Plasma Protein Therapeutics Association

PPTA ADVOCATES AND PROMOTES SAFE AND EFFECTIVE PLASMA PROTEIN THERAPEUTICS.

AS A TRUSTED PARTNER TO HEALTH SYSTEMS, PPTA DRIVES BROAD AND RELIABLE ACCESS TO HIGH-QUALITY PLASMA PROTEIN THERAPIES, WITH A FOCUS ON THE WELL- BEING OF PATIENTS AND PLASMA DONORS. THE PLASMA PROTEIN THERAPEUTICS ASSOCIATION (PPTA) REPRESENTS A UNIQUE SECTOR OF THE BIOLOGICS AND BIOTECHNOLOGY INDUSTRY. PPTA REPRESENTS MORE THAN 1,000 HUMAN PLASMA COLLECTION CENTERS IN NORTH AMERICA AND EUROPE, AS WELL AS THE MANUFACTURERS OF LIFESAVING PLASMA PROTEIN THERAPIES. OUR MEMBERS PRODUCE APPROXIMATELY 80% OF THE PLASMA PROTEIN THERAPIES IN THE U.S. AND 60% OF THOSE MANUFACTURED IN EUROPE. PPTA WORKS GLOBALLY TO ADVOCATE FOR PATIENT ACCESS TO PLASMA-DERIVED MEDICINES, RESPONSIBLY IMPROVE THE AVAILABILITY OF PLASMA, AND ADVANCE THE UNDERSTANDING OF THE PLASMA ECOSYSTEM: -PROMOTE CLINICIAN AUTHORITY AND PATIENT PARTICIPATION IN TREATMENT DECISIONS -PURSUE REIMBURSEMENT MODELS RECOGNIZING THE UNIQUE NATURE OF PLASMA PROTEIN THERAPIES -SHAPE DECISIONS OF AUTHORITIES TO IMPROVE PLASMA PROTEIN THERAPY AVAILABILITY -FORAGE PARTNERSHIPS THAT SUPPORT PATIENT-FOCUSED INITIATIVES -ADVOCATE FOR GLOBAL SUFFICIENCY OF PLASMA -PIONEER PLASMA SAFETY AND MANUFACTURING QUALITY AND SUPPORT DONOR WELL- BEING -DRIVE PROGRESSIVE POLICIES FOR PLASMA COLLECTIONS -ENCOURAGE PLASMA DONATION, GLOBALLY -GENERATE AND PROMOTE EVIDENCE-BASED INFORMATION ABOUT PLASMA -EXPLAIN THE IMPORTANCE, UNIQUE NATURE, AND VALUE OF PLASMA PROTEIN THERAPIES PLASMA AND PLASMA COLLECTION: PLASMA IS THE CLEAR, STRAW-COLORED LIQUID PORTION OF BLOOD THAT REMAINS AFTER RED BLOOD CELLS, WHITE BLOOD CELLS, PLATELETS AND OTHER CELLULAR COMPONENTS ARE REMOVED. IT IS THE SINGLE LARGEST COMPONENT OF HUMAN BLOOD, COMPRISING ABOUT 55 PERCENT, AND CONTAINS WATER, SALTS, ENZYMES, ANTIBODIES AND OTHER PROTEINS. -COMPOSED OF 90% WATER, PLASMA IS A TRANSPORTING MEDIUM FOR CELLS AND A VARIETY OF SUBSTANCES VITAL TO THE HUMAN BODY. -PLASMA CARRIES OUT A VARIETY OF FUNCTIONS IN THE BODY, INCLUDING CLOTTING BLOOD, FIGHTING DISEASES AND OTHER CRITICAL FUNCTIONS. -SOURCE PLASMA IS PLASMA THAT IS COLLECTED FROM HEALTHY, VOLUNTARY DONORS THROUGH A PROCESS CALLED PLASMAPHERESIS AND IS USED EXCLUSIVELY FOR FURTHER MANUFACTURING INTO FINAL THERAPIES (FRACTIONATION). SOURCE PLASMA DONORS MAY BE COMPENSATED