Novartis Pharma AG Global Clinical Trials Innovation Summit

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1 Novartis Pharma AG Global Clinical Trials Innovation Summit Cornelis Winnips MD, Global Program Medical Director Berlin, October 20, 2016

2 Novartis is one of the world s largest pharmaceutical companies... Sales By Region 2015 USD Billion Latin America Net Sales Net Income Asia/Africa/ Australasia 21% 9% 33% Europe People (FTEs) 118,700 37% US Clinical Trials Innovation Summit, Berlin , C.Winnips 2

3 Our mission and vision for sustainable growth Mission Discover new ways to improve and extend people s lives Using science-based innovation Delivering breakthrough treatments to as many people as possible Aim to provide shareholder return Vision Be a trusted leader in changing the practice of medicine Clinical Trials Innovation Summit, Berlin , C.Winnips 3

4 The Macro-Environment Opportunity and Change Business 4 Use Only

5 A golden age for medical science Accelerating Innovation +64% The US approved an average of 36 new drug molecules a year in , vs 22 in Clinical Trials Innovation Summit, Berlin , C.Winnips 5

6 Demographic trends are changing healthcare Growing Populations +1Bn by 2030 About 8.5 billion people are expected to inhabit the planet by the year 2030 Clinical Trials Innovation Summit, Berlin , C.Winnips 6

7 Demographic trends are changing healthcare Rapid Aging +500m over by About 1.4 billion people worldwide are projected to be 60 or older by the year 2030, fueling a rise in chronic illness Clinical Trials Innovation Summit, Berlin , C.Winnips 7

8 Putting healthcare systems under pressure Increasing Healthcare Demand 13T by 2025 If growth trends continue, global healthcare outlays could rise 70% by 2025 to USD 13 trillion Clinical Trials Innovation Summit, Berlin , C.Winnips 8

9 We need to Reimagine Medicine Use science- How we fight disease based innovation How we deliver treatments How we operate Clinical Trials Innovation Summit, Berlin , C.Winnips 9

10 ...and deliver better health outcomes for patients Outcome-based pricing Real-world evidence of effectiveness Data and analytics Digital health, beyond the pill Health system capacity building Clinical Trials Innovation Summit, Berlin , C.Winnips 10

11 Clinical Trial Environment Some Basics

12 Clinical Trials General Legal/Moral Framework Objectivity Good Clinical Practice! Public Interest Patient Interest Documentation 12 Clinical Trials Innovation Summit, Berlin , C.Winnips

13 Guiding Ideas and Principles Protection of humans is the utmost objective Guidelines are not laws but sensible help to navigate Economy: use first the most simple and quickest test system Results must be reproducable!!! Statistically significant is not identical to clinically relevant (medically meaningful) Be aware of the limitations preclinical and surrogate markers have for the relevance for humans and the target disease The dose plays the game (dosing interval; treatment duration; dose-response curve is linear, sigmoid, U-shaped, bellshaped) 13 Clinical Trials Innovation Summit, Berlin , C.Winnips

14 Drug Development Regulatory Dimensions 14 Clinical Trials Innovation Summit, Berlin , C.Winnips

15 15 Example of Today s Clinical and Commercial Challenges Increasing development hurdles?

16 Setting the scene prepare for access Product X, demonstrated efficacy in phase 3 trials in patients with moderate-to-severe disease compared with placebo or a current standard of care. Registration The global clinical program for registration included 6 placebo and active controlled studies in patients with moderate to severe disease. Label at submission was Second line : Product X is indicated for the treatment of moderate to severe disease in adults who failed to respond to, or who have a contraindication to, or are intolerant to other systemic therapies. Taken together, clinically highly relevant efficacy has been shown in patients with moderate to severe disease, including systemic treatment naïve patients as well as those previously exposed to systemic therapies including biologic therapies. Therefore, the EMEA agreed to broaden the initially proposed indication to: Product X is indicated for the treatment of moderate to severe disease in adults who are candidates for systemic therapy* Clinical Trials Innovation Summit, Berlin , C.Winnips

17 Example prepare for access in Germany Access The Patient Access team has highlighted the need for active controlled H2H RCT. The H2H studies are gateway to access for many countries, including some of the top 10 countries. Ideally the H2H are superiority studies vs the best comparator (efficacy/price). Access in Germany Demonstrate considerable therapeutic improvement In accordance with AMNOG (German Access Law), the German team needed to demonstrate considerable therapeutic improvement with Product X relative to an appropriate comparator, in order to receive a sustainable reimbursement price The German team identified Product Y as their primary choice of comparator vs. Product X. (existing H2H data). However, Product Z, a recent entrant in the indication, and which was considered for a next H2H by global, was also regarded as a possible comparator Add to existing trial data To add to the existing trial data for Product X versus Product Y, Global also initiated a phase 3b study, CLEAR, to assess the efficacy and safety of ProductX vs. ProductZ However, the G-BA did not consider ProductsY or Z to be appropriate comparators in the indication. This was because ProductY was not as effective as other available treatments and ProductZ had not been utilized enough in the healthcare system ProductXY as G-BA appropriate comparator The G-BA identified ProductXY or Product XX as appropriate comparators for product X Conducting a head-to-head trial of ProductX versus ProductXY or Product XX in the approved/ targeted patient population would generate high quality evidence that would meet G-BA comparator specifications, but would be expensive and time-consuming Clinical Trials Innovation Summit, Berlin , C.Winnips

