NPP / MR Practices for Early Stage Therapeutics

Transcription

1 IBI 18 - Aiming to become Top Pharmaceutical Company NPP / MR Practices for Early Stage Therapeutics prepared exclusively for: 2017 Chugai Pharma USA Inc / Dean Slack Dr. Dean Slack, MBA Head, Business Assessment & (New Products) Planning Translational Clinical Research Division Chugai Pharma USA, Inc.

2 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ Outline I. Quick Introduction to Chugai II. Why Earlier Matters to PMRG Members III. BA&P (MR) as a Member of R&D IV. Actions (Questions) to Ask Yourself 2017 Chugai Pharma USA Inc / Dean Slack

3 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ Outline I. Quick Introduction to Chugai II. Why Earlier Matters to PMRG Members III. BA&P (MR) as a Member of R&D IV. Actions (Questions) to Ask Yourself 2017 Chugai Pharma USA Inc / Dean Slack

4 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ I. Quick Introduction to Chugai Chugai 2017 Chugai Pharma USA Inc / Dean Slack

5 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ I. Quick Introduction to Chugai Chugai Pharma USA Inc / Dean Slack dyb0

6 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ I. Quick Introduction to Chugai -orhttps:// dyb Chugai Pharma USA Inc / Dean Slack

7 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ I. Quick Introduction to Chugai Chugai Innovation Beyond Imagination Innovation All For The Patients Corporate Vision: A company that focuses on first-in-class [1] and best-in-class [2] products and services, and continuously provides new solutions to patients and medical communities around the world. Notes: 1. An original drug that is highly novel and useful, and will significantly change the therapeutic system. 2. A drug that offers clear advantages over other existing drugs in the same category, such as those with the same molecular target. Public Information. Source: Chugai 2016 Annual Report. p Chugai Pharma USA Inc / Dean Slack

8 ACCEL 15 Chugai Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ I. Quick Introduction to Chugai 1. Growth Engine is Innovation 2. Unique Business Model Built Over the Past 15 Years Strategic Alliance w/ Roche Autonomous Publicly Traded Company Member of Roche Group Chugai Revenue, Operating Profit & Market Cap ~Tripled ~5K Employees (~7K Consolidated) In JPY Billion: Revenue 491.8; Core Operating Profit 80.6; [$4.52 B; $0.74 B ( JPY/USD in 2016)] 3. Global Development Focuses on Early Proof of Concept Public Information. Source: Chugai 2016 Annual Report, p. 4, 5 & 23 & Mid-term Business Plan IBI Results and 2017 Outlook 2/1/2017. p Chugai Pharma USA Inc / Dean Slack

9 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ ACCEL 15 I. Quick Introduction to Chugai Chugai Very Robust Pipeline: P1 through Filing 2017 Chugai Pharma USA Inc / Dean Slack

10 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ I. Quick Introduction to Chugai Chugai Very Robust Pipeline: Pre- P1 (sorry, can not show) (s) 2017 Chugai Pharma USA Inc / Dean Slack

11 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ Outline I. Quick Introduction to Chugai II. Why Earlier Matters to PMRG Members III. BA&P (MR) as a Member of R&D IV. Actions (Questions) to Ask Yourself 2017 Chugai Pharma USA Inc / Dean Slack

12 ACCEL 15 II. Why Earlier Matters Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ Eroom's Law (hypothesis) in Pharmaceutical R&D Scannell, et. al., (2012) suggest 4 primary causes: 1. The better than the Beatles problem. 2. The cautious regulator problem. 3. The throw money at it tendency. 4. The basic research-brute force bias Chugai Pharma USA Inc / Dean Slack Scannell, J., Blanckley, A., Boldon, H. & Warrington, B., Diagnosing the decline in pharmaceutical R&D efficiency Nature Reviews Drug Discovery 11, (March 2012) doi: /nrd3681

13 ACCEL 15 II. Why Earlier Matters Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ Tufts Study (2016, Peer Reviewed) The estimated average out-of-pocket cost per approved new compound is $1.4 B. Capitalizing out-of-pocket costs to the point of marketing approval at a real discount rate of 10.5% yields a total pre-approval cost estimate of $2.6 B. Capitalized costs were shown to have increased at an annual rate of 8.5% above general price inflation. Adding an estimate of post-approval R&D costs increases the cost estimate to $2.9 B. *Costs are in 2013 dollars and rounded to 1/10 th B Chugai Pharma USA Inc / Dean Slack J Health Econ May;47: doi: /j.jhealeco Epub 2016 Feb 12. Innovation in the pharmaceutical industry: New estimates of R&D costs. DiMasi JA 1, Grabowski HG 2, Hansen RW 3

14 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ PI PIII PII Rx Industry: Years Until Marketed $2.6 B Investment <12% of P1 Reach Approval Biopharmaceuticals in Perspective, Spring 2016, PhRMA with Tufts (2016) 2017 Chugai Pharma USA Inc / Dean Slack P.13

15 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ Time (X-Axis) Not Shown in Proportion; Overlay of PLC & LOE Added to 2017 Chugai Pharma USA Inc / Dean Slack PhRMA P.14 Chugai s BA&P is an Innovative Deployment Typical BA&P MR (R&D) New Products & BD MR Typical Pre-Launch & In-line MR LO E

