Interdisciplinary Perspectives on Development of Naming Standards for Biosimilar Medicines

Transcription

1 Interdisciplinary Perspectives on Development of Naming Standards for Biosimilar Medicines Richard Dolinar, MD Chairman, Alliance for Safe Biologic Medicines

2 Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to Drug Information Association, Inc. ( DIA ), its directors, officers, employees, volunteers, members, chapters, councils, Communities or affiliates, or any organization with which the presenter is employed or affiliated. These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United States of America and other countries. Used by permission. All rights reserved. Drug Information Association, Drug Information Association Inc., DIA and DIA logo are registered trademarks. All other trademarks are the property of their respective owners. 2

3 About ASBM 2010 ASBM formed to provide STAKEHOLDER GUIDANCE to FDA in development of US Biosimilar Pathway STEERING COMMITTEE STEERING COMMITTEE composed of physician and patient groups, runs day-to-day operations. ADVISORY BOARD: Composed of Physicians, Researchers, Pharmacists, and Patients from around the world. Serves as resource on the science and clinical use of biosimilars, guides our policy recommendations. 3

4 ASBM Serves as a Resource for People Around The World Who Wish To Learn More About Biosimilars Edmonton, Canada United Kingdom Netherlands Brussels, Belgium Versailles, France Dusseldorf, Germany Madrid, Spain Ankara, Turkey Porto, Portugal Msida, Malta Kunming, China Venezuela Madras, India Brazil South Africa

5 The Four Pillars ASBM S GUIDING PRINCIPLES PRIORITIZING PATIENT SAFETY LEVERAGING WHAT WE HAVE LEARNED PROMOTING PHARMACO- VIGILANCE PROTECTING THE PATIENT- PHYSICIAN RELATIONSHIP

6 Review: Why Are Biologics Different Than Chemical Drugs?

7 Biologic vs. Chemical Medicines SIZE: significantly larger STRUCTURE: more complex STABILITY: susceptible to light, heat, denaturing, and degradation SENSITIVITY: small manufacturing changes can cause AE's DRIFT: can change with time 7

8 Molecular Comparison: Aspirin vs. Monoclonal Antibody Source: New England Journal of Medicines, Developing the Na=on s Biosimilars Program, August 4,

9 Example of Extreme Sensitivity of Biologics Pure Red Cell Aplasia (PRCA) In 1998, Johnson & Johnson substituted the stabilizer polysorbate 80 for human serum albumin in EPREX/ ERYPO prefilled syringes marketed outside the US. Manufacturer received increased reports of erythropoietin antibody-mediated PRCA in patients who had received EPREX/ERYPO in the subcutaneous dosage form to treat anemia associated with chronic renal failure. After years of investigations, likely cause revealed: uncoated rubber stoppers, when exposed to polysorbate 80, released organic compounds (leachates) into the formulation, enhancing immunogenicity.

10 Small Differences Source: Bilao LLC,

11 Small Differences = Large Impact Testosterone Progesterone Estradiol Source: Bilao LLC,

12 Biosimilar Naming: Physician

13 ASBM Surveys PROVIDE EMPIRICAL DATA regarding perspectives on these new medicines by those most familiar with their clinical use. U.S. Prescriber Survey (September 2012) E.U. Prescriber Survey (November 2013) Canadian Prescriber Survey (planning for 2014)

14 Goal of the WHO s INN Programme: Ensuring the Medicine Prescribed is the Medicine Received. ASBM has twice presented its survey findings to the WHO to aid in the development of international naming standards for biosimilars: U.S. Prescriber Survey results presented at the 57 th Consultation on International Nonproprietary Names (INN), October 22, 2013 European Prescriber Survey results presented at 58 th INN Consultation, April 8,

15 Challenges Facing the WHO DISTINGUISHABLE NAMES WHO has attempted to move from Do we need biosimilars to have distinguishable names? to What form would a distinguishable name take? A shared root with a biological qualifier has been proposed. 15

16 Challenges Facing the WHO NATIONAL REGULATORY AUTHORITIES There is a patchwork of naming standards by the world s NRA s. Some use distinguishable names. Some don t. Some have no consistent policy. A voluntary global standard relies upon cooperation. 16

17 Benefits of the Proposed BQ System For countries whose National Regulatory Authorities have STRONG pharmacovigiliance systems, adopting the BQ would provide an ADDITIONAL SAFEGUARD to avoid a single point of information failure. While potentially redundant, our survey data indicates that prescribers do not in fact always record all the required data when reporting adverse events. For countries whose National Regulatory Authorities have WEAK pharmacovigiliance systems, adopting the BQ will provide a valuable international framework for clarity and patient safety.

