R&D Portfolio Planning In- and Out-licensing of Medicines

Transcription

1 R&D Portfolio Planning In- and Out-licensing of Medicines

2 Portfolio Planning Role of portfolio management Importance to Industry Tools, methods and models to make Go/No-Go decisions To help Prioritise

3 Portfolio management: Today, an important senior management function Rapidly changing technologies Shorter product life-cycles Heightened global competition A key part of business strategy for R&D spending for Resource alignment for efficiencies in business for survival

4 R&D Investments and Portfolio management R&D investments (products) are like financial investments (stocks) Maximising the value of the portfolio Hence product Return on Investment (ROI) A balanced portfolio A portfolio investment strategy aligned with: company s overall business strategy therapy area expertise perception in the marketplace future aspirations.

5 What is Portfolio management? Dynamic decision process List of active new and R&D products constantly updated & revised New projects evaluated, selected and prioritised Projects accelerated, killed or de-prioritised Resources allocated and re-allocate to active projects Decision process characterised by: Uncertain and changing information Dynamic opportunities Multiple goals and strategic considerations Interdependence amongst projects Multiple decision makers and locations.

6 Portfolio decision process Encompasses many and overlapping in-company decision-making processes: Periodic reviews of total portfolio (all projects; comparing between) Making Go / No-Go decisions on individual projects Developing new product strategy for the business Strategic resource allocation decisions

7 Why do it? Negative consequences if you do poor portfolio management or not at all. Strategic Projects not aligned with business strategy Deficient project selection Too many projects Low value projects Mediocre pipeline projects Too many extensions, modifications, short term projects No focus Reluctance to kill projects Too many projects, resources thinly spread The wrong projects Decisions based on politics, opinion, emotion, not facts and objective criteria

8 Eight reasons why portfolio management is critical to business. Financial Competitive positioning Resource allocation Strategic issues: Strategy begins when you start spending money Achieving better focus Right balance of projects Improved communication in organisation (vertically and horizontally) Providing objectivity in project selection

9 Methods used for portfolio management Financial Business Strategy Bubble diagrams or portfolio maps Scoring models Checklists Hybrids

10 Challenges in portfolio management Creating climate, culture and buy-in Better allocation of resources, project selection and balance Short-term / long-term balance Better input data & forecasting estimates Better strategy portfolio linkage Better balance and resource allocations across business units, divisions and technologies Better balance across functions and level of involvement More credible financial metrics and tools

11 Benefits expected Common basis for discussion, discipline and consistency Focus on major projects, breakthrough projects Better strategic fit of portfolio Balances short and long-term goals Helps to concentrate on fewer more worthwhile projects Achieves improved times to market Unifies support and creates better buy-in Improves strategic planning

12 Acknowledgement Portfolio Management for New Product Development: Results of an industry* practices study. *multiple industries. Cooper R.G., Edgett S.j. and Kleinschmidt E.J. R&D Management (Industrial Research Inst Inc.) 2001: Vol.31(4);

13 Licensing and Medical Due Diligence In- and Out-licensing of medicines

14 In-Licensing Opportunity In-licensing increasingly important to fill product gaps In-licensed products make up 25% of total revenue of top 20 companies Licensing deals increasingly significant over last years Pfizer and GSK are most active in-licensees In 2002 Pfizer generated 53% revenue from in-licensed products, inc. Lipitor, the world s best selling medicine. In 2015 AstraZeneca had 20% of sales from Crestor (rosuvastatin), the company s #1 product.

15 Commercial potential of in-licensed product Over 3-5 years following launch average Rate of Return for inlicensed product over in-house product: 8.2% for in-licensed Phase III product 4.0% for in-licensed Phase I/II product 3.9% for in-licensed pre-clinical product And 2.3% for in-house product Mercer Management Consulting

16 Commercial potential for In-licensing opportunity Over 20-year period, situation is different: Gross Margin Profit: In-House product 42% Phase III purchase 24% In-House products eventually produce higher margins and make business more profitable as have no royalties to pay but need to wait 14 years for this to happen! (which means business has cash tied up for years and is not liked by investors in-licensed products generate more case in short-term.) Mercer Management Consultancy

