James E. Valentine

Prescription Drugs and Biologics
Provides strategic advice on matters involving product development and marketing approval, including clinical trial design, expedited programs, and incentives programs.
Counsels clients on submission strategies, as well as preparing for meetings with FDA and Advisory Committee meetings.
Prepares requests for orphan drug designation, breakthrough therapy and  RMAT designation, and priority review vouchers.

Patient Engagement
Provides strategic advice for patient and patient group engagement with sponsors and FDA.
Plans and moderates Externally-Led Patient-Focused Drug Development meetings.
Counsels clients on development of ‘patient experience data’ to inform product development and review , including patient narratives, surveys of patient experiences and preferences, and focus groups.
Develops Voice of the Patient reports, draft benefit-risk frameworks, and Draft Guidance for submission to FDA.

Clinical Trials Enforcement and Compliance
Provides counsel on clinical trials operations and compliance matters, including informed consent, reporting adverse events, and registration and results reporting.
Assists with responses to BIMO inspection reports, warning letters, and clinical investigator disqualification proceedings.

Corporate Transactions
Conducts FDA regulatory due diligence related to financings, mergers and acquisitions, and licensing deals involving pharmaceutical and biotechnology companies.

• FDA Outstanding Service Award
  2010
• FDA Commissioner’s Special Citation
  2012
• CDER Team Excellence Award
  2013
• FDA Group Recognition Award
  2011, 2012, 2013, 2014
• Global Genes RARE Champion of Hope Nominee
  2017

• Session Chair, DIA 2023 Global Annual Meeting – ILLUMINATE, FDA Rare Disease Town Hall, June 25-29,2023
• Webinar, Chan Zuckerberg Initiative’s Rare As One program, Rare Disease Advocacy, RWE, RWD, Natural History Data, and the FDA, December 1, 2022
• DIA’s Annual Meeting, Panel Moderator: FDA Rare Disease Town Hall, June 19-23, 2022
• DIA’s Annual Meeting, Panelist: Patient-Focused Drug Development: Reflecting on a Decade of Insights, June 19-23, 2022
• Biopharma Congress Virtual Series, Panelist: Patient Focused Drug Development: Lessons Learned and Moving Forward, January 18, 2022
• Rare Disease Week on Capitol Hill, Panelist:  Rare Disease Congressional Caucus Briefing: “Rare Disease Therapy Development: Existing Challenges and Proposed Innovative Solutions, July 14-22, 2021
• San Diego Regulatory Affairs Network Conference, A Window into the Changing Landscape of FDA Drug Effectiveness Standards, March 17 2021
• Session Chair, 2021 MDA Virtual Clinical & Scientific Conference, The Changing Regulatory Environment: Impact on Neuromuscular Disease Drug Development, March 16, 2021
• Moderator, Jett Foundation Celebrating Rare Disease Day, February 26, 2021
• RAPS Regulatory Convergence 2020, Gene Editing – Where are we now?, September 13-15, 2020
• DIA Annual Meeting, Patient Voice and Its Role in Bringing Meaningfulness into Selection of Clinical Outcome Measures, June 17, 2020
• DIA Annual Meeting, Global Rare Disease Town Hall, June 16, 2020
• DIA Annual Meeting, Measuring the Impact of Patient Centricity in Drug Development, June 14, 2020
• Global Genes and Orphan Disease Center at the University of Pennsylvania School of Medicine’s RARE Drug Development Symposium,  Panelist, Strategies for Small Patient Populations, June 12, 2020
• Webinar, NY/NJ RAPS Chapter, Conducting Clinical Trial Amidst COVID-19 Pandemic, April 27, 2020
• DIA All Hands Meeting on COVID-19 (VIRTUAL), April 14, 2020
• Moderator, Regenerative Medicine: Regulatory, Enforcement, and Legal Challenges for Cell and Gene Therapies, Regulatory Issues Across Both Cell and Gene Therapies, April 2, 2020
• PRA Health Sciences World Rare Disease Day, Reframing How Regulators Use Patient Voice in Decision Making, February 28, 2020
• RARE Patient Advocacy Summit, “Technologies for Learning About Rare Disease for Patients and Caregivers”, “Breakthrough for Rare Neurological Syndromes”, September 19-20, 2019
• DIA 2019 Global Annual Meeting, Global Rare Disease Town Hall, June 23-27, 2019
• FDLI’s Patient Organizations: An Introduction to U.S. Drug Law and Regulation Conference,  Submission of Marketing Applications & the FDA Approval Process, November 7-8, 2018
• Moderator, Externally-Led Patient-Focused Drug Development Meeting on Cystic Fibrosis, October 29, 2018
• Moderator, Externally-Led Patient-Focused Drug Development Meeting on Charcot-Marie-Tooth (CMT), September 28, 2018
• Moderator, Externally-Led Patient-Focused Drug Development Meeting on Chemotherapy-Induced Hearing Loss in Pediatric Oncology, September 13, 2018
• Moderator, Externally-Led Patient-Focused Drug Development Meeting on Alport Syndrome, August 3, 2018
• Moderator, Externally-Led Patient-Focused Drug Development Meeting on Barth Syndrome, July 18, 2018
• DIA Global Annual Meeting, The Patient’s Assessment of the Patient-Focused Drug Development Meeting Initiatives and Global Rare Disease Town Hall, June 24-28, 2018
• BIO International Convention, Patient-Focused Drug Development 2.0: Implementation Update under 21st Century Cures & PDUFA VI, June 4-6, 2018
• Rare Patient Advocacy Symposium: Sharing Patient Voices Throughout the Drug Research and Development Process, Drug Development 101: The Basics of the Process, May 19, 2018
• World Orphan Drug Conference 2018, FDA & High Performance Communities: Understanding Compliance and Patient Experience Data, April 25-27, 2018
• Moderator, Externally-Led Patient-Focused Drug Development Meeting on Epidermolysis Bullosa, April 6, 2018
• Moderator, Externally-Led Patient-Focused Drug Development Meeting on Pachyonychia Congenita, April 6, 2018
• American Society for Experimental Neurotherapeutics Annual Meeting, Dinner Symposium: Patient Involvement in Drug Approval, March 7-10, 2018
• Food and Drug Law Institute Patient Organizations: An Introduction to U.S. Drug Law and Regulation, Panelist: Question & Answer Panel, November 7-8, 2017
• Food and Drug Law Institute Patient Organizations: An Introduction to U.S. Drug Law and Regulation, Submission of Marketing Applications & the FDA Approval Process, November 7-8, 2017
• CDER Rare Diseases Public Workshop: Strategies, Tools and Best Practices for Effective Advocacy in Rare Diseases Drug Development, So you want to engage with CDER at FDA? Strategies for effective engagement, October 30, 2017
• Fourth Annual Rare Disease Summit, ADVOCACY INNOVATION: Harness Patient Reported Outcomes for FDA Interactions and Enhancement of Clinical Development, October 23-24, 2017
• Leerink Partners Roundtable Series: Rare Disease & Immuno-Oncology, Assessing the Regulatory Approval Landscape for Orphan Drugs, September 27-28, 2017
• Tuberous Sclerosis Complex (TSC) Patient-Focused Drug Development Meeting, June 21, 2017
• DIA 2017 Conference, Defining the Science of Patient Input to Enhance Drug Development and Approval: Regulatory, June 19-20, 2017
• DIA 2017 Conference, Integrating the Patient’s Voice Across the Development Program of Rare Diseases: At the Table – Where to Sit?, June 19-20, 2017
• 2017 Rare Patient Advocacy Symposium: Sharing Patient Voices Throughout the Drug Research and Development Process, No Longer Just the Research Subject: New Roles for Patients in Clinical Trials, May 19, 2017
• Food and Drug Law Institute Annual Conference, Patient Engagement in Regulatory Decision-Making – Navigating the FDA & its Medical Product Centers, May 4-5, 2017
• 8th Annual Biomedical Sciences Day, May 3, 2017
• Spinal Muscular Atrophy (SMA) Patient-Focused Drug Development Meeting, April 18, 2017
• 6th Annual Health Law Regulatory & Compliance Competition, March 4, 2017
• Webinar: 21st Century Cures Act – Pharmaceutical and Biologic Product Development Provisions, January 12, 2017
• Strengthening Patient Advocacy Relations Conference, Legal Review in Trends in Regulations Affecting Advocacy, July 14, 2016
• DIA 2016 Annual Meeting, The Utility of Natural History Studies in Drug Development and Approval, June 28, 2016
• BIO International Conference, Drug Development for Mitochondrial Disease: Examining the Current Landscape and Scientific/Regulatory Gaps, June 6, 2016