FOR THEIR TIME AND EFFORT. -RECOVERED PLASMA IS COLLECTED THROUGH WHOLE BLOOD DONATION IN WHICH PLASMA IS SEPARATED FROM ITS CELLULAR COMPONENTS. RECOVERED PLASMA MAY BE USED FOR FRACTIONATION. SOURCE PLASMA IS COLLECTED THROUGH A PROCESS CALLED PLASMAPHERESIS. IN MORE THAN 1,000 SPECIALIZED DONATION CENTERS LOCATED IN THE U.S. AND EUROPE, INDIVIDUALS MAY DONATE PLASMA. PLASMAPHERESIS IS A STERILE, SELF-CONTAINED, AUTOMATED PROCESS THAT SEPARATES PLASMA FROM RED BLOOD CELLS AND OTHER CELLULAR COMPONENTS WHICH ARE THEN RETURNED TO THE DONOR. SOURCE PLASMA COLLECTION IN THE U.S. IS REGULATED BY THE U.S. FOOD AND DRUG ADMINISTRATION (FDA) AND, IN EUROPE, BY THE EUROPEAN MEDICINES AGENCY (EMA) AND NATIONAL REGULATORY AUTHORITIES. PLASMA MANUFACTURING: SOURCE AND RECOVERED PLASMA IS THE STARTING MATERIAL USED TO MANUFACTURE LIFESAVING THERAPIES. THE MANUFACTURING PROCESS IS KNOWN AS FRACTIONATION. PROTEINS ARE SEPARATED THROUGH THIS PROCESS TO CREATE NUMEROUS PLASMA PROTEIN THERAPIES. THIS PROCESS IS CARRIED OUT USING WELL-ESTABLISHED PURIFICATION METHODS SUCH AS PRECIPITATION, CENTRIFUGATION, SEPARATION, AND FILTRATION. PLASMA IS POOLED AND PROCESSED THROUGH A PROCESS CALLED "FRACTIONATION" THAT EMPLOYS TIME, TEMPERATURE, PH, AND ALCOHOL CONCENTRATIONS TO EXTRACT SPECIFIC THERAPEUTIC PROTEINS. THESE ARE THEN SUBJECTED TO VARIOUS PURIFICATION METHODS AND VIRAL INACTIVATION AND REMOVAL PROCESSES TO FURTHER ENSURE THEIR SAFETY AND EFFICACY. PREPARING A THERAPY OFTEN TAKES FROM 7-12 MONTHS BETWEEN DONATION AND FINAL PRODUCT RELEASE. THIS SETS THE PRODUCTION OF PLASMA PROTEIN THERAPIES APART FROM CHEMICAL PHARMACEUTICALS AND OTHER BIOLOGICS WHOSE MANUFACTURING PROCESSES ARE MUCH MORE CONDENSED AND WHOSE DIRECT MANUFACTURING COSTS ARE A SIGNIFICANTLY SMALLER PORTION OF THE OVERALL COST. VOLUNTARY STANDARDS: IN ADDITION TO MEETING REGULATORY REQUIREMENTS, PPTA MEMBER SOURCE PLASMA COLLECTION CENTERS ARE ALSO CERTIFIED BY THE INTERNATIONAL QUALITY PLASMA PROGRAM (IQPP); A RIGOROUS, VOLUNTARY PROGRAM THAT GOES BEYOND REGULATORY REQUIREMENTS TO HELP ENSURE DONOR SAFETY AND FURTHER IMPROVE THE QUALITY OF SOURCE PLASMA USED FOR FRACTIONATION. THESE STANDARDS FOCUS ON DONOR MANAGEMENT, DONOR HEALTH AND CENTER MANAGEMENT: -COMMUNITY-BASED DONORS -DONOR ADVERSE EVENTS REPORTING -DONOR EDUCATION -DONOR FLUID ADMINISTRATION -DONOR DEFERRAL REGISTRY -QUALIFIED DONOR -VIRAL MARKER -CENTER PERSONNEL EDUCATION AND TRAINING -PROFESSIONAL FACILITY -QUALITY ASSURANCE PPTA HAS ALSO ESTABLISHED THE QUALITY STANDARDS OF EXCELLENCE AND LEADERSHIP (QSEAL) PROGRAM TO PROVIDE INDEPENDENT CERTIFICATION OF ADHERENCE BY FRACTIONATORS TO THE VOLUNTARY STANDARDS. THESE VOLUNTARY STANDARDS COVER: -CONTROLS ON INCOMING PLASMA -INVENTORY HOLD -TESTING FOR HIV, HBV, HCV, HAV AND PARVOVIRUS B19 -INTERMEDIATES PURCHASED FROM AN EXTERNAL SUPPLIER -RECOVERED PLASMA -QUALIFIED DONOR -VIRAL MARKER NATIONAL DONOR DEFERRAL REGISTRY: THE NATIONAL DONOR DEFERRAL REGISTRY (NDDR) HELPS ENSURE THAT DEFERRED DONORS CANNOT DONATE THEIR PLASMA AGAIN. THE NDDR IS A DATABASE OF PERMANENTLY DEFERRED PLASMA DONORS IN NORTH AMERICA. IT IS ONE OF MANY VOLUNTARY, SELF-REGULATING INITIATIVES TAKEN BY THE SOURCE PLASMA COLLECTION INDUSTRY AND IS AN IMPORTANT COMPONENT OF THE INDUSTRY-DRIVEN SAFETY MEASURES THAT HELP ENSURE THE SAFETY OF THE FINAL THERAPIES. PATIENT NOTIFICATION SYSTEM: IN 1998, THE PLASMA PROTEIN THERAPEUTICS ASSOCIATION (PPTA) AND ITS MEMBERS COLLABORATED WITH PATIENT ORGANIZATIONS TO DEVELOP THE PATIENT NOTIFICATION SYSTEM (PNS). THIS SYSTEM PROVIDES REGISTERED PATIENTS WITH EASILY ACCESSIBLE, COMPREHENSIVE, AND UP-TO-DATE WITH INFORMATION ABOUT ALL BRANDS OF IMMUNE GLOBULINS, BLOOD CLOTTING FACTORS, ALPHA-1 PROTEINASE INHIBITORS, AND OTHER LIFESAVING PLASMA PROTEIN THERAPIES. MAINTAINING REGISTRANT CONFIDENTIALITY IS A PRIMARY CONSIDERATION. TO ENSURE CONFIDENTIALITY, THE PATIENT NOTIFICATION SYSTEM IS OPERATED AN INDEPENDENT VENDOR THAT SPECIALIZES IN PHARMACEUTICAL NOTIFICATIONS. ALL REGISTRANTS, PATIENTS, PHYSICIANS, FAMILY MEMBERS, NURSES, OR PHARMACISTS, ARE GUARANTEED THAT THEIR INFORMATION IS NEVER SHARED NOR IS IT ACCESSIBLE BY ANYONE OTHER THAN THE THIRD-PARTY COMPANY THAT HOUSES THE COMPUTERS TO RUN THE SYSTEM AND SEND THE NOTIFICATIONS.

THE NORTH AMERICA DATA PROGRAM IS A VOLUNTARY MANUFACTURER INITIATIVE UNDERTAKEN IN RESPONSE TO DRUG SHORTAGE CONCERNS, AND IS INTENDED TO PROVIDE A RELIABLE, NATION-WIDE ASSESSMENT OF THE AVAILABILITY OF THESE LIFE-SAVING THERAPIES IN THE UNITED STATES. PPTA'S WEB-BASED SYSTEM PROVIDES AGGREGATE, MONTHLY DISTRIBUTION DATA FOR THE FOLLOWING CATEGORIES OF PLASMA PROTEIN THERAPIES: 1. ALBUMIN 5% 2. ALBUMIN 25% 3. IMMUNE GLOBULIN (IG) 4. PLASMA-DERIVED FACTOR VIII 5. RECOMBINANT FACTOR VIII