1. Career Opportunities for PharmDs in the Pharmaceutical Industry: Research & Development

2. Outline Describe the functions and responsibilities performed by various departments within pharmaceutical industry R&D. Highlight career opportunities within these departments for which individuals with a PharmD degree are well-suited.

3. Goal of Pharmaceutical R&D The underlying goal of pharmaceutical R&D is to create new knowledge, upon which a commercial product (and company) can be financially successful. Specific R&D goals vary for different pharmaceutical companies based on focus, size, history, risk tolerance, available resources, and expertise.

4. Major R&D Departments (bringing “molecules to market”) Chemistry PK Regulatory Literature Services Licensing Discovery Pharmacology Toxicology Clinical Development Pharmaceutics Project Management Pharmacy Market Planning

5. Steps From Concept to Marketed Product Identify the medical need.

6. Steps From Concept to Marketed Product Identify the medical need. Define the molecular target.

7. Steps From Concept to Marketed Product Identify the medical need. Define the molecular target. Design a series of new compounds that hit the target.

8. Steps From Concept to Marketed Product Identify the medical need. Define the molecular target. Design a series of new compounds that hit the target. Screen these compounds preclinically for optimum effects and potential safety and pharmacokinetic considerations. Select the best candidate based on desirable properties and conduct more definitive tests.

9. Steps From Concept to Marketed Product Identify the medical need. Define the molecular target. Design a series of new compounds that hit the target. Screen these compounds preclinically for optimum effects and potential safety and pharmacokinetic considerations. Select the best candidate based on desirable properties and conduct definitive tests. Formulate the compound.

10. Steps From Concept to Marketed Product Study the compound clinically through a series of clinical trials that establish the safety and efficacy for the intended population.

11. Pr. 994-13/-14 Study Design Baseline Evaluation S T R A T I F I C A T I O N + R A N D O M I Z A T I O N Regimen A*: Gemcitabine+ HDAC-I Regimen B*: Gemcitabine+ Placebo Follow-Up for 12 months after last patient enrolled *Administered as a 28-day course as long as medically appropriate Regimen A: Gemcitabine 1000 mg/m 2 IV on days 1, 8 and 15 of a 28-day cycle HDAC-I 6 mg/m 2 PO days 1-21 of a 28-day cycle Regimen B: Gemcitabine 1000 mg/m 2 IV on days 1, 8 and 15 of a 28-day cycle Placebo 6 mg/m 2 PO days 1-21 of a 28-day cycle Study Design

12. Steps From Concept to Marketed Product Study the compound clinically through a series of clinical trials that establish the safety and efficacy for the intended population. Satisfy Regulatory agencies that the compound is safe and effective for the intended purpose.

13. Steps From Concept to Marketed Product Study the compound clinically through a series of clinical trials that establish the safety and efficacy for the intended population. Satisfy Regulatory agencies that the compound is safe and effective for the intended purpose. Launch, promote, and sell the product.

14. Steps From Concept to Marketed Product Study the compound clinically through a series of clinical trials that establish the safety and efficacy for the intended population. Satisfy Regulatory agencies that the compound is safe and effective for the intended purpose. Launch, promote, and sell the product. Support the commercial success of the product with post-marketing studies.

15. Major R&D Departments (bringing “molecules to market”) Chemistry PK Regulatory Literature Services Licensing Discovery Pharmacology Toxicology Clinical Development Pharmaceutics Project Management Pharmacy Market Planning

16. Pharmacy Services in R&D Support R&D through participation on all project teams. Manage supplies and flow of NCE between Chemical Development, Pharmaceutics/ Formulation, and users (preclinical and clinical studies). Package, label, distribute, and maintain accountability for clinical trial materials. Common entry point for PharmDs with no industry experience.

17. Clinical Development Department Responsible for creating the optimal strategy to assess the clinical utility of the new compound. Responsible for planning and conducting a series of clinical protocols designed to assess the efficacy and safety of the new compound, with the goal of getting the compound registered.

