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PFDD community forum
- 1. f r a g i l e x . o r g Patient-Focused Drug Development Meeting (PFDD) Community Forum
- 2. 2 • Opportunity to share patient and family perspectives, needs, priorities and experiences with FXS to inform FDA’s decisions and oversight of drug development and review of market applications. • Wednesday, March 3rd from 10:00AM - 3:00PM ET Externally-led Patient-Focused Drug Development (PFDD) meeting for Fragile X Syndrome
- 3. 3 • The topic is drug development in Fragile X syndrome. • No one can read our minds; we have to be clear and vulnerable. • Four hours is not a lot of time! • This will make a difference. We need YOU. Setting the stage for a successful meeting
- 4. 4 History of the PFDD program at FDA “… As experts in what it is like to live with their condition, patients are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation.”
- 5. 5 What to expect- BEFORE • Register for the PFDD meeting online • If you registered, you have already seen the discussion questions! • Agenda & discussion questions posted • Pro tip: Review the agenda and questions, think about how you would answer them.
- 6. 6 What to expect- DURING • Honest, focused conversations moderated by experts. Support and active listening by the audience. • Live and pre-recorded panelists. • Raising your hand in Zoom. • Pro tip: Be prepared, write out your points and make sure you leave room for others to share.
- 7. 7 What to expect- Agenda
- 8. 8 What to expect- Discussion questions Discussion Topic 1: Health Effects and Daily Impacts of FXS • Which 1-3 symptoms of FXS have the most significant impact on life? How do this impact daily life on the best days? How about the worst days? • Are there specific activities or skills that are important to you/your child but that you/your child cannot do at all or as fully as you/your child would like because of FXS? Discussion Topic 2: Current Approaches to Treatment of FXS • What symptoms would you most like a treatment to target? • What specific things would you look for in an ideal treatment for FXS? What would you consider to be a meaningful improvement?
- 9. 9 What to expect- AFTER • Debriefing sessions • Open docket for comment • Meeting recording and transcript • Voice of the Patient Report • Precompetitive work groups
- 10. 10 Let’s look at three communities who had PFDD meetings: • CLDK5 • Cystic Fibrosis • Autism Haven’t convinced you yet?
- 11. 11 Let’s ask some experts
- 12. 12 • The topic is drug development in Fragile X syndrome. • No one can read our minds; we have to be clear and vulnerable. • Four hours is not a lot of time! • This will make a difference. We need YOU. Remember…
- 13. Happy to answer any questions: hilary@fragilex.org
Editor's Notes
- Happy New Year! Thanks for joining the PFDD Community Forum, we are so excited you are here. I am Hilary Rosselot, the DoRF and I am going to be sharing details about the upcoming PFDD meeting tonight. We need caregivers and self-advocates to participate to make the PFDD meeting valuable. I realize you may not be sure what to expect, are nervous about sharing your story, so I hope you will leave this forum feeling excited, informed, and ready to share your story on March 3rd. Please know you can always reach out to me with questions! Tonight I will share a bit more about the PFDD initiative at FDA and the purpose, format and outputs of our PFDD meeting for FXS. Then Drs. Randi Hagerman, Liz Berry-Kravis, and Craig Erickson will join us to share more on why the PFDD meeting will make a difference for Fragile X. Finally, we will open it up for your questions. Please use the Q&A function at the bottom of your Zoom window to submit your questions.
- The NFXF has secured an Externally-led Patient-Focused Drug Development (PFDD) meeting for Fragile X Syndrome This is an opportunity to share YOUR voice! NFXF applied to host an EL-PFDD meeting for Fragile X syndrome because we feel there is a need for patient input in the development of treatments for FXS. This community has experienced several failed trials, and the opportunity to share your experience to guide future drug development is powerful. I Hope you have noticed NFXF has built programming like the NFXF Research Readiness Program (link) to support the patient and expert voice in research and hosting a PFDD meeting amplifies your voice even more. The purpose of PFDD meetings is to hear directly from patients, their families, caregivers and patient advocates while all other attendees will be listening and learning from you. You have to apply for PFDD meetings. Not all applications are accepted. Each condition can only host one PFDD meeting, you only get one shot. This is our shot to create change for FXS.
