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Drug Development, the FDA and the Role of the PWS Community [WEBINAR]

In this webinar, our expert panelists share how drugs get approved and how we, the PWS community, can help advance new treatments for PWS.

Where are we with respect to new treatments for PWS? What are our next steps and how can the patient community help ensure that treatments for PWS are made available as soon as possible? In this webinar, our panelists discuss:

  • The process to get drugs FDA approval and how the patient perspective is incorporated into the process.
  • Steps the PWS patient community has taken to date to facilitate the advancement of new PWS treatments, and possible next steps.
  • Current challenges in the drug development process and strategies to overcome them.
  • Actions our community can take to help achieve a positive outcome for potential drugs

This webinar has been recorded and is available for viewing below. Short on time? Below is an outline of important segments with timestamps to help you fast forward to the content you most want to hear! A list of questions with their corresponding responses can also be found below. 

Panelists Include:
James Valentine, Associate, Hyman, Phelps and McNamara
Rob Lutz, Board Member, PWSA | USA
Dr. Theresa Strong, Director of Research Programs, FPWR
Alicia Secor, CEO Atalanta Therapeutics, past Chief Commerical Officer, Zafgen



Content Outline and Timestamps

James Valentine, Associate, Hyman, Phelps and McNamara

Has the FDA already decided to NOT approve DCCR? Why is this process taking so long? At what point can my voice make an impact and what can I do? In his 12-minute presentation, James Valentine answers these questions and more! Short on time? Here are a few important timestamps to help you fast forward to the content you most want to hear!

  • Bio and Introduction of James Valentine 4:54
  • Where are we at today? 7:19
  • The Challenges of PWS trials 8:08
  • Has the FDA already decided to not approve this drug? Why is this process taking so long? 9:38
  • When will your voice be needed? How can you provide your voice? 12:57

James Valentine has spent the past 13 years working to ensure patients have a voice in regulatory decision-making. He started his career at the FDA facilitating patient input into the drug review and approval process, and is now an associate attorney at Hyman, Phelps & McNamara. He has helped countless rare disease organizations capture the patient experience to help the FDA understand the medical needs of those communities. He has work in the PWS clinical trial space for more than 5 years and has participated in FDA meetings including FDA meetings with the PWS Clinical Trial Consortium. In 2019, Global Genes awarded James the “RARE Champion of Hope” Award for advocacy.

Dr. Theresa Strong, Director of Research Programs, FPWR

In her 8-minute segment, Theresa Strong discusses: What discussions have taken place between the PWS Community and the FDA? How are we advocating for new treatments for PWS? How are we responding to the current situation? Here are a few important timestamps to help you fast forward to the content you most want to hear!

  • What discussions have taken place with the FDA to date? 18:10
  • What resources have we provided per FDA request? 19:44
  • Our current situation. Covid’s impact on PWS clinical trials 22:33
  • YOUR advocacy efforts (past and present)   24:22

Theresa Strong is co-Founder and Director of Research Programs at FPWR; a member of the FDA Patient Engagement Collaborative, and mom to a young adult with PWS. Theresa has led FPWR's advocacy efforts to develop a library of one dozen resources that have been shared with the FDA to support the review and approval of treatments for PWS. 

Rob Lutz, Board Member, PWSA | USA

As a biotech exec and father to a young adult with PWS, Rob has the unique experience of seeing drug development from the perspective of both drug companies and patient advocates. In his 7-minute segment, Rob gives us insights into the roles and relationships of pharma, the FDA, and patient advocates and the constraints of those relationships.

  • What does the FDA want to hear from patient advocates? 27:10
  • How do we (patient advocacy groups) work with drug companies? 28:02
  • What constraints do the FDA and drug sponsors have in working with advocacy groups? 28:40
  • How do we channel our energy in the most productive way possible? 31:31

Rob Lutz is a biotechnology executive and is currently the CFO Strongbridge Biopharma. Rob also has a young adult daughter with PWS and is a 13-year veteran of the PWSA|USA board of directors and chair of PWSA|USA's Research Committee. 

Alicia Secor, CEO Atalanta Therapeutics, past Chief Commerical Officer, Zafgen

In this segment, Alicia Secor brings her perspective working in the pharma industry, and specifically her experience working on the Beloranib trial for PWS, to share how the patient voice was critical in order for Zafgen and the FDA to understand PWS and the potential impact of the therapy.

