The GSA will be participating in a PFDD on Friday, October 8, 2021 from 10AM EST to 3:30PM EST. This meeting is a way for the FDA to hear the voice of the patient. The GSA wants FDA officials to experience days in our lives, and to walk away with an understanding of what we want to see in future treatments for Gorlin syndrome. This includes giving the FDA meaningful insights into the level of risks we would be willing to accept in exchange for potential benefits in treatments as well as what treatment outcomes matter most to us. This meeting provides a unique forum to help achieve this objective. Will you join us?

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If you are a person living with Gorlin syndrome or a caregiver, you are invited to answer one or more of the following questions. We encourage you to provide specific examples to help the FDA understand your feedback.

  1. Of all the symptoms that you experience (or your loved one experiences) because of Gorlin syndrome, what are the 1-3 symptoms that have the most significant impact on quality of life?
  2. How do these symptoms impact specific activities that are important to you?
  3. What are you doing (or what is your loved one doing) to help treat Gorlin syndrome, and how well are those treatments working?
  4. What are the most significant downsides to those treatments?

The U.S. Food and Drug Administration (FDA) is hosting this virtual meeting on Friday, October 8 from from 10:00 a.m. to 3:00 p.m. EST. Click here to register to participate.

Yes. Please register even if you cannot attend virtually all day. This will allow you to obtain the link to watch / listen, answer polling questions, ask questions, and make comments when you are able to.

  • We need to educate the FDA and regulators on Gorlin syndrome. They need to hear what the manifestations are, how we deal with them, how the manifestations and care needed affect our lives. Drug developers may be interested in learning this as well and will be invited to the meeting. This needs to be done by as many people as possible, not just a few. The FDA needs information on the condition and treatment options. This helps assess the benefit and risk assessment / evaluation of a potential new treatment.
  • The FDA wants to hear from you in the meeting. This is done by answering polling questions that will be asked on your computer, tablet, or smartphone and calling in to provide statements and responses to questions.
  • Your attendance shows that you are part of a community that cares about one and other, and the future of all those affected with and by Gorlin syndrome.
  • The FDA needs to know what is important to you to help make drug approval decisions.
  • FDA wants to hear from patients & caregivers. This is your opportunity to do so. This provides you with the opportunity to have a meaningful impact on clinical trial design and drug development.
  • Trials will be designed that meet member’s needs. It is up to us to tell them what our needs are.
  • The FDA assesses the risk vs. the benefit of each therapy they review. This is a subjective decision that must be calibrated to the specific disease. The PFDD meeting provides them with data on the subjective benefit vs. risk as it relates to GS manifestations. Because we are a very rare disease, we need to teach the FDA about all the body systems affected. The FDA needs to be educated on all the manifestations. We need to inform the FDA on the impacts on people of all ages. This can only do accomplished with participation by affected individuals & parents / caregivers.

Ideally, attending the whole time would be best.  However, if you can only make it for a few hours, or come and go the morning session will run 10:00 AM  to 12:15 PM  and the afternoon session will go from 12:45 PM to 3:00 PM.

A PFDD meeting is a way for the FDA, which is responsible to reviewing and approving new medical treatments, to learn what it is like to have and live with a certain condition or to deal with existing treatments for that condition. Since 2012, the FDA has hosted more than 20 of these meetings to learn about different diseases and conditions. This year, they are hosting a virtual PFDD meeting on Gorlin syndrome.

The intent of the PFDD movement is to ensure that the FDA understands the perspectives of individuals and caregivers living with certain disorders and keeps those in mind when reviewing prospective treatments. We want and need the FDA to understand the experiences of people with Gorlin syndrome, including the manifestations, limitations it places on daily activities of life, the social frustrations, and the emotional pain. Simply put, we want FDA officials to experience days in our lives, and to walk away with an understanding of what we want to see in future treatments for Gorlin syndrome. This includes giving the FDA meaningful insights into the level of risks we would be willing to accept in exchange for potential benefits in treatments as well as what treatment outcomes matter most to us. This meeting provides a unique forum to help achieve this objective.

