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Effective Strategies for Successful Global Development

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Learn how to leverage effective strategies for global drug development, including expedited regulatory pathways, personalized medicines and genomics. View the full presentation from PAREXEL Consulting experts.

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Effective Strategies for Successful Global Development

  1. 1. © 2018 PAREXEL INTERNATIONAL CORP. EFFECTIVE STRATEGIES FOR SUCCESSFUL GLOBAL DEVELOPMENT Sugato De Principal Consultant, Integrated Product Development, PAREXEL® Consulting April 17, 2018 PAREXEL KOREA SYMPOSIUM 2018
  2. 2. © 2018 PAREXEL INTERNATIONAL CORP. / 2 EFFECTIVE STRATEGIES FOR SUCCESSFUL GLOBAL DEVELOPMENT • Smarter, Faster Approvals: Navigating FDA’s Accelerated Approval Programs • New Frontiers In Personalized Medicine • Evolving Regulatory Paradigms for Digital Health OVERVIEW
  3. 3. © 2018 PAREXEL INTERNATIONAL CORP. / 3 SEOUL, SOUTH KOREA SMARTER, FASTER, APPROVALS
  4. 4. © 2018 PAREXEL INTERNATIONAL CORP. / 4 ARE ACCELERATED PATHWAYS A COMPETITIVE ADVANTAGE FOR US? FDA THINKS SO…. “In the US, the FDA uses expedited programs (fast track, priority review, accelerated approval, and breakthrough therapy) for drugs and biologics more than comparable drug and biologic regulators in other countries use theirs and, as a result, FDA is the first to approve a majority of novel drugs compared to our foreign counterparts.” -- ROBERT CALIFF, M.D., FDA COMMISSIONER, DECEMBER 13, 2016
  5. 5. © 2018 PAREXEL INTERNATIONAL CORP. / 5 PERCENT OF CDER’S NME/NBE APPROVALS USING ACCELERATED PATHWAYS (APPROVALS IN 2015-2017) 33% 45% 25% 18% 3% 0% 10% 20% 30% 40% 50% 60% Fast Track Orphan Drug Breakthrough Therapy Accelerated Approval QIDP ► Just over a third (36%) used multiple accelerated pathways ► 64% used at least one accelerated pathway
  6. 6. © 2018 PAREXEL INTERNATIONAL CORP. / 6 WHAT ARE ACCELERATED PATHWAYS FOR? • Intended for drugs, devices and biologics that address an unmet medical need in the treatment of a serious or life- threatening condition. • Ensure that innovative therapies for these conditions are approved and available to patients as soon as it can be concluded that the therapies’ benefits justify risk. • Allow earlier attention to medical products that have promise.
  7. 7. © 2018 PAREXEL INTERNATIONAL CORP. / 7 ACCELERATED PATHWAY EXAMPLES Fast track is a process design to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet clinical need. A process designed to expedite the development and review of drugs and devices which may demonstrate substantial improvement over available therapy. These regulations allow drugs that fill an unmet medical need to be based on a surrogate endpoint. Priority review designation means FDA’s goal is to take action on an application within 6 months.
  8. 8. © 2018 PAREXEL INTERNATIONAL CORP. / 8 THE FDA FAST TRACK PROGRAM • Introduced under FDA Modernization Act of 1997, which sought to streamline FDA procedures • FDA approval times were competitive with peer regulators, but US sought mechanism under which potential important breakthroughs could be designated early in development, permitting early/more intense FDA/sponsor interaction to streamline the development/ approval process for products beyond just AIDS or cancer • Designed to facilitate the development/expedite FDA review of new drugs intended to treat serious* or life-threatening conditions and that demonstrate the potential to address unmet medical needs * “. . . a disease or condition associated with morbidity that has substantial impact on day-to-day functioning. Short-lived and self-limiting morbidity will usually not be sufficient, but the morbidity need not be irreversible if it is persistent or recurrent. Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one.”
