Shoot for Share! From Vial to Pre-filled Syringe

Transcription

1 WEBINAR June 25, 2009 Shoot for Share! From Vial to Pre-filled Syringe SPEAKER: Raul Soikes Senior Director, Program Management Baxter BioPharma Solutions 1

2 Today we will address: Why? What? Value proposition of moving to a pre-filled syringe Regulatory plan to enable move How? Process to move from vial (liquid or lyophilized) to syringe Who? CMO qualifications 2

3 WHY? VALUE PROPOSITION 3

4 Why? Market driven Line extension Life cycle management New presentation New administration route Latest Technology / Market edge Competition Pharmacoeconomics Customer driven Safety- fewer manipulations Accuracy- Dose delivered Quality of life- self administered Product driven Manufacturing Less API waste increased units filled increased revenue 4

5 Why? - Strategies to Improve the Value of a Biologic Molecule Due to the inherent challenges of biologics, enhancements are difficult yet the market rewards improvements Short-Term Strategy (6-18 Months) Attach a safety device to a pre-filled syringe Kit vials with a ready to use diluent syringe Move from a stable liquid vial to a pre-filled syringe Mid-Term Strategy (18-36 Months) Move from a vial or syringe into an autoinjector or cartridge Reformulate lyophilized vials into a liquid vial or pre-filled syringe Long-Term Strategy (>36 Months) Develop sustained release formulation Develop alternate route formulation Short and Mid -Term strategies can help to differentiate a biologic molecule Enhanced Packaging and Reformulation Give New Life to Biologics, Tom Polen, BioPharm International, October

6 Why? - Mid-Term Strategy Teva Pharmaceutical Industries Ltd changes the presentation of Copaxone from a dry vial preparation to a pre-filled syringe Unit data in (M) Millions of units 2003, patients had all but completely switched to PFS formulation Copaxone PFS Copaxone Lyo Vial Copaxone PFS achieved rapid uptake in the US, with 64% of patients switching within the first three months from the dry vial formulation to the new formulation, and the remainder had switched within six months of launch. Dry vial to Syringe rapid uptake 64% switched in 3 months, remainder in 6 months after launch Source: IMS Health 6

7 Why? - Mid-Term Strategy The Copaxone reformulation was priced at a premium as compared to the original formulation. In 2002 the premium started at 5%, however, by 2005 the price premium was 48.6%. $60.00 $50.00 $40.00 $30.00 Unit pricing of Copaxone in $ USD 2002 Pricing - $26.02 Vial vs. $28.04 for PFS Pricing - $25.51 Vial vs. $37.88 for PFS $ $10.00 $ Copaxone PreFilled Syringe Copaxone Lyo Vial For patients, the switch reduced average preparation time from 235 seconds (reconstituted Copaxone) to 38 seconds, saving more than 20 hours over the course of a year. Premium pricing - from 5% in 2002 to 49% by Preparation time reduced from 4 minutes to 38 seconds. Source: IMS Health 7

8 Why? - Mid-Term Strategy Amgen, Inc. changed the presentation of Enbrel from a dry vial preparation to a pre-filled syringe. To further differentiate and add value, in 2006 Enbrel launched in an auto injector. Enbrel Presentation Mix Enbrel WW Sales 14,000 5,000 Units (Thousands) 12,000 10,000 8,000 6,000 4,000 2, Vial (Kit) Syringe SureClick $ (Millions) 4,000 3,000 2,000 1, Sales ROW Sales US Sales Japan Overall Enbrel unit sales have remained stable, however revenue has increased due to the price premium the PFS and SureClick format. Revenue increased due to price premium; unit sales stable IMS Data 8

9 Why? - Selecting a Drug Delivery Type Doctors top factors : minimal side effects, patients ease of use, satisfaction, convenience, comfort. Three of five factors (underlined) impacted by product presentation. Minimal side effects 9% 15% 16% 11% Easy for patient to use 16% 12% 6% 9% Patient satisfaction 14% 13% 6% 11% Patient convenience 11% 12% Patient comfort 9% 13% 7% 8% 9% Dose accuracy 6% 9% 6% 11% 8% Rapid onset of action 7% 6% 6% 8% 7% Low cost 5% 5% 9% 9% 5% Bioavailability 8% 3% 4% 6% 3% Low frequency of dosing 3% 6% 7% 5% Speedy recovery 6% 2% 1% 1% 3% Requested by patient 2% 3% 2% 5% 5% Higher drug absorption 2% 3% 2% 4% 4% Easy for doctor to administer 1% 2% 2% 3% 2% Formulary tiering of product with the delivery form 1% 1% 1% 1% 3% Ease of cost reimbursement 2% 1% 4% Well designed device 3% 3% Question: In order of importance, please select the top 5 factors that you consider when selecting a drug delivery type? (1= most important, 2=2 nd most important, 3=3 rd most important, etc.) US Drug Delivery - Frost & Sullivan

