Biotechnology Forum 2015

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1 Biotechnology Forum 2015 WHO Guidelines on scientific principles for regulatory risk assessment of biotherapeutic products Mexico, 23 rd February 2015 Dr Ivana Knezevic, WHO/HIS/EMP 1

2 Outline WHO International Standards - written (eg, Guidelines, Recommendations) - measurement (Int. Standards and Reference Preparations) Resolution on BTP and SBP: WHA th ICDRA recommendations Regulatory Risk Assessment new document Plan for

3 WHO Biological Standardization WHO has played a key role for over 60 years in establishing the WHO Biological Reference Materials necessary to standardize biological materials as well as developing WHO guidelines and recommendations to assure the quality, safety, and efficacy of biological products. These norms and standards, based on scientific consensus achieved through international consultations, assist WHO Member States in ensuring the quality and safety of biological medicines and related in vitro biological diagnostic tests worldwide. The Organization accomplishes this biological standardization work through its biological programme coordinated by a Secretariat at WHO HQ; the WHO Expert Committee on Biological Standardization (ECBS) selected from an Expert Advisory Panel on Biological Standardization; and WHO Collaborating Centres for Biological Standardization. 3

4 WHO norms and standards for biologicals Global written standards Global measurement standards Scientific evidence 1) Standardization of assays 2) Further development and refinement of QC tests 3) Scientific basis for setting specifications Reference preparations for vaccines and biotherapeutics Measurement standards: essential elements for development, licensing and lot release 4

5 WHO standards for Biotherapeutic Products (BTP) 1. Written (eg, Guidelines, Recommendations): - Guidelines on BTP made by rdna technology, WHO TRS 977 ECBS 2013 (requested by ICDRA 2010) - Guidelines on SBP (Similar Biotherapeutic Products), WHO TRS 987 ECBS 2009 (requested by ICDRA 2006) - Guidelines in the pipeline to be submitted to the ECBS 2015: Regulatory Expectations and Risk Assessment of Biotherapeutic Products (requested by ICDRA 2010) 2. Measurement (Int. Standards and Reference Preparations) - information available on the following links: - WHO web ( - NIBSC web ( Additional information: 1. Review article by Thorpe R, Wadhwa M, Biologicals 2011 (requested by ICDRA 2010) 2. Link to special issue in Biologicals (2011), 39 devoted to SBPs: 25 articles with WHO experience in working with regulators worldwide: th ICDRA recommendations published in WHO Drug Information Vol. 28, No. 3,

6 Timeline for WHO Written Standards: Biotherapeutics rdna RERA for BTP SBP Informal consultation ECBS submission Implementation workshop 6

7 WHA 67.21: Urges Member States to develop or strengthen, as appropriate, national regulatory assessment and authorization frameworks, with a view to meeting the public health needs for BTPs, including SBPs; to develop the necessary scientific expertise to facilitate development of solid, scientifically-based regulatory frameworks that promote access to products that are affordable, safe, efficacious and of quality, taking note of the relevant WHO guidelines that may be adapted to the national context and capacity; to work to ensure that the introduction of new national regulations, where appropriate, does not constitute a barrier to access to quality, safe, efficacious and affordable BTPs, including SBPs; 7

8 WHA 67.21: Requests WHO to support Member States in strengthening their capacity in the area of the health regulation of BTPs, including SBPs; to support, as appropriate, the development of national regulatory frameworks that promote access to quality, safe, efficacious and affordable BTPs, including SBPs; to encourage and promote cooperation and exchange of information, as appropriate, among Member States in relation to BTPs, including SBPs; to convene the WHO Expert Committee on Biological Standardization to update the 2009 guidelines, taking into account the technological advances for the characterization of BTPs and considering national regulatory needs and capacities and to report on the update to the Executive Board; to report to the Sixty-ninth World Health Assembly on progress in the implementation of this resolution. 8

9 16 th ICDRA recommendations Biosimilars (Workshop H) part 3 3. WHO guidelines on biotherapeutic products and on SBP 3.1. Member States Implement existing WHO guidelines and subsequent updates in full, and monitor levels of implementation over time. If national standards differ from WHO standards, inform WHO of the rationale for this situation WHO Amend Guidelines on evaluation of SBP by providing additional information on: - extrapolation of indication; - special considerations for evaluation of monoclonal antibodies; - acceptance criteria and evaluation of reference biotherapeutic products (RBP) including the reliance on reference agencies; - the design, conduct and interpretation of data for comparability exercise. 9

10 ICDRA recommendations Biosimilars (Workshop H) 3.2 cont. Facilitate implementation of existing guidelines on SBP (adopted in 2009), and subsequent updates, and on biotherapeutic products made by recombinant DNA technology (adopted in 2013). Develop e-learning tools for different levels (e.g. basic, advanced). Prepare case studies for illustrating practical application of guiding principles to different scenarios, e.g. mimic the real situation. Make all materials from implementation workshop (i.e. lectures, discussions, and case studies) available to all regulators. Develop criteria and/or tool for assessing implementation level of WHO written standards (guidelines) into regulatory practice. 10

11 11

12 Outcomes of various consultations and implementation workshops Agreed Biosimilars should not be regulated under generic (chemical ) drugs regulations additional considerations essential Agreed Head to head comparability exercise of quality, safety and efficacy is essential for a product to be considered to be a biosimilar (SBP). However, once licensed, a biosimilar has its own life cycle. If major differences found in quality, nonclinical or clinical studies, a product should not be considered to be a Biosimilar. Other options for its further development and licensing need to be considered (eg a stand alone pathway) Such products should not be called similar 12

