Drug Development and Clinical Trials Process

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Jamie Young
Jamie Young

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All participants will be muted as they enter the webinar.
Please don’t use webcams or video, audio only.
This webinar is being recorded.
Questions or comments can be submitted through chat option.
Purpose of Meeting?
To educate the Food & Drug Administration (FDA)
What it is like to live with FA
Your concerns as the disease progresses
How you currently manage FA
What meaningful treatments look like
Your thoughts & experiences related to
clinical trials
Why Should I Participate?
• Opportunity to have Your Voices heard
• The FDA approves all treatments for FA and
needs to know what is important to you
• This knowledge will impact their decisionmaking, and lead to better treatments and
faster approvals for FA
Why Now?
• The Patient’s Voice has historically been
absent from the drug development process
 until a drug was approved by the FDA (sometimes
with non meaningful endpoints for patients), or
 a clinical trial was failing and the drug company or
FDA wanted input from actual patients to
understand the problem
• Want our Voices heard at the beginning of the
process and throughout
Why Now?
• FDA Safety Innovation Act became law in 2012
 Added expedited development & review of new drugs
with preliminary clinical evidence of a substantial
improvement over existing therapies for those with
serious/life threatening diseases (FDA website)
 Recognized value of Patient Input to the ENTIRE drug
development process (including FDA review & decision
making) (FDA website)
• Stakeholder engagement = FDA Priority
 Initiated a 5-year Patient Focused Drug Development
Program to learn about disease impact on Patient’s lives.
Meeting Logistics
• Who?
FA patients and caregivers
• When? June 2, 2017 from 8:00 am – 12:30 pm
• Where? College Park Marriott Hotel & Conference Ctr.
Hyattsville, Maryland
• How?
In-person or online via a live webcast (meeting
will be video taped and recorded)
* Following the FDA meeting, there will be a research update for
anyone who is interested (1:30-3:00 pm)
• Background: FDA & Drug Development
• Overview: FDA Patient-Focused Drug Development
• Meeting Participation
 Logistics, Format and Tips
• Other key information
FDA and Drug Development
Drug Development and Clinical Trials Process
FDA is responsible for public health and safety.
2 critical approval steps:
1. Early - reviews all drugs that go from lab to clinical trials
• Investigational New Drug (IND)
2. Late – reviews all animal and human data to approve drug for treatment of disease
• New Drug Application (NDA)
Initial IND
Phase 1
Phase 3
Phase 2
FDA review
& approval
Pre-BLA or NDA
End of Phase
2 meeting
Market Application
Opportunities for researchers and pharma to interact with FDA
Drug Discovery
Researchers learn about a disease/disease process, including
what organs/systems are affected and how
• Identify and test thousands of molecular compounds in
the lab
 Which are most likely to have a positive effect on a specific
target or pathway for the disease?
• Once a promising compound is found:
 Drug development follows a very specific path, established
by the FDA, to be sure that drugs are safe and effective
when approved for broad use in humans
Preclinical Development
• Manufacturing
• Laboratory studies & Animal testing (often in mice
& monkeys)
• Occurs before a new drug/biologic is tested in
• Purpose:
 Reasonably safe for initial use in humans?
 Sufficiently effective against a disease target in chemical
assay tests or animal models?
Clinical Development – Phase 1
• If Preclinical Development is positive, Investigational
New Drug Application (IND) application filed with FDA
 Human clinical trials can now begin
• Small trial; usually healthy human volunteers (20-80)
• Purpose:
 Determine drug safety & dosage
 Determine most common side effects of drug
 Determine how a drug is metabolized/excreted
Clinical Development – Phase 2
• Larger trial, with affected patient volunteers (100-300)
 Note: For FA/other rare diseases, fewer patients are required
• Purpose:
 Evaluate safety, side effects
 Evaluate pharmacokinetics and pharmacodynamics
Pharmacokinetics – how organism affects a drug
Pharmacodynamics – how drug affects an organism
 Evaluate effectiveness of drug
 Compare drug against placebo
Clinical Development – Phase 3
• Traditionally large-scale, randomized, placebo- controlled
trials with affected patient volunteers (1000-3000)
 Note: For FA/other rare diseases, # of patient volunteers is < 200
 Note: For rare diseases, the FDA has several approaches for
expediting the development & review of drug candidates
• Purpose:
 Continued assessment of effectiveness, duration of effect in
different populations etc.