10 Why? Selecting Drug Delivery Type - Physician Ease of self administering is the top reason for selecting a device type. Has Reusable Component 1% 3% 6% 6% 7% Complexity of device 12% 16% 14% 17% 11% Does not require power source 2% 5% 4% 5% 8% Published Clinical Data 15% 13% 9% 11% 9% Appealing appearance of device 1% 2% 1% 2% Formulary Tiering of Product 2% 3% 7% 7% Cost to patient 9% 13% 17% 16% 20% Requested by patient 6% 6% 7% 13% Specific adverse side effects to device 2% 12% 14% 12% Size of device 3% 9% 7% 6% 3% Easy to self administer 46% 19% 15% 0% 20% 30% 40% 50% 60% 70% Rank 1 Rank 2 Rank 3 Rank 4 Rank 5 Question: For Device Driven Drug Delivery (Examples: Inhaler, Autoinjector), please select the top 5 factors that most influence your decision to prescribe the product to your patients? US Drug Delivery - Frost & Sullivan

11 Why? Device Drug Delivery Method - Patient Top Factors are ease of self administration (37%) followed by Physician s recommendation (24%) 100% 90% 4% 80% 70% 60% 50% 40% 30% 20% 0% 14% 26% 37% Easy to self administer 13% 12% 11% 9% 7% 6% 3% 6% Size of Device 12% 16% 24% 13% 13% 12% 14% Specific Recommended Out of adverse side by Pocket Cost effects Physician 11% 12% 9% 12% 8% 9% 8% 7% 9% 6% 7% 13% 8% 4% 7% 5% 2% 3% 1% 3% 4% 3% 5% 3% 2% 3% Appealing appearance Published Clinical data Does not Complexity of require device batteries / pow er source Need to refrigerate drug portion Reusable / Environmentally Friendly Rank 1 Rank 2 Rank 3 Rank 4 Rank 5 Question: For Device Driven Drug Delivery what are the top 5 factors that you consider? (1=most important, 2=2 nd most important, 3=3 rd most important, etc.) US Drug Delivery - Frost & Sullivan

12 Why? Device Drug Delivery Method Patient Ease of self administering is the most important factor for selecting drug delivery type followed by convenience. 100% 90% 80% 8% 70% 13% 14% 60% 50% 40% 30% 20% 0% 17% 20% 26% Easy to self administer 9% 8% 7% Rapid Onset of Drug Action 8% 7% 5% 2% Low frequency of dosing 14% 12% 16% 11% 8% 15% 13% 15% 13% 15% 12% 9% 8% 11% Minimal Side effects No Discomfort / Pain Low Cost / Insurance Co-Pay 7% 4% 3% 3% 2% Likelihood of Reimbursement 13% 15% 20% 17% Very Convenient 3% 3% 4% 1% Well designed device 14% 6% 4% 8% My doctor prefers it 1% Must be administered by another person Rank 1 Rank 2 Rank 3 Rank 4 Rank 5 Question: In order of importance, please select the top 5 factors that you consider when selecting a drug delivery type? (1= most important, 2=2 nd most important, 3=3 rd most important, etc.) US Drug Delivery - Frost & Sullivan

13 WHAT? REGULATORY STRATEGY 13

14 What? Define Regulatory Strategy Regulatory considerations Major change to regulatory market approval... Manageable 14

15 What? Evaluate & compare the proposed syringe system to approved materials Protects the drug product? Introduce a new material? Need drug product formulation changes? Define Regulatory Strategy Regulatory considerations Major change to regulatory market approval... Manageable 15

16 What? Evaluate & compare the proposed syringe system to approved materials Protects the drug product? Introduce a new material? Need drug product formulation changes? Define Regulatory Strategy Need supporting clinical data? Safety or efficacy effect? New indication? New route of administration? Regulatory considerations Major change to regulatory market approval... Manageable 16

17 What? Evaluate & compare the proposed syringe system to approved materials Protects the drug product? Introduce a new material? Need drug product formulation changes? Define Regulatory Strategy Need supporting clinical data? Safety or efficacy effect? New indication? New route of administration? Perform Stability protocol - New Dosage Form Define stability requirements Execute stability protocol Analyze stability data Regulatory considerations Major change to regulatory market approval... Manageable 17