18. Major Components of the Clinical Development Department Statistician Outcomes Research Clinician Data Services Medical Writer Clinical Research Associate

19. Conducting a Protocol (101) Clinician writes the protocol to accomplish specific scientific objectives. Clinician recruits Investigators (MDs) to conduct the study by treating their eligible patients according to the protocol and recording the study data. CRAs monitor the completeness and accuracy of the investigator’s data and bring the data in-house.

20. Conducting a Protocol (101) Data handling specialists enter the data and program the output. Statisticians analyze the data. Clinician interprets the data. Medical Writer summarizes the data. Clinician utilizes the new knowledge in the design of the next protocol.

21. Phases of Clinical Development Phase 1 protocols: –Objectives are to establish dose ranges, pharmacokinetics and safety/tolerance. –Conducted in normal volunteers or in patients with targeted indication

22. Phases of Clinical Development Phase 2 protocols: –Objectives are to assess the activity of the compound in patients with the target disease and to assess safety/tolerance.

23. Phases of Clinical Development Phase 3 protocols: –Objectives are to assess the efficacy and safety of the compound in patients with the target disease, relative to standard therapy.

24. Phases of Clinical Development Phase 4 (post-marketing) protocols: –Objectives are to further characterize the effects in subpopulations or with an emphasis on additional endpoints.

25. Job Description for Clinical Scientist “Responsible for developing new protocols, implementing, conducting, interpreting, and summarizing clinical studies. Presentation of plans and data internally and externally as appropriate.”

26. Job Description for Clinical Scientist Requirements cited from several current job postings: –“PharmD + minimum of 1 year (to 5 yrs) of related experience” –“Fellowship in Clinical Pharmacology is desired” –“Experience in academic and industry early phase clinical trial design conduct” –“Knowledge of [bone metabolism] desirable”

27. Ideal Candidate for Clinical Scientist Position (in Oncology) Formal training in a clinical discipline; knowledge of clinical labs, disease states, pharmacology, pharmacokinetics, … Specific knowledge of oncology disease states, treatments, complications, … Knowledge of clinical trial methodologies, statistics, endpoints, … Experience in industry-sponsored R&D.

28. Clinical Research Associate Responsible for ensuring the validity, accuracy, and completeness of clinical research records by monitoring clinical trial data. Responsible for ensuring that study conduct complies with required Regulatory procedures. Common entry point for PharmDs with limited industry experience.

29. Medical Writer Responsible for creating internal documents that summarize the results of clinical trials. Works with research projects from early studies through final FDA document preparation. A possible, but not common, entry point for PharmDs with limited industry experience.

30. Outcomes Research Scientist Responsible for developing protocols, implementing, conducting, interpreting, and summarizing clinical studies that focus on quality of life endpoints, clinical utility measures, pharmacoeconomic assessments, etc. Generally would require some specialized training (as per a Fellowship?)

31. Regulatory Affairs Department Responsible for managing the interface between the Sponsor and Regulatory Agencies (eg, FDA) so that the information gained through the R&D process meets the criteria for product registration. A possible, but not common, entry point for PharmDs with limited industry experience.

32. Additional Departments with Possible Career Opportunities for PharmDs No further degree or formal training program is required: –Clinical Quality Assurance Department –Safety Surveillance Department Possible, but not common, entry points for PharmDs with limited industry experience.

33. Additional Departments with Possible Career Opportunities for PharmDs Formal degree, training, or experience is usually required/preferred: –Pharmacokinetics & Drug Metabolism Department –Licensing & Acquisitions Department –Literature Services Department –Project Management –Market Planning Department

34. ‘So, how can I get a cushy R&D Job without having R&D Experience?”

35. 1.Be prepared to start with a job other than your ‘ideal’ job. Gain industry-based R&D experience, establish your reputation, look for internal or external opportunities.

36. ‘So, how can I get a cushy R&D Job without having R&D Experience?” 2.Initially seek employment with: a Contract Research Organization (CRO) that supports pharmaceutical-based R&D, or an academic center that has a strong clinical research component.

37. ‘So, how can I get a cushy R&D Job without having R&D Experience?” 3.Build your knowledge and experience level to match the desired attributes. Seminars or courses in clinical trial design, statistics, business, etc. Specialized disease knowledge Publish (something -- anything!) Research fellowship

38. Career Opportunities for PharmDs in the Pharmaceutical Industry: Research & Development