 Continued safety evaluation, including potential
drug-drug interactions
New Drug Application (NDA)
• If positive Phase 3 trial (showing safety & efficacy), a New
Drug Application (NDA) is submitted to the FDA
 Includes all animal & human data
• Purpose:
 Allows FDA to begin review process for drug approval
 FDA initiates pre-NDA meeting
 uncover major unresolved issues
 discuss methods of statistical analysis
 identify any further studies needed
FDA Review
• If NDA is complete:
 Review Team is assigned to evaluate it
 Has 6-10 months to approve drug or not
• Purpose of Review:
 Is drug effective for it’s proposed use?
 Is the drug safe? (e.g., do benefits outweigh risks)
 Are the manufacturing methods & the controls used to
maintain the product quality adequate?
• Review team seeks Advisory Committee input, as
 Opportunity for patient input
Post-Market Safety Surveillance
• Knowledge about a product will always be limited at
the time of approval
 Clinical studies are brief in duration and involve a
limited patient population
 New safety information often emerges after a product is
used in a wider patient population
• FDA maintains an active program in post-market
safety surveillance to monitor adverse events
What exactly is the FDA’s role?
What it does and does not do
Drug Development & Clinical Trials
• FDA does not develop drugs
 Researchers, pharmaceutical companies, and nonprofit
groups conduct disease research and drug development
• FDA does not test drugs in clinical trials
 FDA does not run clinical trials
 FDA staff are not onsite for clinical trials
 Researchers conducting trials must seek FDA approval
before beginning
Practice of Medicine & Drug Costs
• FDA does not have authority to regulate the practice
of medicine
 FDA regulates the development & marketing of medical
 Doctors are free to prescribe FDA-approved drugs for
other conditions (known as “off-label” use)
• FDA does not regulate the price of medicine
 Consideration of drug prices is not part of FDA’s mandate
 FDA cannot base an approval decision on drug price
 FDA does not even receive pricing info. during a review
FDA Facilitates Drug Development
• FDA’s mission includes “Promoting Public Health”
• Review teams have expert knowledge about drug
development and clinical trials
 Meets numerous times with companies throughout the
drug development process
 Helps companies design clinical trials & studies
 Provides technical assistance
• Manufacturers pay user fees under the Prescription
Drug User Fee Act (PDUFA)
 Supports FDA involvement in drug development &
review of marketing applications
FDA’s Regulatory Authority
• Foods, including dietary supplements
• Drugs, prescription & non-prescription
• Biologics, including vaccines, cellular & gene
therapy products, and tissue products
• Medical Devices, pacemakers, surgical implants
• Electronic products that give off radiation
• Cosmetics
• Veterinary Products
• Tobacco Products
For more information on FDA
regulation of medical products,
and other ways to get involved
FDA Patient-Focused Drug
(some of the following material reproduced from presentations by
FDA’s Dr. Theresa Mullin and the MDF Webinar presentation)
Purpose of the Meetings?
• FDA wanted a more systematic way to gather the patient
perspective about the condition and available treatment
• Helps inform their understanding of the context for
benefit/risk assessment and decision making for new drugs
• Patient input helps the FDA during drug development and
their review of an application for marketing a new drug
FA Patient Focused Meeting
• Framework for panel questions came from FDA’s
benefit-risk framework; represents important
considerations in their decision making
• We reviewed this & other disease area questions and
tailored our agenda/panel questions to communicate
the most important information related to FA
• FDA emphasizes that:
“Voice of the Patient” Report
• FARA to prepare a written “Voice of the Patient”
report after the meeting
 Summary of patient & audience testimony, polling data &
submitted written comments
• Key communication to FDA review staff & the
regulated industry about what patients most want to
see in a treatment
• FDA wants this information; informs them about
ways to develop meaningful treatments for FA
Participating in the Meeting
Meeting Logistics
• Who?