  • What impact has PWS advocacy had on the FDA and drug sponsors? 32:49
  • Why is educating the FDA important? 34:25
  • What is the importance of remaining neutral? 35:00
  • What can you do to advocate for your loved one with PWS? 35:44

Alicia Secor has held a number of leadership positions in the pharmaceutical industry and is currently CEO of the biotech start up Atalanta Therapeutics. She has been part of the PWS community since 2014 when she was the chief commercial officer at Zafgen. She has been on the Board of Directors at FPWR since 2016.


Community Response Submission to the FDA

In response to the FDA's decision requiring Soleno Therapeutics to complete an additional controlled trial for DCCR, a potential treatment for PWS, FPWR and PWSA |USA collected community comments and on April 7th, submitted a comprehensive patient voice submission to the FDA. The submission addresses:

  1. Our community's urgent and dire need for new therapies
  2. Our positive views of the trial data
  3. Our community's preferences for treatment access despite any uncertainty
  4. The real need and potential benefit DCCCR could provide (as evidenced by the thousands of comments submitted through the sign-on survey)

You can read our letter to the FDA in our blog, or download the full community response below. 

Community Petition Download (2)-png

Questions and Answers

  1. FPWR and PWSA|USA Recently submitted a community response letter to the FDA asking for regulatory flexibility. What can we expect next and what is the timeline? (37:40)
    • The FDA has acknowledged our letter and has pledged to review the package and get back to us with regards to further dialogue.
    • The FDA oversees thousands of rare diseases so sometimes it takes multiple tries to educate the FDA on the patient preferences, experiences, etc, But its worthwhile to take that time because ultimately you want them to be as educated as possible so they will make the most informed decision possible. (James)
    • We don’t know what conversations are taking place as industry is very private. There is no obligation for a company to share their conversations with the FDA and the FDA cannot discuss any drug applications they might have with the public. Therefore, the recommendation of the panel is to wait for an invitation from the company or FDA. When a company or the FDA needs to hear from us [the PWS community] they will invite us in and that will be a very powerful time.
    • PWSA|USA and FPWR will remain in contact with all players and will continue to advocate when needed.
  2. Political Pressure – would if be effective to reach out to our congressmen or senators at this time? (43:49)
    • At this time, the company and the FDA are having conversations. No decisions have ultimately been made in regard to the new drug application. Its ok to keep your representatives up to date on what is going on, but at this time, there is not a specific ask to make of them.
  3. What about Media contacts? If you know someone in the media, is now the right time to reach out to them and ask them to spread the word? (45:43)
    • In my experience [James Valentine] that would not be helpful at this time. Conversations are still ongoing and we want the FDA to remain open-minded. When media pressure is applied, the FDA often feels the need to defend themselves. Right now, we don’t want anything to distract them from their conversations with the companies.
  4. In what instance is a Phase 3 trial not sufficient for a new drug application? Why would the FDA require an additional drug trial? (46:44)
    • Overall, in the DCCR trial, the primary endpoint did not reach significance. Pre-specified subsets did have a significant reduction in hyperphagia (see Soleno’s website for their presentation). Data from the study was significant up to the start of the pandemic. Soleno is stating the pandemic did impact the later study data. A survey conducted by FPWR in regards to the impact of Covid on the PWS community does indicate the pandemic negatively impacted behaviors in our loved ones with PWS.
  5. Why not approve the drug conditionally and then collect more data once the drug is on the market? (49:16)
    • In some cases, the FDA will approve a drug AND also require additional work to be done after it is approved and on the market. Typically, you need to reach a certain bar of proof, that the drug is both safe and efficacious, before they will give an accelerated approval. If the FDA thought the drug was ‘close enough’, this could be an option.
  6. Assuming other drug studies (non-pws) were also impacted by Covid, have any other drugs been able to gain approval during the pandemic? (53:25)
    • The FDA has indicated they are going to try to apply flexibility in regards to evaluating drug applications during Covid, however, we are at the very early days of this and are unaware of any other drug approvals that have taken place.
  7. Have other patient organizations successfully convinced the FDA to change their decisions? (54:24)
    • There are many examples of successful advocacy efforts. When an application is under review, a panel is assembled to make a recommendation to the FDA. That panel will often open the floor to patient advocacy groups to help educate them on the specific disease, and this can be very influential. We can certainly look at many of these case studies and learn from them so that we can be deliberate about how we engage with the agency when its our turn.
