Genetic AllianceGenetic Alliance

Therapy Development Webinar Series

Overview

When our CEO, Sharon Terry , came into the advocacy community in 1994 after the diagnosis of her children, there were no advocacy organizations venturing into the therapy development space. Advocacy organizations were designed to provide patient support and clinician education. There was, in fact, a memorable moment at a major organization’s advocacy meeting where she stood - wearing her PXE International hat - and proposed that advocacy organizations become the drivers of research. She was derided by the leadership and asked to leave the meeting. While shocked and hurt, she was undeterred and pursued building tools and resources to enable all advocacy organizations to take the reins on needed therapies.

Now, 28 years later, we advocacy organizations not only have a place at the table, but we create the table and set the menu. During these years, Genetic Alliance created dozens of easy-to-use tools to help communities drive research and development. We have also identified like-minded partners who share our principles and values. Several of those partners have useful opportunities for you.

  • Luna, the technology company that underpins all of Genetic Alliance’s registries and studies, is offering the platform to advocacy communities to manage registries and studies, and empower you to gather and analyze your group’s health and wellness information with simple, fast onboarding in days, not weeks.
  • EspeRare, our sister nonprofit, offers novel approaches to therapy development for rare diseases. EspeRare will offer start-up resources and funding to develop one new therapeutic program addressing high unmet medical in collaboration with an advocacy organization. A call for proposals will be launched to award this joint effort.

In this webinar series, we will start with the map we drew many years ago of the therapeutic development process. We will walk you through the map and its associated resources. Then we will dive in on a couple of resources we think can help you serve your members and advance research. Finally, throughout the series, we will point out free resources to support advocacy efforts.

Webinars

Navigating the Ecosystem of Translational Science (NETS)
November 29, 2022 • 9 AM PT/12 PM ET/6 PM CET
View the webinar here

When Sharon Terry first started considering how she was going to accelerate research on her children’s disease, pseudoxanthoma elasticum (PXE), she was lost in the maze of terms and processes that are part of discovering and developing a treatment. “Even though I had no science training, I quickly realized that the typical chevron diagram of a couple of linear steps was not representative of the process of moving from basic science to a therapeutic or device.” With the help of key knowledgeable people, Genetic Alliance created the NETS map, including an online version that connects to many of the open-source resources available.

NETS was interesting to the National Academies of Medicine Drug Forum, particularly Chris Austin, then director of the National Center for Accelerating Translational Science. That forum examined the map and revised it slightly, and created a second map for biologicals. Therefore, three peer-reviewed papers describe the map.

In this webinar, we will tour the map, visit the various neighborhoods, and discuss how best to advance the science in each area. You can visit the map here . You might also want to look at one or more of the papers, which you will find here .

~Sharon Terry, president & CEO, of Genetic Alliance and PXE International

Disease InfoSearch and GaugeRx
December 14, 2022 • 9 AM PT/12 PM ET/6 PM CET
View the webinar here

Newly diagnosed individuals must find support and information for participating in research. Disease InfoSearch is a consumer-facing directory of more than 10,000 diseases, with up-to-date information about current support and research curated by advocacy organizations themselves. Each advocacy organization gets to decide what is offered about their disease. Gauge RX is a brand-new tool of Genetic Alliance that will launch in 2023. The data is very useful for advocacy organizations to attract interest in their readiness for research by academics and pharmaceutical companies. However, it is not accessible because reading 100 data points for each disease is too onerous. We have conceptualized an algorithm that will distill the data into four domains. The quantitative data will be given a score, and it can be rendered more qualitative in the transformation. Thus, a “gauge” will make it much simpler for communities to see where they might focus so they can position themselves to accelerate therapy development, and the industry can assess which diseases and their associated organizations are ready for the investment of time, money, and attention.

~Sharon Terry, president & CEO, Genetic Alliance and PXE International

Repurposing Drugs in a Novel Way: EspeRare
January 18, 2023 • 9 AM PT/12 PM ET/6 PM CET
View the webinar here

Hand-in-hand with patients and the communities that represent them, EspeRare is a nonprofit that rescues and repositions therapies in rare diseases, thus accelerating the development of accessible treatments for these underserved patients. Genetic Alliance has worked with EspeRare since its founding. This foundation has a great deal to offer communities – from consultations about how to stay in the driver’s seat when negotiating terms and IP, to bringing a molecule off the shelf of a pharma company through clinical trials.

The webinar will focus on presenting the foundation, its patient-center model and its drug development approach that relies on complementarities and partnerships with the rare disease community. EspeRare will describe how shelved drugs can accelerate the development of life-changing therapies and lay out the practical examples of its programs in X-linked Hypohidrotic Ectodermal Dysplasia and Duchenne. Finally, through its call for proposals that will be launched during the webinar, EspeRare will encourage its audience to engage and co-develop a new therapeutic opportunity for its community.

~Caroline Kant, the co-founder and CEO of EspeRare & Sharon Terry as the president of EspeRare.

Community Driven Innovation™
February 15, 2023 • 9 AM PT/12 PM ET/6 PM CET
View the webinar here

As advocacy leaders, we know there is a great deal to do and not enough time. We also know that serving our members is our north star. Discovering our group’s priorities, understanding interconnections, and forecasting needs with the power of real-world data is critical and possible now. Luna will describe their Community Driven Innovation™ (CDI) methodology that optimizes and humanizes technology and techniques to uncover real-world priorities. CDI allows a deep understanding of a population’s critical priorities aligned to the outcomes that matter most. It focuses attention on areas that are being underserved, so you don’t waste emphasis on misunderstood priorities. CDI enables researchers to make informed decisions on the goals of their studies and trials to ensure the participants’ most valued outcomes.

~Ian Terry, Senior User Experience Researcher, LunaPBC, and Kaitlyn Esposito, Programs & Research, Bobby Jones Chiari & Syringomyelia Foundation

Registries and Studies
March 1, 2023 • 9 AM PT/12 PM ET/6 PM CET
View the webinar here

Genetic Alliance has offered registries for all since 2003, building on the PXE International lay-created and led registry. Over the years, we became ever more sophisticated and always grounded in these principles:

  • Individuals control their data and have simple options to allow them to share it with their registries in compliance with domestic and international consumer data privacy laws
  • Communities are excellent stewards of individuals
  • Registries and studies should be as inexpensive as possible
  • Clinical studies and trials should be convenient for participants, and advocacy organizations should have a leadership role in them
  • The highest data quality standards must be upheld so that data gathered is of a standard acceptable to all your research partners
  • Data governance must be guided by what people need and want as the priority, not in order to cover an institution’s liability
  • Diversity in participation and variety of data types is key for robust research, including PRO, -omics, health records, etc.

In 2019, we partnered with LunaPBC to provide a new, innovative registry and studies platform to our communities that aligns with all our principles. The Luna platform enables your registry with limited fees, broad data collection capabilities (including custom PROs, validated instruments, connectivity to 98% of the US’s electronic health portals, and genomic data capture), and no waiting – you can start your registry tomorrow. This webinar will demo major features and answer your questions.

~Dawn Barry, president and co-founder, and Deb Thompson, Vice President of Strategy and Operations of LunaPBC