control and Prevention (CDC), as well  
          as standards established by the National Hemophilia  
          Foundation.  In California, HTCs are primarily based at  
          university-based tertiary care hospitals, including the  
          Lucile Packard Children's Hospital of Stanford, University  
          of California (UC) Davis Children's Hospital, and the  
          Schools of Medicine at UC San Francisco and UC San Diego.  
          HTCs in California don't directly dispense clotting factor,  
          but through the 340 B federal pricing program, they have  
          contracts w/ outside pharmacies to distribute/dispense  
          clotting factor to patients in the home setting.

          Specialty care pharmacies 
          Many blood clotting product providers in California are  
          considered specialty care pharmacies.  These pharmacies  
          specialize in the provision of treatments and supplies to  
          individuals with specific chronic diseases.  They generally  
          concentrate their services on their target patient  
          population, rather than providing services that meet the  
          needs of the general public.  Specialty care pharmacies  
          must comply with all statutory and regulatory requirements  
          as traditional pharmacies, but do not need to meet  
          additional licensing requirements in order to provide their  
          specialty services. 

          Specialty pharmacies often ship or deliver blood clotting  
          products to the homes of individuals with hemophilia, and  
          other bleeding disorders, and when necessary, provide or  
          arrange for nursing services to patients who may need  
          assistance with infusion of the blood clotting products.

          Medi-Cal contracting requirement
          As noted in a March 2010 Provider Bulletin, the Department  




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          of Health Care Services (DHCS) has received statutory  
          authority to begin contracting with outpatient providers of  
          specialized drugs, including blood factors, in order to  
          meet the unique specialized care needs of the Medi-Cal  
          population who utilize specialty drugs.  Effective July 1,  
          2010, DHCS will contract with "any specialty pharmacy that  
          will sign a contract to meet a list of performance  
          obligations. These include, but are not limited to,  
          delivery time requirements, providing patient education and  
          submitting quarterly and yearly reports to DHCS. Providers  
          who do not sign an agreement to become a provider under  
          these provisions will no longer be allowed to provide the  
          specialized drug to Medi-Cal, California Children's  
          Services (CCS) or Genetically Handicapped Persons Program  
          (GHPP) recipients."
          
          Prior legislation
          SB 1594 (Steinberg) of 2008 would have imposed requirements  
          on providers of blood clotting products for home use that  
          are used to treat hemophilia and other bleeding disorders.  
          Failed passage in Senate Appropriations Committee.
          
          AB 2408 (Negrete-McLeod), Chapter 777 of the Statutes of  
          2007, defined the scope of professional practice for  
          pharmacists to include authorization for pharmacists to  
          administer drugs and biologics by injection, pursuant to a  
          prescriber's order.  

          Arguments in support
          The Plasma Protein Therapeutics Association represents  
          manufacturers of plasma-derived and recombinant biological  
          therapies, collectively known as plasma protein therapies.  
          They believe this bill will ensure that residents of  
          California affected by bleeding disorders have access to  
          medically appropriate care.  Delayed access to clotting  
          factor can cause painful and crippling injury to the joints  
          and organs, which can lead to increased costs for  
          hospitals, skilled nursing and other specialty services.  
          Grifols, Inc, a producer of biologic therapies used to  
          treat bleeding disorders, claims that each product is a  
          distinct, sole-source product and none have generic  
          equivalents. They write in support of the bill, citing the  
          importance of preserving access to all of the life-saving  
          therapies available because patients respond differently to  
          different brands of therapy. 
          




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          Arguments in opposition
          The California Society of Health-System Pharmacists  
          recognizes the value of providing the proper medications in  
          a timely manner to individuals with hemophilia. However,  
          they believe this bill contains several specific provisions  
          that have serious consequences for pharmacists, and creates  
          barriers to providing quality and timely care in an  
          affordable manner.  They believe many of the provisions of  
          the bill are impossible to implement because many of the  
          hemophilia drugs require prior approval from the health  
          insurer of the patient. The requirement in the bill for  
          providers to supply all brands of clotting factors approved  
          by the FDA in multiple assays and vial sizes they feel  
          creates undue financial burden on pharmacies and provider  
          organizations if they are required to have all of these  
          drugs in stock. 
          

                                     COMMENTS
             
            1.   Definition of provider. The current definition of  
            provider indicates that in order to be considered a  
            provider of blood clotting products, entities must also  
            provide "ancillary infusion equipment, home nursing  
            services, and patient assistance for the management of  
            bleeding disorders for home use." Not all entities  
            currently providing blood clotting products fulfill each  
            of these requirements, particularly the requirement to  
            provide home nursing services. A suggested amendment  
            would be to delete Page 5, lines 6-9 and insert the  
            following: "Provider of blood clotting products for home  
            use" includes but is not limited to any hospital  
            pharmacies, health system pharmacies, pharmacies  
            affiliated with hemophilia treatment centers, specialty  
            home care pharmacies, and retail pharmacies that provide  
            blood clotting products and ancillary infusion equipment  
            for home use, and patient assistance for the management  
            of bleeding disorders. These providers may also provide  
            home nursing services for persons with bleeding  
            disorders.

            2.  Clarifying amendments. There are ambiguities in some  
            of the bill language. The author may wish to clarify what  
            is meant by "sufficient knowledge and understanding of  
            bleeding disorders," and "sufficient clinical expertise."  





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            3.  Technical amendment. The requirement for physicians  
            to specify the type of clotting product to use may not be  
            enforceable by the Board of Pharmacy, because the Board  
            does not have jurisdiction over prescribing physicians.  
            Suggested amendment:  Page 7, line 23, after "article"  
            insert "with the exception of subsection (f) of section  
            125286.4." 


                                    POSITIONS  


          Support:  Hemophilia Council of California (sponsor)
                 California Medical Association
                 CSL Behring 
                 Grifols, Inc. 
                 Herndon 1 Pharmacy
                 Plasma Protein Therapeutics Association
                 
          Oppose:  California Pharmacists Association
                 California Society of Health-System Pharmacists

                                   -- END --