    • Even within PWS there are examples of successful advocacy. For example, in the Beloranib study the FDA did a complete 180 on what they were going to require as primary endpoints because of the testimonies shared with them from the patient advocacy groups (FPWR and PWSA|USA).
    • The input provided by our community in our community response letter will help provide color and context to the DCCR new drug application and if the FDA has any questions, they will be able to use that letter as a resource.
  8. Has a drug ever been approved based on its secondary endpoints rather than the primary? (58:36)
    • YES! In rare disease, the FDA needs to understand the context that this is a serious condition and the unmet need is very dire and that the community understands that if the drug were to be approved on rare disease data, that is not black and white, that the uncertainty is acceptable. That is all very important context to allow the FDA to apply more flexibility.
    • Any company that has invested millions of dollars and many years into developing this product, if they see any efficacy signal, they will do everything in their power to negotiate with the FDA so that they can move the drug forward.
  9. How can patients speak directly to the FDA? When is the paitent voice most effective? What activities are most effective? (1:00: 50)
    • Every parent should participate in the Global PWS Registry! PWS is NOT the same for every individual and its important for the FDA to have data that shows these differences. With registry data, we can help the FDA be very informed and represent the full breadth of the PWS community. When you see a new survey offered from the registry, its very important to complete it!
    • Its important that we, as a community, stay aligned and channel our energy to the right place, at the right time. We are not privy to all the conversations that are taking place. We need to be at the ready so that when we are called to action we can act swiftly and efficiently. Please sign up with one of the patient organizations, PWSA|USA or FPWR so that you stay up to date.
  10. Who does the FDA want to hear from? Do they want to hear from trial participants or simply members of the community and when would it be appropriate for us to share our voice? (1:05:55)
    • All voices are important. When discussing a specific drug, people who have taken the study drug will be the most relevant voice, however, when it comes to risk tolerance, how many people are interested in taking a drug, or understanding the unmet medical need – everyone’s voice will be equally important. At different times, different voices should be elevated but across the board, every voice is important and needs to be heard.
    • The 143 page community submission to the FDA includes every comment submitted by the more than one thousand community members who responded, as well as over 26,000 signatures of community supporters.
  11. What is happening with the Carbetocin trial? (1:08:25)
  12. One very important action everyone in the community can take to advocate for their loved one with PWS is to participate in the Global PWS Registry. How often should you update your surveys? (1:09:57)
    • We recommend updating surveys annually or anytime a new survey is shared. It is meant to be a living medical record that you can update regularly. If you have medical changes, it’s good to update at that time.
    • The Global PWS registry is critical to our advocacy efforts. It is telling a story over time. It helps us understand the natural progression of PWS and can help develop clinical trials as well as advocate for new treatments. We can also use registry data to see how a drug changes the progression of PWS and in the long term, it may give us more safety and efficacy data. The bulk of the dozen resources we have shared with the FDA have derived from registry data and we will continue to pull data from the registry in our ongoing advocacy efforts.
  13. Related to our population size, is there any concern we will run out of people to participate in PWS clinical trials? (1:14:12)
    • That is always a concern in rare disease because for every clinical trial there will be various eligibility requirements such as age, and there will be conditions that exclude some from participating.
    • The only people who are going to help us get treatments are us, the members of this community. So its important to learn about the clinical trials and participate when its right for your loved one and your family.
  14. Who has access to the registry information data sets? (1:16:00)
    • When you participate in the registry you are required to complete a consent which details who has access to registry data. We do not share any identifying information and we have a registry advisory board that review every data pull prior to sharing the data to ensure the safety of our participants.
  15. In regards to the FDA’s decision to require an additional trial for DCCR, is this request surprising? Is it unusual for the FDA to require additional trials? (1:17:21)
    • When they feel there is not enough data, it is common for the FDA to tell the sponsor to get more data. Our question is whether the data Soleno has collected is actually sufficient. Our disappointment has come from their decision that an additional trial is necessary without a full review of the study data.
  16. Where can more information be found regarding current PWS Clinical Trials? (1:19:27)

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.