  9. 9. © 2018 PAREXEL INTERNATIONAL CORP. / 9  Perceived Benefit: • Value of the Fast Track Designation  Defined Benefits (in law and FDA Guidance): • Fast Track Product “could be eligible for priority review if supported by clinical data.” – 78% of approved FT products (2011-2016) ultimately gained priority status (85% in 2014-2016) vs. ~47% overall (60% in 2015/2016) – Of 105 P-rated NME/NBEs app’d 2011-2016, 41 were non-FT (although 30 were in 2014-2016) • Possibility of “rolling” NDA/BLA – Renaissance of interest? – About half of 2015/2016 app’d FT drugs had confirmed rolling NDA/BLAs vs. 29% in earlier PXL study (2011-2014) – 2016 rolling FT NDA/BLAs (3) had mean app’l time of 9.0 Months vs 10.1 months overall – 2015 rolling FT NDA/BLAs had mean app’l time of 9.4 Months vs 12.1 months for 2015 NDA/BLAs overall * – Biologics applicants appear to have greater interest • “Opportunities for frequent interactions” with FDA – Solid clinical development time advantage for FT drugs historically – Development time advantage for FT drugs in oncology, but BTDs minimizing BENEFITS OF FAST TRACK DESIGNATION * Excluding lengthy review times for Bridion and Addyi All data presented here is from public FDA databases.
  10. 10. © 2018 PAREXEL INTERNATIONAL CORP. / 10 BREAKTHROUGH VS. FAST TRACK * “One of the best things about BTD is that it drives patients to clinical trials.” – Leading cancer researcher, 2014. **All six BTD approvals (through 6/10/2014) are for products with “large price tags,” ranging from $41,000 to $300,000 for annual course of therapy (www.healthaffairs.org, May 15, 2014) Breakthrough Therapy • Highest order of FDA designation. • IND submission EOP2 Meeting • Substantial improvement over existing therapies for serious/LT • Transformational, not marginal. • Depends on both magnitude of effect on clinically meaningful endpoint (could be duration of effect) and importance of effect. • Preliminary clinical evidence of “treatment effect” (generally P1 or P2). • All FT benefits plus “intensive FDA guidance” on development program and senior-level CDER input. • Expedited NDA/BLA review (March 2015 MAPP). • Multi-disciplinary. Fast Track • IND submission Pre-NDA/BLA Meeting • Potential to address unmet medical need for serious/LT. • Theoretical rationale, mechanistic rationale (PC), NC activity. • Frequent interactions, rolling NDA, and priority review designation (none guaranteed).
  11. 11. © 2018 PAREXEL INTERNATIONAL CORP. / 11 BREAKTHROUGH DESIGNATIONS: WHAT PRODUCT TYPES ARE OBTAINING THEM? (BY CDER REVIEW DIVISION) Hematology, 18% Antiviral, 19% Oncology, 28% Pulmonary/Allergy/ Rheumatology, 8% Gastroenterology/ Inborn, 7% Neurology, 3% Psychiatry, 6% Dermatology/Dental, 2% Anti-Infective, 2% Anesthesia/ Analgesia/Addition, 2% Cardiovascular/ Renal, 1% Transplant/ Ophthalmology, 2% Source: FDA, 11/30/2015
  12. 12. © 2018 PAREXEL INTERNATIONAL CORP. / 12 UNIQUE ASPECTS OF ACCELERATED APPROVAL (AMONG OTHER EXPEDITED PROGRAMS) • No special designation, so no formal application/grant process • Permits initial FDA approval of medicines for serious or life-threatening diseases based on drug’s effect on surrogate endpoint or “intermediate endpoint.” Confirmatory studies necessary post approval. • Process leads to a conditional approval (full approval follows confirmation of clinical benefit) • Companies enter AA pathway in different ways • AA pathway has more limited applicability