• FDA Approval of New Therapy, Duvyzat, for Duchenne Muscular Dystrophy Represents Several Meaningful FirstsMarch 26, 2024

  By Charles G. Raver & James E. Valentine & Frank J. Sasinowski —

• FDA Creates a New Advisory Committee for Genetic Metabolic Diseases – Could This Be an Opportunity to Support Rare Disease Product Development More Broadly?December 13, 2023

  By Mark A. Tobolowsky & James E. Valentine & Charles G. Raver & Frank J. Sasinowski —

• FDA Moves Beyond COVID-19, But Impacts on COVID-19 Era Clinical Trials RemainOctober 11, 2023

  By James E. Valentine & Charles D. Snow —

• Long-Awaited Guidance on FDAMA 115: Confirmatory Evidence Finally Has Its Moment (to be Crossed Off the FDA’s Guidance To-Do List)October 2, 2023

  By Charles G. Raver & James E. Valentine & Frank J. Sasinowski —

• FDA Brings its Formal Meetings Guidance Up to Date: What You Need to Know About Type D, INTERACT, and “In-Person” Meetings Under PDUFASeptember 27, 2023

  By James E. Valentine & Mark A. Tobolowsky —

• Toward patient‑centered treatment goals
  for duchenne muscular dystrophy: insights
  from the “Your Voice” studyApril 20, 2023
• Orphan Drugs and Rare Diseases, a chapter in the book, Principles and Practice of Clinical Trials.August 23, 2022
• The Evolving Regulatory Space – And the Advent of Patient Focused Drug DevelopmentNovember 12, 2021
• Symptoms and Treatment Needs of People with Dementia-Related Psychosis: A Mixed-Methods Study of the Patient ExperienceAugust 9, 2021
• Recent Advances in Systems and Network Medicine: Meeting Report from the First International Conference in Systems and Network MedicineApril 3, 2020
• The Case for the Use of Patientand Caregiver Perception of Change Assessments in Rare Disease Clinical Trials: A Methodologic OverviewJanuary 22, 2019
• The Rules of Engagement: CTTI Recommendations for Successful Collaborations Between Sponsors and Patient Groups Around Clinical TrialsJuly 27, 2017
• FDA Deskbook: A Compliance and Enforcement GuideSeptember 26, 2016
• Quantum of Effectiveness Evidence in FDA's Approval of Orphan Drugs: Update, July 2010 to June 2014, Therapeutic Innovation & Regulatory ScienceApril 27, 2015
• Bringing Your Pharmaceutical Drug to MarketApril 1, 2015