- Before we get into the nitty gritty, I want to share a few key takeaways with you. If you don’t remember anything else you hear tonight, please remember these four things: We know that treatments are not limited to drugs. For this meeting, we are focusing on drugs (FDA’s jurisdiction) and Fragile X syndrome specifically. One condition/meeting and one meeting/condition. We were specific on purpose You have shared so many amazing stories about your family members and friends with FXS, we love celebrating the wins and creating community to support you through the challenges. We do not want treatments that impact the wins, we want treatments to minimize the challenges of FXS. That means we have to be clear, honest, and vulnerable about the tough stuff in FXS. This is going to help FDA and drug developers understand what YOU want in YOUR treatments. They can’t read your minds, you have to share. While four hours sounds like a long time, it will go be quicky! We have a tight agenda based off of FDA recommendations of this type of meeting. We want to make sure we have as many people share as possible, but please know there are multiple ways to actively provide feedback to have your voice be heard. This will make a difference, and it cannot be successful without you. Your voice is the integral piece of this meeting, this is your opportunity to share. We cannot wait to hear what you have to say.
- When you open the FDA website on PFDD, this is what you see. It is the best summary I can find, they want to know what are patients saying. You are the experts. The PFDD sits within the Center for Drug Evaluation and Research (CDER) at FDA and leads initiatives and provides strategic guidance to ensure patient input is incorporated into decision-making. PFDD is not just about the drugs themselves. It includes integrating your input on all facets of the drug development process, including best practices for clinicial trials (enrollment, decrease burden), how to capture/measure the changes in relation to the drug, information that is most important to you related to treatment benefits, risks, and decision making. Let me put this into context, albeit oversimplified: All drugs are regulated by FDA. There is a very specific regulatory process these drugs go through that evaluates safety and efficacy (or effectiveness (ability to produce a desired result) of each drug. Clinical trials are used to measure safety and efficacy, and when the drug has successfully gone through the phases of a CT, met its endpoints, and shows both safety and efficacy, the developer can submit an application to FDA for the drug to go to market. The FDA reviews the drug application and decides on labeling (which includes recommendations on who can take the drug & influences insurance coverage). A big part of the PFDD initiative is understanding patient preferences and the potential acceptability of tradeoffs between treatment benefit and risk outcomes. The FDA uses this feedback in a Benefit-Risk Assessment Framework. So for a drug to be approved for marketing, FDA must determine that the drug is effective and that its expected benefits outweigh its potential risks all while considering the severity of the condition and the current medical needs for patients. These frameworks for are posted publicly on the FDA website as part of the review documentation for approved products. Our PFDD meeting can inform the benefit-risk analysis framework used by FDA when considering drugs for FXS. So let’s talk a little more about meeting details.
- Good news, you’ve already started to prep by being here today! We are about 6 weeks out from the meeting. Before: access to agenda, discussion questions, this forum! Discussion Topic 1: Health Effects and Daily Impacts of Fragile X Syndrome Discussion Topic 2: Current Approaches to Treatment
- 1) Remember those living with FX sharing, others in listening mode Discussion Topic 1: Health Effects and Daily Impacts of Fragile X Syndrome Discussion Topic 2: Current Approaches to Treatment Large-group facilitated discussion
- I know this is small, will be on website. Data from surveys, thanks for participating Discussion Topic 1: Health Effects and Daily Impacts of Fragile X Syndrome Discussion Topic 2: Current Approaches to Treatment
- These are only some of the discussion questions. D? Topic 1, #2, Examples may include daily living or communication skills, learning or social activities. D? Topic 2, #2, How big of a change/effect would you need to see to use a new treatment, assuming there will be potentially mild-moderate side effects? What impact level would convince you to trial a treatment? (For example, a treatment that positively effects 80%, 50%, 20%, 10% of patients with FXS) Message from us: win for some is a win for all, we will need multiple treatments
- After: debrief calls, public docket (60 days, BRA), FDA website, live stream posted, final report, workgroups Voice of the Patient Report We will submit to FDA to post on their website as well. Handed out to RRP, integrated into CTC recommendations