18 Support for the Biological Qualifier among NRAs Proposed at 57 th INN, October 22, 2013 Proposal has the support of representatives from Japan and Australia, which have or are developing their own BQ standards, but expressed willingness to conform with the global standard. EMA thought it unlikely that a biological qualifier would be adopted in Europe but did not rule it out. FDA has indicated its support for distinguishable names, as in the case of tbofilgrastim, but have not issued any formal policy for biosimilar naming.

19 Prescribing by INN: Encouraged, even Required in Much of the World REQUIRED by NRAs in China, Colombia, Latvia. ENCOURAGED by NRAs in India, Netherlands. PROPOSED as requirement in Russia. In many countries, it is at the DISCRETION of the prescriber whether to use INN or brand name. 19

20 Distinguishable INNs Aid in Pharmacovigilance IDENTIFICATION Distinguishable INNs help accurately identify the product, PHARMACOVIGILANCE Distinguishable INNs help connect adverse events to the products that caused them Distinguishable INNs help avoid a single point of information failure. MANUFACTURER ACCOUNTABILITY Distinguishable INNs help ensure that manufacturers are accountable for their products.

21 GABI White Paper on E.U. Prescriber Survey 21

22 E.U. Prescriber Survey First large-scale survey on biosimilars in Europe. Examined physician knowledge and prescribing practices. 15-Minute Web-based Survey 470 Prescribers distributed equally between 5 countries in Western Europe: France Germany Italy Spain United Kingdom Roughly equal distribution between six specialties in which biologics are frequently prescribed. 18% Nephrology 17% Rheumatology 17% Dermatology 16% Oncology 16% Endocrinology 16% Neurology 22 Conducted by Industry Standard Research, October-November 2013

23 E.U. Prescriber Survey: Familiarity Level with Biosimilars 54% possess only a basic understanding of biosimilars 20% 4% 22% Only 22% consider themselves very familiar with biosimilars 20% have heard of them but cannot define 54% 4% have never heard of them Very Familiar - Complete understanding Familiar - Basic understanding Heard of them - Can't define Never heard of them 23

24 E.U. Prescriber Survey Same Nonproprietary Name = Structurally Identical? If two medicines have the same non-proprietary scientific name, does this suggest to you or imply that the medicines are structurally identical? (N=470) 53% of respondents mistakenly believe biosimilars with identical nonproprietary name as its reference biologic is structurally identical to that reference biologic. Yes No No Opinion 15% 53% 32% 24

25 E.U. Prescriber Survey Same Non-proprietary name = Same Indications? If two medicines have the same non-proprietary scientific name, does this suggest to you or imply that the medicines are approved for the same indications? (N=470) 61% of respondents believe biosimilars with an identical nonproprietary name as its reference biologic is approved for the same indications, which may not be the case. Yes No No Opinion 9% 31% 61% 25

26 E.U. Prescriber Survey Identifying Biologic Medicines in Patient Record Only 32% of respondents use brand name and nonproprietary name (INN) to identify the exact biologic being prescribed. 24% use INN only, which could result in patients receiving the wrong medicine. 24% 14% 30% 32% Brand and Generic Brand Only Generic Only Varies by Medicine 26

27 E.U. Prescriber Survey: Batch Number Inclusion How often do you include the batch number when reporting adverse events? (N=470) Always Sometimes Never 27% of prescribers NEVER include. 33% only SOMETIMES include. 40% ALWAYS include. 27% 33% 40% 27

28 Summary: Key Findings of Surveys There is a need for further education about biosimilars among physicians. The current patchwork approach of each NRA developing its own naming system is not adequate to ensure that patients receive the medicine prescribed by their physician. Distinguishable nonproprietary names are important to practicing physicians, and it is our hope that this will be useful to WHO in crafting a global standard that will improve safety for patients worldwide. 28

29 Thank you Richard Dolinar, MD Chairman, Alliance for Safe Biologic Medicines Join the conversation #dia