Registration, Coffee & Tea
Co-Chair’s Opening Remarks
Jim Roach, MD
Bloomberg Intelligence on Biotech Landscape
- How the current pricing structure will impact the biotech industry
- What can CMOs do to help inform the discussion of the future?
- How is this affected by the evolving relationship between industry and academia?
- We’re seeing many more companies focused on rare diseases and immuno-oncology than common diseases now. How is the shift in therapeutic areas affecting the industry?
Asthika Goonewardene, MBA
Senior Biotech Analyst, Bloomberg Intelligence
How is the Shifting Biotech Life-Cycle Changing Clinical Partnerships with Big Pharma and the Role of the CMO?
- Why are many biotechs staying intact as opposed to looking for an exit strategy?
- What is the CMO’s role with big pharma in the partnership?
- What is big pharma’s role in the partnership? Do they have a say in trial design and operations?
Christopher Heery, MD
CMO, Bavarian Nordic
Robert Ross, MD
CMO, Surface Oncology, Inc
Annual Keynote Address on the Changing Clinical Trial Landscape: Anticipating the Migration of Clinical Research Into Clinical Care and the Impact on Biotech
Identifying, initiating and conducting clinical trials among traditional investigative sites remains highly inefficient and unpredictable. Several major trends are moving industry-funded clinical trials into large health systems. This session:
- Reviews major trends and conditions driving this migration
- Explores new clinical trial models and processes emerging in clinical care settings
- Anticipates new and modified capabilities to best leverage health system advantages
Director of Sponsored Research/Founder, Tufts CSDD/CISCRP
How can CMOs Ensure Strategic, Impactful, and Mutually Beneficial Patient Engagement?
- How can CMOs promote compliant and impactful engagement to enhance objectives and meet goals for both their organizations and patient groups?
- How can patient engagement can enhance trials? What is their contribution to benefit/risk? What about engaging patients/groups outside of trials?
- What is the CMO’s role in patient engagement? What are best practices and how can we move the needle to prepare for the future?
Jodie Sherman Gillon
Global Medical Lead, Patient Engagement Rare Diseases, Pfizer
Fireside Chat: Taking a Strategic Approach to Global Regulatory and Market Access Challenges
- Is a global regulatory and market access strategy the way to go?
- Why isn’t EMA approval the same as market access in Europe
- What is the difference between EMA and FDA in regulating and approving medicines?
- When and how do you generate evidence that will satisfy regulators and payers?
- What is the best strategy to interact with regulators and payers?
- Early access schemes in EU and how to qualify
- How will trends like Real World Evidence influence regulatory decision making?
- Brexit and its consequences for the EU regulatory system and how will it affect pharmaceutical companies
Lunch and Therapeutic Specific Round-Tables
Take the first 25 minutes to get lunch and take a break. At 12:55 pm, you have the option of joining a Therapeutic Specific Round-Table where a leader is assigned to help facilitate discussion in your specific therapeutic area of interest.
Breakout Sessions Addressing Therapeutic Areas
Join a discussion with other researchers in your therapeutic area for a collaborative session exchanging creative solutions, lessons learned, and creating new pathways.
Table 1: Addressing CNS Disorders
Table 2: Addressing Rare Disease
Table 3: Addressing Oncology
Table 4: Addressing Gene Therapy
How to Find the Right People and Build the A-Team
The model of the recruiter has stayed the same, but the ability to successfully recruit the right people for your team has drastically changed.
- How do you recruit for specific skill sets within your therapeutic area?
- What is the best way to fill difficult positions?
- If you’re based in an area full of talent, like Boston, how do you retain that talent?
- If you’re based in an area without high recruiting potential, how do you attract talent?
Alan Roemer, MBA, MPH
SVP, Corporate Development, Roivant Sciences, Inc
Best Practices in Clinical Development and Outsourcing
- How do you go about establishing clinically relevant endpoints?
- When is it best to involve KOLs of your field? Expert researchers?
- How do you involve KOLs in your communications with FDA/regulatory agencies?
- Do you have a good measure on what the endpoints are? Including patient forward outcome measures?
- How do you design a clinical trial where you get an early stage read out?
- What is an example of accelerated clinical development pathways?
- How can you create value as quickly as possible?
Ralf Rosskamp, MD
CMO, Dicerna Pharmaceuticals
David Clark, MD
CMO, Aldeyra Therapeutics
Jack Modell, MD
VP and Senior Medical Officer, Rho, Inc
Adrian Senderowicz, MD
SVP, CMO, Constellation Pharmaceuticals
Model-informed drug development? Why it matters especially to early stage and limited portfolio companies
FDA Commissioner Scott Gottlieb has stated repeatedly that innovations in clinical research – specifically, adaptive trials and generally, more model-informed approaches, will speed the development process. But what are the actual experiences among biotechs and special pharmas? And what if your development goals stop short of filing for an NDA?