13 Outcomes of more recent implementation workshops Increasing alignment between jurisdictions: noted importance of WHO in furthering standardized global approach, a convergence, but many challenges Most biotherapeutics in developing countries licensed by a stand alone approach with reduced data package rather than strict comparability exercise Some countries have regulatory pathway for non-innovative biotherapeutic products but requirements generally unclear Comparability studies with RBP: concept not well understood and used Lack of expertise and capacity for evaluation of biotherapeutics at NRA 13

14 An important outcome of all implementation workshops Identification of some copy products licensed with insufficient or inappropriate data Some copy products already licensed as biogenerics, a term which should not be used since it suggests a generic pathway Lack of harmonization of regulatory oversight of biotechnology products in general (not just biosimilars) Sometimes a range of different products on the market in one jurisdiction 14

15 "Copy products" and related uncertainties Slight differences in products can have unintentional effects on clinical performance and safety Generally little known about the safety and efficacy of individual inappropriately licensed products since pharmacovigilance is weak in most countries concerned. Lack of terminology for products developed as copy products with only partial comparability to a reference has compounded the problem 15

16 New Document on Regulatory Risk Assessment for Biotherapeutics licensed with insufficient data Draft Guidelines consultation process - 1 st round of public consultation Feb-Mar 2015: 19 sets of comments received - 2 nd round of public consultation Dec Jan 2015: 31 sets of comments - Face-to-face meeting with regulators, manufacturers and other experts on April in Geneva The approach described in the draft Guidelines allows licensed products to remain on the market for a specified period During this time manufacturers are required to submit a Risk Management Plan and, subsequently, submit any missing quality, nonclinical or clinical data Intended to assist regulators to develop approaches to evaluate these already licensed products according to WHO guidelines or for phasing them out in a reasonable period of time. 16

17 Scope Regulatory expectations for biotherapeutic protein products, including those for in vivo diagnosis Regulatory expectations for copy products (non innovative products) Regulatory expectations for biosimilars Also, polysaccharide based medicines (heparins) 17

18 Timelines Timeline for completing a review and providing new data would depend on the risk assessment of each individual product. Products from manufacturers who did not submit a Risk Management Plan nor appropriate data, or submitted data which were considered insufficient to support continued licensing, would be removed from the market. A risk based approach protects the supply and authorization could be regularized following further regulatory evaluation 18

19 Stepwise Risk Based Assessment (1) Nature of product /potential alternatives licensed by experienced NRA Is the product manufactured and licensed in a country with NRA well experienced in evaluating biotherapeutics Extent to which the submission dossier meets WHO Recommendations and Guidelines Consequences of treating or not treating a disease (supply issue) What sort of disease -life threatening or not. Patient population paediatric, adult geriatric 19

20 Stepwise Risk Based Assessment (2) Seriousness of potential lack of product efficacy and potential safety issues, especially immunogenic effects. Pharmacovigilance - how effective in monitoring possible adverse reactions. Traceability issues. Expertise and capacity of NRA for licensing biotherapeutics. Possibility of regulatory evaluation support by experienced NRA (mentoring). Transparency- informing healthcare professionals of review process and timelines. 20

21 Comments from first round of public consultation More clarity on goal of the risk assessment exercise Terminology, especially - copy product. Possible alternatives might be non innovative products to distinguish biosimilars or inappropriately licensed product or licensed by inappropriate pathway or with insufficient data Examples given in text (eg to Health Canada s approach to heparins) could be broadened to include others (eg ANVISA s approach to heparins) Add risk of switching between therapeutic alternatives 21

22 Comments from second round of public 1. Title of document consultation This document does not provide scientific principles, so it may be better to state it in the title. 2. Scope Since the document covers innovative and copy products including biosimilars, more details on following products are requested as provided for copy products: a. RRA for originator/innovator products b. BTP for prevention c. Proteins for diagnosis and engineered moieties d. Details for pegylated and conjugated products e. Polysacharide-based medicines f. Cell therapy product 22

23 Comments from second round of public consultation 3. Terminology a. Copy product b. Non-innovator product c. Non-comparable product 4. Nonclinical and clinical evaluation It is emphasized that the nonclinical and clinical studies are key components for RA Multiple interpretations of the EMA s simplified approaches 5. Clarify the stepwise approach 6. Need to clarify each step from initial assessment to decision 23

24 Plan for WHO consultations in April 2015, in Geneva: Review of SBP guidelines: to identify issues to be amended/ updated: April Regulatory Risk Assessment of BTP: April Development of new and replacement of existing reference preparations (measurement standards) Regional workshops and support to several networks of regulators and manufacturers from April to Dec 2015 Workshop in American region June 2015: subject of further discussion Training materials, case studies, Q&A Other activities in response to the requests from WHO member states (pre-icdra and ICDRA) as stated in ICDRA recommendations 24

25 Questions for regulators and manufacturers in Mexico What do you consider to be the most difficult problem in applying, in your country, the stepwise risk based approach set out in the WHO document? How would you deal with a lack of NRA experience / capacity in evaluating biotherapeutic products? What would you propose as the most needed assistance from WHO in this context? 25

26 Thank you Further information and contact Biological standardization website: Contact details: Dr Ivana Knezevic ( 26

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