- Let me share some examples. CDLK5: Robyn Bent joined the US FDA in 2019 as the director of the Center for Drug Evaluation and Research (CDER) Patient-Focused Drug Development (PFDD) Initiative. Having previously worked with patients and families for 20 years as a pediatric nurse, Bent understands many of the priorities these people have. But she was proven wrong early during an externally led PFDD meeting on a rare disease known as CDKL5 deficiency disorder (once classified as an atypical form of Rett syndrome), whose earliest symptoms are seizures and developmental delays. Bent had predicted most parents would be concerned with treating seizures. Yet one mother of a girl with CDKL5 told her that treating constipation was a bigger priority, because of the distress and discomfort it caused her daughter. Takeaway: Even people know “know” your condition will learn. According to CT.gov, there are 13 active studies Cystic Fibrosis (CF) is a rare, genetic, life-threatening disorder that damages the lungs and digestive system. Parents, caregivers and self-advocates shared what living with CF was like and details about their ideal treatments. Before the meeting, drug development in CF was focused on respiratory complications, and self-advocates and families agreed while stressing the mental health and GI issues experienced significantly impact CF patients’ quality of life. Gastrointestinal issues ranked among the top two problematic disease symptoms experienced by those with CF, which was cited often in panel discussions and polling. The need for treatment for GI issues was a main takeaway from the meeting, one that drug developers had not focused on previously. One panelist shared, “I pray for new medical trials that result in treatments that aid my complicated digestive issues...”. Meeting participants expressed strong willingness to assume risks in participation with clinical trials, also indicating that distance, placebo, blood draws, missed work, and multiple clinic visits would not discourage their participation. Now drug developers know while the CF community wants treatments that improve lung function, they also want drugs that minimize or relieve the uncomfortable GI symptoms of CF. Drug developers also know the CF community is dedicated to participating in clinical trials. The FDA uses information from the community, integrating the need for increased lung function and GI relief into their decision making. Since the PFDD meeting in 2018, there have been multiple drugs approved for CF. While there is still much work to do, some individuals with CF are experiencing relief with the help of these drugs. Takeaway: other things besides the main thing, threshold for improvement may be defined differently between stakeholder groups. Another interesting thing, they were measuring lung function. Patients want to be able to get up an answer the phone, not walk a lap around a track. Multiple approvals since their PFDD meeting and over 170 actively recruiting studies on CT.gov—not all drug trials. Autism: Parents, caregivers and self-advocates were able to share what living with Autism was like and what they wanted to see in treatments. Before their meeting, a lot of drug development in ASD was focused on stimming/repetitive behaviors, but self-advocates shared that repetitive behaviors were not bothersome or even their main concern. . As one participant explained, “Therapies intended to suppress autistic traits hurt autistic people.” Individuals with autism stressed that treatments that target nonharmful repetitive behaviors, or stimming, are counterproductive, because those behaviors are useful to individuals with autism as mechanisms to cope with stress or sensory over stimulation. Instead, self-advocates and families stated their ideal treatment would improve overall quality of life and help treat many of the reported co-occurring conditions, including depression, anxiety, and sleep issues. drugs are approved for treatment of irritability associated with autism; however, no drug therapies currently target the underlying causes or core manifestations of autism. FDA-approved products for irritability associated with autism include risperidone and aripiprazole. There are over 200 actively recruiting studies in ASD (CT.gov) PFDD meetings facilitate a conversation around what patients want improved by targeted treatments, what may be the best way(s) to measure that change, and what would be considered improvement. Even people who know about FXS may not know what YOU want.
- Drs. Randi Hagerman, Liz Berry-Krahhhhvis, Craig Erickson will be joining us to talk about the impact of our PFDD meeting on FXS treatment development. Each of these docs are clinician-researchers in FXS, they have been PIs on many of the large drug trials, and are all on the NFXF CTC. Each of them have gone to FDA about drugs for FXS and advocated for the community with drug developers. Welcome and thanks for joining us, please share 1) what the PFDD meeting means for FX and 2) what our community should be thinking about in preparation for this meeting.
- If you don’t remember anything else I say tonight, please remember these four things: We know that treatments are not limited to drugs. For this meeting, we are focusing on drugs (FDA’s jurisdiction) and Fragile X syndrome specifically. One condition/meeting and one meeting/condition. We were specific on purpose You have shared so many amazing stories about your family members and friends with FXS, we love celebrating the wins and creating community to go through the challenges with you. We do not want treatments that impact the wins, we want treatments to minimize the challenges of FXS. That means we have to be clear, honest, and vulnerable about the tough stuff in FXS. This is going to help FDA and drug developers understand what YOU want in YOUR treatments. They can’t read your minds, you have to share. While four hours sounds like a long time, it will go be quicky! We have a tight agenda based off of FDA recommendations of this type of meeting. We want to make sure we have as many people share as possible, but please know there are multiple ways to actively provide feedback to have your voice be heard. This cannot be successful without you. Your voice is the integral piece of this meeting, this is your opportunity to share. We cannot wait to hear what you have to say.