18 HOW? VIAL TO SYRINGE 18

19 How? REGULATORY STRATEGY Formulation Study Stability Study Clinical Study Filing 19

20 How? REGULATORY STRATEGY Formulation Study Stability Study Clinical Study Filing Liquid Lyophilized 20

21 How? REGULATORY STRATEGY Formulation Study Stability Study Clinical Study Filing Liquid Container/ Closure Lyophilized Extractables/ Leachables Accelerated Long Term 21

22 How? REGULATORY STRATEGY Formulation Study Stability Study Clinical Study Filing Liquid Container/ Closure Indication Lyophilized Extractables/ Leachables Administration route Accelerated Degradation profile Long Term Impurity profile 22

23 How? REGULATORY STRATEGY Formulation Study Stability Study Clinical Study Filing Liquid Container/ Closure Indication Formulation Lyophilized Extractables/ Leachables Administration route Container/ Closure Accelerated Degradation profile Storage Conditions Long Term Impurity profile Clinical Data 23

24 How? Pre-filled syringe filing details: Attribute* Move From Liquid Vial Move From Lyo Vial Container/Closure Similar? Leverage previous extractable/leachable studies. Functionality: siliconization may be needed. Formulation No change. Change from freeze-dried powder to liquid. Storage Conditions No change. Stability Study: 3 months comparative accelerated and long term of at least 1 batch (3 may be required). Clinical Study: If degradation profile or impurities profile change. May change Stability Study: 3 months comparative accelerated and long term data on 3 batches. Clinical Study: If degradation profile or impurities profile change. * Indication and Administration Route changes need clinical data 24

25 WHO? CMO REQUIREMENTS 25

26 Who? 26

27 Who? Choosing your CMO... Experience Capacity CMO of Choice Capability Qualification 27

28 Experience Regulatory Global and local compliance Audit history Regulatory review and approval process Technical Product Scope Seasonal Campaign Market/Industry Presence Experience Regulatory Technical Product type Product Type Small Molecules Biologicals 28

29 Capability Resources Part of global company Redundancy Multidisciplinary Physical Facility Aseptic Formulation Cold Chain Management Personnel Education Training Experience Capability Resources Physical Personnel 29

30 Qualification Good Manufacturing Practices Compliance Documentation Training Audit Company Policies Business Quality Regulatory Manufacturing Qualification GMP Policies Registration Registration National entities Regulatory Agencies 30

31 Capacity Equipment Preparation Formulation Fill, Lyophilization, & Cap Packaging Laboratory Environmental monitor Product specific test Compendial testing Foot Print Multiple lines Aseptic areas Waste treatment Supporting equipment and processes Capacity Equipment Laboratory Foot Print 31

32 Vial to Pre-filled Syringe: Why? Value Proposition Market differentiation, premium pricing Customer safety, accurate dosing, self-administration Product less API waste more units filled What? Regulatory Strategy Evaluate and compare syringe system to current presentation Need supporting clinical data? Perform stability study 32

33 Vial to Pre-filled Syringe: How? Vial to Syringe Container/Closure - extractables/leachables Formulation liquid to liquid? Lyo to liquid? Storage Conditions stability study, degradation profile Filing clinical study? Stability supporting data Who? CMO requirements Experience Capability Qualification Capacity 33

34 References 1 Moving to a Pre-Filled Syringe: Stability Considerations A Regulatory Perspective. Raenel Gibson, RAC Regulatory Affairs Manager, Baxter Pharmaceutical Solutions LLC. Presented at the PDA The Universe of Pre-filled Syringes and Injection Devices, San Diego, CA, October 6-7, Financial Model For Converting From a Vial To a Pre-filled Syringe. Michael Borlet, Director of Marketing, Baxter Pharmaceutical Solutions LLC. Presented at the PDA The Universe of Pre-filled Syringes and Injection Devices, San Diego, CA, October 6-7, Copaxone is a registered trademark of Teva Pharmaceutical Industries Ltd. Baxter is a trademark of Baxter International Inc. Enbrel is a trademark of Immunex Corporation PFS is a trademark of Pharmacia & Upjohn Company SureClick is a trademark of Amgen Inc. 34

35 Shoot for Share: From Vial to Pre-Filled Syringe Thank you for participating in this complimentary webinar. If you have further questions or would like to contact me: Raul Soikes, Senior Director, Program Management Baxter BioPharma Solutions Phone For information about Baxter BioPharma Solutions, please visit our website at 35