16 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ Outline I. Quick Introduction to Chugai II. Why Earlier Matters to PMRG Members III. BA&P (MR) as a Member of R&D IV. Actions (Questions) to Ask Yourself 2017 Chugai Pharma USA Inc / Dean Slack

17 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ III. BA&P as a Member of R&D Reports to CMO (M=Medical, Marketing) Member of Translational Clinical Science Science & Strategy Within Research Division of R&D (R D) Senior Commercial Person: New Product Planning & MR Drugs are Projects, not yet Products Biologic & Pharmaceutical Broad Portfolio; Innovation-based, not TA-based 2017 Chugai Pharma USA Inc / Dean Slack

18 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ III. BA&P as a Member of R&D Grounded in the Usual MR Tenet's: Objectivity Voice of Customer Double Blinded Collaboration Profession s Practices 1. Desk Research Secondary is the Main Course Primary is a bit Different Insight Generation Unique 2. KOL + High Treaters 3. Qualitative Market Research 4. UMN Findings of & Understanding 2017 Chugai Pharma USA Inc / Dean Slack

19 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ Outline I. Quick Introduction to Chugai II. Why Earlier Matters to PMRG Members III. BA&P (MR) as a Member of R&D IV. Actions (Questions) to Ask Yourself 2017 Chugai Pharma USA Inc / Dean Slack

20 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ IV. Conclusion/Actions IV. Actions (Questions) to Ask Yourself 5 Questions to Ask Yourself/Client: 1. Is your foundational work (your DMR/secondary analysis) solid? Are you going beyond the audits? Beyond syndicated? Internally vetted with MD s, Research Scientists? Clinical? You should be going well beyond. Audits, etc. are a very small part of it. 2. What is your relationship with R&D? 3. How early in the portfolio are you working? Are you working early enough to have an impact? 4. Do you, your team, clients, vendors have The right skills? The right prioritization and availability for foundational work? Management/Client buy-in? 5. Is your ratio of results (Secondary-to-Primary) suitable? Not ratio of spending Not ratio of reporting 2017 Chugai Pharma USA Inc / Dean Slack

21 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ Outline I. Quick Introduction to Chugai Innovation Beyond Imagination II. Why Earlier Matters to PMRG Members We must all start earlier to increase impact III. BA&P (MR) as a Member of R&D An Innovative Approach IV. Actions (Questions) to Ask Yourself For Consideration 2017 Chugai Pharma USA Inc / Dean Slack

22 ACCEL 15 Aiming to become Top Pharmaceutical Company ~Innovation all for the patients~ Thank you! NPP / MR Practices for Early Stage Therapeutics Dean Slack Part 2: Karen Tibbals 2017 Chugai Pharma USA Inc / Dean Slack

23 Market Research for Early Stage Drugs on a Budget Karen Tibbals KarenJTibbals LLC 22

24 Problem with early stage drugs Bullet 1 Bullet 2 Bullet 3 23

25 Human Problems 24

26 The usual solution 25

27 Contents Epidemiology Current practice patterns Competitive landscape Unmet need Ratings of current products Assessment of products in development Anticipated prescribing Forecast 26

28 What s the problem with that?

29 HOW DO WE DO IT?

30 The research process

31 The Sources

32 TOPICS

33 The basics Syndicated reports Review articles in the literature Merck Manual Patient organizations

34 Forecasting the competitive pipeline

35 Create a timeline Phase 3 product Phase 2 product Generic existing product Your product

36 Epidemiology

37 Epidemiology

38 Market sizing example P.37 14% of women of childbearing years Complication rate: 25% Unknown % treated Market size ranges

39 Forecast the patient trends by demos or subgroups Patient Forecast by Gender 14,000 12,000 10,000 8,000 6,000 Women Men 4,000 2,

40 Identify Market Needs

41 Endpoint Questions to Investigate

42 Develop Draft Hypothetical Target Product Profiles P.41 Minimum Mode of Action XXX XXX Target Patients Reach Dose and Administration Efficacy Safety/tolerability

43 Reimbursement

44 Develop working hypotheses Market dynamics Patients Needs Endpoints Unknowns?s

45 Primary research

46 CASE STUDY

47 Case study from the headlines

48 Publically Available Data

49 The basics

50 Market needs Many different orphan diseases Other genetic types Measures of effect on muscle strength Further improvements in efficacy

51 Current website A survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients Treats mutations in chromosome 5q that lead to SMN protein deficiency Some patients achieved milestones such as ability to sit unassisted, stand, or walk when they would otherwise be unexpected to do so, maintained milestones at ages when they would be expected to be lost, and survived to ages unexpected considering the number of SMN2 gene copies of patients enrolled in the studies.

52 Comparison of endpoints from clinical trials Neurological Motor Function Respiratory Scale Spinraza CK RO HINE MFM-32 6MWT HFMS-E RULM HHD TUG CAMP CHOP INTEND FVC/FEV MEP SNIP Serious Resp Events

53 Timeline CK RO Your product Generic Spinraza

54 Patient involvement

55 Reimbursement

56 Lessons learned Early stage products are hard to do Have the greatest potential for impacting the product profile It is possible Not everyone can do it Have to get your future mind set on Do a lit search before primary research Be creative!

57 QUESTIONS?

58 57 THANK YOU!