18 Impact on Planning and Project Selection

19 Developing a quantitative Target Product Profile (TPP) Objective: The main objective is to support decision making where the aim is to develop a product to meet a medical and market need Approach: Define base case and upside quantitative targets for key efficacy and key safety outcomes in the TPP. Identify the relevant evidence to assess these targets Use probabilities to quantify the current evidence in relation to the TPP targets. Based on a results, align on a common interpretation and a set of recommendations Challenges: Requires a team discussion and alignment Potentially guided by a Biostatistician Ensure consistency! 19 Clinical Trials Innovation Summit, Berlin , C.Winnips

20 Defining a e Target Product Profile (TPP) Case study - Product X: Define TPP and the context of the program Drug Indication Standard of care Base case Including minimal acceptable thresholds Upside case Including target thresholds Product X Acute Heart Failure Usual care + Placebo Dyspnea (VAS): difference over 5 days in CFB 468 mm*hr (4mm on average) Dyspnea (Likert): prop of patient with moderate to marked improvement : RR 1.3 CV death and HF hosp at day 60: HR 0.85 Days alive out of hospital: 1 day improvement (60 days) All cause Mortality: RR 1 Clinical blood pressure: 15% decrease Dyspnea (VAS) difference over 5 days in CFB 585 mm*hr (5mm on average) Dyspnea (Likert): prop of patient with moderate to marked improvement : RR 1.4 CV death and HF hosp at day 60: HR 0.75 Days alive out of hospital: 2 days improvement (60 days) All cause Mortality: RR 0.7 Clinical blood pressure: 5% decrease Thresholds must be justified e.g. having a target threshold differentiates the New drug (this could be a non-inferiority margin in some cases e.g. antibiotics) 20 Clinical Trials Innovation Summit, Berlin , C.Winnips

21 Quantify the Clinical Data Product X: Collect the key phase III data Product X Standard of care Threshold stat: Difference to SoC TPP Base TPP Upside Dyspnea - VAS 2756mm 2308mm 447.7mm (120, 775.4) 468mm 585mm Efficacy Dyspnea - Likert CV death and HF hosp 27% 26% RR 1.04 (0.86,1.26) 13.1% 12.9% HR 1.02 (0.74, 1.41) Science Days alive out of hospital 48.3 (# days) (-0.77,1.97) 1 2 Safety AE of special interest All cause Mortality Clinical blood pressure decrease 7.2% 11.2% RR 0.63 ( 0.43, 0.93) 18.8% 12.5 % -6.3% (-10.5, -2.1) RR 1 RR % -5% Include the primary efficacy and AE of special interest for the program. 21 Clinical Trials Innovation Summit, Berlin , C.Winnips

22 What goes into the Holistic Assessment Framework to assess a Project? Science Grid Project Attributes Includes Time to launch, NPV, enpv and ROCE (return on capital employed) based on team input Science Therapeutic Differentiation Strength of Evidence ROI Time to launch NPV - enpv - ROCE Technical & Market Risk Clinical Endpoints Regulatory Risk Market Access Based on the information given in the Therapeutic Fact-Sheet 22 Clinical Trials Innovation Summit, Berlin , C.Winnips Based on short rationale given in the Data Capture Sheet under the section Technical & Market Risk

23 A Template for Clinical quantitative Assessment Process Define Target Product Profile and collate the key clinical data Efficacy Safety Quantify the TPP Summarize clinical data Efficacy Base Nov drug Upside Standard of care Diff Quantitative assessment of the data Probabilities quantifying the current evidence Visual representation Qualitative assessment of the data Mirrors the prioritization framework Generic set of descriptions to assess data Criteria Efficacy Safety Favorable (5) Neutral (3) Unfavorable (1) Judgement of the data Efficacy (5) (4) (3) (2) (1) Safety (5) (4) (3) (2) (1) Safety Clinical Trials Innovation Summit, Berlin , C.Winnips

24 Conclusions The Aligned Clinical Development Strategy 1. Is adapted to today s environment Increasing competition for limited healthcare resources Need for more rigorous generation of evidence Focus on Patient value & outcome 2. Meets medical and business goals Reflected in the Target Product Profile Comparison with «gold standard» where possible 3. Integrates evidence in standard templates Provides a consistent picture of all relevant data of a given project at a given time point Leads disciplined approach to decision making based on the evaluation of the current evidence related to clinical development and commercial objectives Uncertainty assessed by using two targets (base and upside cases) 24 Clinical Trials Innovation Summit, Berlin , C.Winnips

25 Novartis Pharma AG Thank you

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