For many years, Congress and patient advocates have been demanding that patients have a greater voice in the FDA medical product review process. Until recently, patients were seen largely as end users, customers, or as participants (also the unacceptable term, “subjects”) in clinical trials but were rarely viewed as equal partners in this work. This paradigm is now shifting via patient-focused drug development or PFDD for short. Simply put, PFDD is a suite of laws and policies that provide opportunities for patients to play a more engaged role in the FDA review process. The Gorlin syndrome PFDD meeting is one example of PFDD.

The FDA follows a specific model for PFDD meetings. Each meeting includes the following:

  • Presentation from a medical expert about the disease/condition, including current treatment options.
  • Panels of people who speak about their experiences living with the disease or condition as well as with treatments.
  • Discussion sessions where members of the virtual audience can talk about their experiences with the disease or condition.
  • Live polling questions about disease symptoms and treatments that audience members can answer via their mobile device, tablet, or computer.

Yes! There are a couple ways you can make sure your voice is heard at the meeting. Meeting participants can respond to live polling questions and participate in a moderated discussion to tell the FDA about their experience with Gorlin syndrome.

You can still provide input to the FDA! From now until October 31, 2021, you can submit a written comment about your experience living with Gorlin syndrome. Click here to view the full instructions and to submit your comment.

PFDD meetings focus on two topics:

  • What it is like to live with a specific disease and how this affects your daily life.
  • Your experiences with treatments for the disorder, and hopes for future therapies.

Specific topics that will likely be covered at the meeting include:

  • The aspects of Gorlin syndrome that have the biggest impact on your life.
  • How your Gorlin syndrome manifestations and experiences have changed over time.
  • Activities that you would like to do but can’t or won’t do as fully because of your Gorlin syndrome.
  • What worries you most about your or your affected child’s Gorlin syndrome.
  • What, if anything, you are currently doing to treat your Gorlin syndrome or what treatments you have tried in the past.
  • How well those treatments work.
  • The downsides of any treatments you currently do or have done in the past.
  • What factors you take into account when considering a treatment for your Gorlin syndrome.
  • Short of a cure, what would an ideal treatment for Gorlin syndrome look like?

A document called the “Voice of the Patient” Report will be written using information shared in the meeting.  This will become a permanent record at the FDA and on the GSA website.  Regulators and other interested parties will use this document to understand how a potential product will impact individuals affect ted by Gorlin syndrome.

Remember that your objective should be to help the FDA understand what it is like to live with Gorlin syndrome and what you think about current and potential future treatments. The FDA has organized this meeting to learn from members of the Gorlin syndrome community, so you should not use this meeting to criticize the FDA, physicians, or pharmaceutical companies. Although health insurance and the cost of healthcare are important issues, those topics are outside of the FDA’s role and are not the focus of this meeting.

Yes.  You responses are just as important as those of affected adults.  All ages must be represented.

What if I am affected as well as my child(ren)?  How do I answer the polling questions for each of us?  This will require you to sign into the meeting on one device for each affected individual.  You may want to consider asking a family member or friend to be with you and help by answering on one device while you answer on another.

Yes! You are welcome to register for the meeting to show your support for people with Gorlin syndrome. If you are a parent of a child (under 18) with Gorlin syndrome, or a spouse, and/or caregiver we highly encourage your participation.

Yes! Although PFDD meetings are largely designed for the FDA to hear from patients, clinicians and researchers often attend these meetings to show their support and learn more about the experiences of people living with the condition. During the meeting, there will be a public comment period when participants who don’t have Gorlin syndrome will have an opportunity to make brief remarks. You are also welcome to submit a written comment about your experiences treating people with Gorlin syndrome.

Only first names and where you live will be used.  For example: Jim, Boston, Massachusetts.

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