17 Reasons for Licensing Agreements In-licensing To fill a pipeline gap To further enhance therapeutic focus As part of a strategic pre-launch plan To leverage expertise Out-licensing/divestment Lack of strategic fit or expertise A condition of Merger & Acquisition activity To fully realise commercial potential

18 Types of agreement Science agreements Research collaborations Contract research Co-development Marketing agreements Co-marketing Co-promotion Product Fostering Product Acquisition & Licensing Corporate Alliances Joint Ventures Mergers and Acquisitions

19 Marketing Agreements Product Acquisition Where a company 'purchases' an existing product licence from another company, obtaining the right to market an existing fully developed product Advantages Can fuel rapid growth by buying turnover Usually gain access to trade name Disadvantages Usually only available for smaller & older products locally Acquisition of major brands as a result of M&A usually extremely expensive

20 Factors to consider for in-licensing target Portfolio fit Company Strength Corporate equity & competency Therapeutic expertise Available resource Market Attractiveness Level of unmet medical need Level of generic activity Price Market growth potential Competitive Intensity (number, barrier to entry, promotional noise) Long term profitability Agreements European, Global Short, medium or long-term Marketing agreement or other

21 Evaluating Potential Sales Forecasts Factors to Consider Market Size and Structure Size, growth and dynamic component (opportunities) Segmentation Persistence Competitive Environment Competitive Intensity (number, barrier to entry, promotional noise) Competitors lifecycle stage Level of ownership e.g. asthma Recent launch successes and response to innovation Product Potential Level of unmet need in the market Existing products, competitors and Products in development Target profile and likelihood of realisation Promotional Strategy Segmentation, Positioning, Targeting Promotional spend and projected share of voice Lifecycle stage and patent life

22 Role of medical / medicines development in licensing and due diligence process

23 Overview of the Pharmaceutical Licensing Process

24 Review Development Programmes Manufacturing Pre-clinical Results Clinical Results Product Safety Compliance with Regulations Regulatory Strategy and Contacts Publications

25 Medical Due Diligence Data Room Manufacturing Investigative product Manufacturing Finished Product Pharmacology Toxicology Pharmacokinetics Clinical Trials Drug Safety Regulatory Commercial analysis Patents Publications and Media

26 Manufacturing Investigational Product supply of substance on hand for future research (GMP) cost of goods, bulk drug and final formulation final synthetic route, purity (impurity profile) and yields of steps enantiomers resolved scale-up and largest batches made currently any potential difficulties sourcing raw materials (see patents) stability of final products acceptable, reports available route of synthesis is optimized with sufficient due diligence cost of supply of compound for R&D and its implications for future pricing specifications and analytical procedures identification of routes used for different toxicology studies

27 Clinical Trials 1 Obtain documentation from all studies - conducted / ongoing / planned Are protocols applicable for WW regulatory agencies Trials completed / ongoing: has GCP been followed? Numbers of patients studied, ethnic group and/or sub-groups as appropriate Reports outstanding with dates and reports completed Reports in English, date and time for translation Mechanism from animal models consistent with data from humans Dose ranging study(ies) appropriate for dose used in clinic Human pharmacology including PK / metabolism

28 Clinical Trials 2 Clinical investigation QA of studies already conducted Data handling / transferability to company Meet / interview clinical investigators Ethical considerations Identification of future studies required for registration and commercial purposes, along with approximate dates for completion and reporting Estimate of development time-line (including both pre-clinical and clinical) Outline plan with timings

29 Drug Safety Which company holds recognised safety database (if more than one company, how will differences in details be minimised) Responsibility for reporting Responsibility for follow-up Responsibility for scanning literature Reporting responsibilities to Agencies (who is responsible in which country) Preparation of Periodic Safety Update Reports Company core data sheet (CCDS) maintenance Responding to safety related or product-related Regulatory enquiries Handling of product complaints Who are contacts in each company who is QPPV Signatories (one for each company?)

30 Regulatory General compliance Interactions with Regulatory Authorities documentation Check documentation for acceptability to authorities Are there any deviations to opinions obtained Are there regulatory / registrations / or compliance clauses that require review by legal Strategy and timelines is this consistent with clinical development plans