Every successful individual knows that his or her achievement depends on a community of persons working together.
Paul Ryan

Early in the development of DSF we could see that community connections, strategic alliances, and partnerships would be critical. As Paul Ryan said, Every successful individual knows that his or her achievement depends on a community of persons working together. By partnering up, we were able to not only produce meaning and value, but we could do it in an effective and efficient manner with the limited resources we had. This strategy allowed us to address specific problems and issues and to also ensure long-term engagement and community empowerment.  

One of the many problems for all rare disease patients, including those with Dravet syndrome (DS) is finding the best way to treat and manage their disease. For many of our patients, treatments are partially effective at best, and for others there are no effective treatments at all. Being a part of a connected and educated community can change that. We established a system that allows our patient families to have greater input and an understanding of treatments, as well as the opportunity to be recognized as equal partners throughout the drug development process, and this is a key component in developing new treatments and better outcomes.

Rare diseases tend to attract little interest from the pharmaceutical industry, which sees little financial incentive to develop drugs for such a small market. When a company is evaluating whether to pursue a rare disease treatment, it is in their best interest as well as the rare disease patient community to collaborate. They need to be willing to genuinely learn about and commit to meeting the community’s needs, as community support is key to their success. DSF can help industry partners by connecting them with an established community of Dravet stakeholders – caregivers, patients, researchers, and clinicians – that can answer questions that will inform them on effective trial design and product launch, as well as provide expert level knowledge on this very complex disease.

DSF has done the groundwork. We have already connected our community through online support groups, the DSF Family Network, and workshops and conferences. These platforms give families the opportunity to discuss their needs and concerns through surveys, participation in advisory councils, and in person. These efforts ensure that our community and its unique challenges and needs are clearly understood. In turn, this allows the patient voice to be at the center of the drug development process, making DSF and the DS community vital and valuable partners. Through a connected data-driven, patient-focused approach from the beginning, we set our community and industry partners up for success, helping them to alleviate some of the challenges they face and allowing them to accelerate the development of much-needed new therapies.   

Our approach is working. It is very unusual to have multiple companies developing new treatments for the same rare disease. Yet, within a short time, the DS community now has two drugs with FDA approval and indication for DS, several others currently in clinical trial, and even more potential compounds in the pipeline. A shining example of the difference a community and industry partnership can make is certainly the development and approval of Epidiolex, the cannabidiol drug developed by GW Pharmaceuticals (parent company to Greenwich Biosciences). Keep in mind that it typically takes at least ten years for a new drug to go from initial discovery to the marketplace. Clinical trials alone can average six to seven years. GW Pharmaceuticals first met with DSF in 2012. They were open and collaborative from the start and incorporated community input into trial design. They were able to get Epidiolex approved just 6 years later in 2018.  

As might be expected, our community can have intrinsic trust issues – families dealing with DS carry a heavy burden. Often their child was misdiagnosed and/or experienced subpar treatment. They are frustrated by having to give their loved one multiple medications that don’t seem to help and frequently have significant side effects. They are exhausted from the daily pressures and stress that come from with living with DS. If they don’t feel that they are an equal partner and that their voice and their needs are being heard, they won’t feel engaged or make the time to participate. We had to think about what DSF could do help our community overcome these trust issues. As a result, we defined what we expect from our industry partnerships and what we as a community can offer to help move progress forward.

What DSF and the Dravet community expects from industry partners:

  • Mutual respect. We are not an intermediary between patients and industry. We are a strong partner that understands DS from the united personal experience of our community.
  • Honesty and transparency to build trust.
  • Patient-centered designs that can help drive progress to better treatments for DS.
  • Clarity in communication. We expect partners to share as much information as possible on why data is being collected so that participants can better understand the significance of their participation.
  • To share results with the community, to the extent that is possible. This will increase willingness and excitement to participate.
  • A genuine effort to understand the diagnostic and treatment journey, the economic and emotional burden and the quality of life impact for our patients and families.
  • Community engagement through sponsored programs or services that offer our families further disease education and advocacy tools, as well as the opportunity to directly interact with industry partners.
  • Development of a medication assistance grant program if and when the drug receives FDA approval and is available in the marketplace.

To allow DSF to best assist potential industry partners, we:

  • Engage and educate our community members so they better understand the drug development process, as well as the importance of patient centered research.
  • Identify caregiver concerns to help define meaningful clinical endpoints.
  • Aid in protocol design to accommodate patient/family needs for study participation.
  • Offer insights about disease progression.
  • Help with trial site selection.
  • Inform our community about promising compounds and clinical trials.
  • Connect industry with the top medical professionals in the field of DS.
  • Offer a pathway to interaction with patients and families to hear about caregiver concerns first hand.
  • Assistance in establishing and recruiting for patient advisory boards.
  • Advocate during the regulatory review process.

Patient families are always willing and motivated to make time in their busy schedules when they know that their voice is being heard and their input and time will make a difference. This is why DSF chooses to engage with medical professionals, researchers, organizations, and industry who have shown that they genuinely care about our community. By partnering with others who show a sincere commitment to our cause and community needs only sets up everyone involved for success.