Maria Picone

Founder, TREND Community

Maria Picone is the Founder and President of Formed, Inc, a health-focused technology company located in Philadelphia, Pennsylvania. She founded Formed with her husband, Chris, in 2007. After their daughter was born with Prader-Willi Syndrome, Maria and Chris built TREND Community to give patients and their caregivers a platform to tell their stories and share anecdotal data. TREND Community empowers its users to become citizen scientists and actively participate in the search for their own treatments and cures.

TRNDS Speaker Spotlight

Why are you participating in TRNDS 2017?
I am participating in TRNDS 2017 to speak about how we are using our analytics technology and targeted initiatives to incorporate an amplified patient voice into all aspects of the drug development process and drive community-powered science.

What do you see as the biggest opportunities to accelerate rare disease therapeutic development?
With little to no evidence base for treatment options, rare disease patients and caregivers are turning to social media to learn about their diagnoses, share their stories, and seek answers and support from others who understand what they are going through. There is an opportunity to use this data to capture patient and caregiver insights and experiences on living with rare disease to accelerate rare disease therapeutic development.

What are some potential applications of technology in rare disease research?
The output from our analysis of social data can be used to:

  • Capture the patient’s and community’s voice,
  • Design hypothesis-generating proof-of-concepts,
  • Inform clinical trial design,
  • Contribute to natural histories,
  • Assist researchers and FDA in choosing clinically meaningful endpoints,
  • Inform pharmaceutical companies for focused drug development,
  • Identify candidates for clinical trials, and
  • Track outcomes.

What are the top 3 priorities for rare disease research in the 21st century?
As a parent of a child living with a rare disease my top priority is to give every individual an opportunity to be heard so that they might benefit from therapeutic breakthroughs in their lifetime. Absent therapeutic treatments and cures, my next priority is to empower all patients and caregivers to find solutions to their biggest challenges and improve their quality of life. Finally, I hope that our technology will give the undiagnosed a path to diagnosis.

Tell us about how TREND utilizes analytics and community science to advance efforts in the rare disease realm.
Currently our efforts are focused on three areas:

  1. Collecting evidence of the patient perspective. We have partnered with the Friedreich’s Ataxia Research Alliance to analyze Facebook group discussion data to provide the community’s perspectives on the questions posed by FDA for FARA’s patient-focused drug development meeting.
  2. Driving community powered science. Using our analytics on social data (patient stories, discussions, and journals) we are identifying unmet needs of rare disease patient communities and designing TREND Community Health Initiatives to empower participants to find solutions to daily challenges. As an example, we partnered with a world renowned ketogenic specialist, Beth Zupec-Kania of the Charlie Foundation, to explore the effectiveness and benefits of the ketogenic and related diets for those living with Prader-Willi Syndrome (PWS) and Angelman Syndrome. A renowned investigative science and health journalist is using this data to assess the potential for an obesity study in the PWS population, demonstrating TREND Community’s potential to drive research.
  3. Searching for therapeutic options. We are working with our rare disease communities to discover promising treatments. As an example, we partnered with the Chion Foundation to facilitate requests to FDA from individuals in the PWS community for personal importation of a drug, Pitolisant, approved in Europe to treat narcolepsy. Five PWS children have received this medication through this process and TREND is capturing patient experiences to share with all stakeholders.