FA patients & caregivers, FDA staff and
pharma & biotech companies
• When? June 2, 2017 from 8:00 am – 12:30 pm
• Where? College Park Marriott Hotel & Conference Ctr.
Hyattsville, Maryland
• How?
In-person or online via a live Webcast
* Following the FDA meeting, there will be a research update for
anyone who is interested (1:30-3:00 pm)
Overview of the Agenda
• Welcome and introductory comments
from FARA, MDA, NAF and the FDA
• Two patient/caregiver panels:
 How FA symptoms affect your life
 How you manage symptoms and current
& future approaches to treatment
• Polling Questions & Moderated
Discussion with audience after each
• FDA Closing comments
James Valentine
Discussion Questions
• Panel 1: Living with FA
 What 1-3 symptoms impact you the most (e.g.
ataxia/walking, fatigue, speech)?
 How do they affect your daily life? Examples?
 How have your symptoms changed over time?
Any specific activities that are important to you that
you can no longer do or do as fully?
 What most worries you about symptoms or
 If you could fix one symptom, what would it be?
Discussion Questions (cont.)
• Panel 2: Current and future approaches to
treating FA
 How do you manage your symptoms now?
(e.g. PT, surgery, cardiac medications)
 How well are therapies working?
 How well do they improve your ability to do
specific activities important in your daily life?
 What specifically would you like to see in a
treatment? (short of a cure)
Discussion Format
• For each topic, the patient/caregiver panels will speak
 Purpose: to set the foundation for the broader audience
 Panelists reflect a diverse range of experiences: age of onset,
level of progression, key symptoms etc.
• Patients and caregivers (only) in the audience and online
will have a chance to answer “polling” questions
 Purpose: starting point for the discussion
 How? Cell phone (via text message) or through a web page
Discussion with patients & caregivers in the audience
 Purpose: to build on the experiences shared by the panel
 Moderator will ask questions & invite you to raise your hand to
Tips for Effective Participation
• Remember FDA’s role & the purpose of the meeting
• Review each question in advance
• If you have something important to share, relate it to
the most appropriate topic/panel question
• It is OK to reiterate a feeling/experience already voiced
by someone that is similar to your own, but give it a
personal or unique perspective
• Keep your comments concise & focused; there are
many voices to be heard about this emotional topic
• Comments will be collected from those online during
the webcast or can be sent to FARA within 30 days
Participating in the Discussion
• How
 In person (hotel in Hyattsville, MD)
 By webcast (remotely; polling questions & written submissions)
• Register
 If attending in person (only): https://fda-patientmtg.eventbrite.com
• Webinars
Tuesday, April 4, 2017 12:00pm EST; 9:00am PT
Tuesday, April 11, 2017 8:00pm EST; 5:00pm PT
• Submit comments, with a focus on the panel questions, for up to 30
days after the meeting to:
 Jane Larkindale: [email protected]
 Comments included in FA’s “Voice of the Patient” report submitted to FDA
Questions? Need Assistance?
• The webinars will be recorded & available online
if you cannot attend on 4/4 or 4/11. 4/4 link:
• http://www.curefa.org/conference
• For more information or if you have questions
about participating at the FDA meeting, contact
Katie Schultz, [email protected]
• For issue about the hotel, contact Jamie Young,
[email protected] or by calling 484-8796160
Travel Support Application
• FARA has limited support funds to help cover travel
& hotel expenses (only)
• Applications accepted from 4/15-4/25
 Google form - https://goo.gl/forms/YAAgUqHWyPOwyLVy2
• Funding decision announced on 4/30
• Maximum of $750/family (including non family caregivers)
• Room rate is capped at $139 and is included in the
$750 maximum
• Funds awarded on a first-come, first-serve basis
• This is OUR OPPORTUNITY to be part of the process
• We can have a meaningful impact on clinical trial
design & drug development
• Our (collective) voices must be heard at the
beginning of the process to help:
 companies design trials that meet our needs
 FDA assess risks & benefits with a full understanding of
the impact of FA & the patient perspective
• http://www.curefa.org/conference - live April 5th

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