  13. 13. © 2018 PAREXEL INTERNATIONAL CORP. / 13 A BRAND NEW ACCELERATED PATHWAY: THE BREAKTHROUGH DEVICES PROGRAM  Signed into law in 21st Century Cures Act (December 2017)  Only 13 granted as of December 15, 2017.  For medical devices “that provide a more effective treatment or diagnosis of life-threating or irreversibly debilitating human disease or conditions.” • Represents breakthrough technology; • No approved or cleared alternatives exist; • Significant advantages over existing alternatives; or • Availability is in the best interest of patients. Advantages  Priority Designation  Senior Management Involvement  Interactive Review  Data Development Plan
  14. 14. © 2018 PAREXEL INTERNATIONAL CORP. / 14 NEW FRONTIERS IN PERSONALIZED MEDICINE
  15. 15. © 2018 PAREXEL INTERNATIONAL CORP. / 15 WHAT ARE PERSONALIZED MEDICINES? Leverage biomarkers, often genetic, to determine who is most likely to benefit from a treatment, who is at higher risk of a side effect, or who needs a different dose. Source: Adapted from Bayer Healthcare, “Personalized Medicine.” https://pharma.bayer.com/en/research-and-development/research-focus/oncology/personalized- medicine/index.php (accessed May 2105). With Personalized Medicine Each patient receives the right medicine for them. Patients Therapy 1 Therapy 2 Therapy 3 Biomarker Diagnostics Without Personalized Medicine Some benefit, some do not. Benefit No Benefit Adverse Effects Therapy Patients Each patient benefits from individualized treatment. Minimize Adverse Effects
  16. 16. © 2018 PAREXEL INTERNATIONAL CORP. / 16 RISE AND IMPACT OF PERSONALIZED MEDICINES? Interactive Review
  17. 17. © 2018 PAREXEL INTERNATIONAL CORP. / 17 THE “RIGHT” REASONS TO INCLUDE GENOMICS IN DRUG DEVELOPMENT…
  18. 18. © 2018 PAREXEL INTERNATIONAL CORP. / 18 A JOURNEY WITH GREAT PROMISE…. AND CHALLENGES TOO
  19. 19. © 2018 PAREXEL INTERNATIONAL CORP. / 19 REGULATORY PARADIGMS FOR DIGITAL HEALTH
  20. 20. © 2018 PAREXEL INTERNATIONAL CORP. / 20 THE ACCELERATING PACE OF CHANGE : INFORMATION AGE AND DIGITAL REVOLUTION TIME Magazine (March 2017)
  21. 21. © 2018 PAREXEL INTERNATIONAL CORP. / 21 DIGITIZATION ACROSS THE HEALTHCARE CONTINUUM Healthy Living Prevention Diagnosis Treatment Recovery Homecare Moving healthcare from the clinic to the patient. Understanding patient's behavior and physiology “in the wild.” Focusing on prevention for earlier/smaller interventions. Leverage computing power, sensors, connectivity and software.
  22. 22. © 2018 PAREXEL INTERNATIONAL CORP. / 22 A REGULATORY PARADIGM SHIFT Premarket Timeline Suited For Hardware-based Products Stable Program Volume: 3,500 510(k) Submissions/Year Deterministic Risks, Designated Responsibilities, Physical Products Potential For Exponential Increase In Volume Of Submissions Evolving Issues: Cybersecurity, Distributed Responsibilities, Non- physical Products Software Development Timelines, Software Development Practices, And Rapid Iterations Current Regulatory Paradigm Digital Health Paradigm Shift
  23. 23. © 2018 PAREXEL INTERNATIONAL CORP. / 23 SMART REGULATION PRINCIPLES
  24. 24. © 2018 PAREXEL INTERNATIONAL CORP. / 24 KEY MESSAGES  Regulatory paradigms are evolving • Stay up to date • Engage regulators early and often • Leverage regulatory programs that optimize development  Personalized medicine is the future of healthcare • Not just genomics, also customized devices and digital health • Take a patient-centric approach to product development  Always think about the big picture • Consider combination product issues, reimbursement considerations, and market access at the very beginning of the product lifecycle • Think about the future evolution of the product and plan ahead
  25. 25. © 2018 PAREXEL INTERNATIONAL CORP. / 25 THANK YOU © 2018 PAREXEL INTERNATIONAL CORP. / 25

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