Quantitative pharmacology, data science, adaptive designs, trial simulations and model-supported decision-making: Hear the real impact from the adopters.
Director, Clinical Research Services Business, Cytel
New Methods for Safety Signal Identification and Refinement, Including Machine Learning
- How have patient safety measures evolved?
- How do you prepare for the challenges of pharmacovigilance once your drug is approved?
- How is the digitization of health data creating new pathways in pharmacovigilance?
Best Practices in Communication and PR Strategy
- How can CMOs more effectively communicate with CEOs, CSOs, and Clinical Operations executives in order to provide a comprehensive summary of the science and business aspects of the company?
- Learning to communicate to investors, analysts, board members, professional groups, investigators, patient advocacy using language they respond to
- How can you help your CEO sell the accelerated clinical development path?
- How can we better educate the public on expectations for clinical trial outcomes?
- What can we be doing better to address negative press coverage?
- How do you manage a crisis, like a death in your clinical trial, to the public?
- How can we prepare for negative outcomes before they happen in real-time?
Zain Kassam, MD, MPH
CSO, Finch Therapeutics
Coffee & Tea
Co-Chair’s Opening Remarks
Jim Roach, MD
Keynote Fireside Discussion: Lessons Learned from a Veteran Biotech and Investor MD
Elizabeth Stoner, MD, MS
Managing Director, MPM Capital
De-Risking a Failed Development Program
In this session, we explore how expanding phase 2 programs, balancing development for both regulatory and marketing purposes, and de-risking a failed development program can further advance your clinical trial in the long term.
Keynote: Best Practices for Biotechs to Collaborate with Large Pharma
CMO Exchange Between Small and Large Pharma: Addressing Mutual Challenges
- What experiences can be translated across the industry divide?
- What can large pharma learn from small pharma to increase efficiency?
- What are the advantages of large pharma?
- Lessons learned
Steven Zelenkofske, DO
Case Study: Preparing for the Challenges of Late Stage Trials
Case study from one company who lived to tell the tale.
Mark Currie, PhD
SVP, CSO, President of R&D, Ironwood Pharmaceuticals
Adaptive Site Management: A Novel, Right the First Time and Quality by Design Approach to Managing Your Clinical Trials
- Site-Focused: Using a proprietary technology to empower your sites and incorporate a high-performing study coordinator into the process
- Increase quality, transparency and efficiencies while reducing your clinical trial costs by more than 35%
- Case Study: How to run a Phase III trial with 250 patients and 50 sites monitored by only 1 CRA
Pricing Trends and the Impact on R&D
- How has pricing structure evolved for better or for worse?
- How do we determine pricing of new drugs going to market?
- Is the current pricing structure sustainable?
- What are we doing to make drugs more affordable?
Finding the Common Ground between CMOs and VCs
- CMOs are looking for VCs who take a long term view at their science as an investment
- Is it always important to be the quickest, cheapest option?
- Are you looking for an exit strategy or a long-term strategy?
Going from Big Pharma to Small Biotech Leadership: Challenges and Opportunities
- Handling the false assumption that small pharma is less secure
- Big pharma provides structured learning opportunities, but small pharma provides opportunities for exploration and collaboration
- Dealing with potential lack of resources when looking for answers
Manu Chakravarthy, MD, PhD
CMO, SVP Clinical Development, Axcella Health, Inc
Keith Gottesdiener, MD
CEO, Rhythm Pharmaceuticals
Robert Wasserman, MD
CMO, Northern Biologics
CMO Leadership Case Study: A Unique Approach to Registration in a Large Indication
Hear from global and multicultural pharmaceutical executive, Christophe Arbet-Engels, about how Poxel decided in a very crowded market to go to a smaller country first, Japan, to start a Phase 3 registration program and find a commercial partner.
Christophe Arbet-Engels, MD, PhD
CMO, Poxel Pharmaceuticals
Should Patients Have the Right to Try?
The debate of compassionate use and right to try has created a lot of controversy in the industry. In this discussion, we unpack the implications, both positive and negative, of opening up that door.
Alison Bateman-House, PhD, MPH, MA
Assistant Professor, Division of Medical Ethics, NYU Langone Health
What Could Be a CMO’s Career Trajectory?
- Are all “CMO” roles equal? Are there opportunities for career growth and development across different CMO roles?
- What are the possibilities for CMOs to expand their horizons within biotech/pharma? Head of R&D? CEO? Other?
- What about other possibilities outside of a “company” environment? Consulting? Venture Capital? Board roles?
- How can a CMO best position themselves to be considered for different types of leadership opportunities?
- What are your individual/personal goals and ambitions and how should these influence your career choices?
- How do you balance the difference between what you want to do vs what you (or others) think you should do?
Jim Roach, MD
Mark Corrigan, MD
Co-Founder, President R&D, Tremeau Pharmaceuticals
Gregory Fiore, MD
President and CEO, Sollis Therapeutics
Steven Zelenkofske, DO