Biocom Brings California Companies to Nation's Capital to Advocate for Rare Disease Research

Biocom, the association representing the California life science industry, concluded its 2020 Advocacy Fly-In to Washington, D.C., this week, connecting research companies to legislators and regulators for the sixth consecutive year. This year's participants advocated for advancing research and development (R&D) for rare disease products, just a few days after Rare Disease Day.

"More than 300 million people live with a rare or orphan disease, which is defined as a disease or condition that affects fewer than 200,000 people worldwide," said Joe Panetta, president and CEO of Biocom. "Of the 7,000 known rare diseases, only 600 have a treatment or cure. Because rare diseases affect very small populations, it is extremely complex, costly and risky to develop orphan therapies. At Biocom, we are dedicated to supporting the work of rare disease research companies, advocating for federal programs that incentivize orphan drug development and ensuring that patients have access to the most innovative therapies."

Biocom 2020 Fly-In participants advocated for protecting the Orphan Drug Act of 1983 (ODA), which provides incentives to innovators for developing products with orphan designations. According to the National Organization for Rare Disorders (NORD), only 34 therapies for rare diseases existed before the law's passage; as of 2019, there were more than 770 therapies available to treat rare diseases. Yet, in 2017, Congress reduced the Orphan Drug Tax Credit by 50 percent. Participants also learned about programs at the Food and Drug Administration (FDA) and National Institutes of Health (NIH) that provide resources, assistance and funding for orphan product R&D.

"Biocom's Fly-In to Washington, D.C., was a great example of how stakeholders in the rare disease community work together to advocate for the continued advancement of R&D that can make a difference in the lives of people living with a rare disease," said Bill Rote, Ph.D., senior vice president of research and development at Retrophin (San Diego). "It was an honor to participate in this event, and we are grateful to the legislators, regulators and researchers who spent time discussing some of the key issues that will shape the future development of therapies for rare diseases."

"As a startup with limited resources, we immensely value the knowledge and connections that we wouldn't have access to without Biocom's support," said Je Min Yoo, regional director at BioGraphene (Los Angeles). "Biocom's Fly-In gave us the rare opportunity to get an insider's look into how the regulatory and legislative processes affect the development of novel therapies and establish relationships with high-level officials at the FDA, NIH and Congress. On-the-ground advocacy is extremely important to the rare disease research community, as we need supportive policies to incentivize the development of orphan products."

Biocom 2020 Fly-In participants had the opportunity to meet with officials at FDA, including at the Office of Orphan Products and Development (OOPD) and the Rare Diseases Program in the Office of New Drugs (OND) at the Center for Drug Evaluation and Research (CDER), as well as at NIH, including at the Office of Rare Disease Research (ORDR) at the National Center for Advancing Translational Sciences (NCATS) and the Small Business Education and Entrepreneurial Development (SEED) Office. They also met with Congressmen Mike Levin and Scott Peters of San Diego and Congressional staff from the offices of California Senator Kamala Harris and Los Angeles Congressman Jimmy Gomez.

About Biocom

Biocom is the leader and advocate for California's life science sector. We work on behalf of more than 1,300 members to drive public policy, build an enviable network of industry leaders, create access to capital, introduce cutting-edge STEM education programs, and create robust value-driven purchasing programs.

Founded in 1995 in San Diego, Biocom provides the strongest public voice to research institutions and companies that fuel the local and state-wide economy. Our goal is simple: to help our members produce novel solutions that improve the human condition. In addition to our San Diego headquarters, Biocom operates core offices in Los Angeles and the San Francisco Bay Area, satellite offices in Washington, D.C. and Tokyo, and has a continuous staff presence in Sacramento. Our broad membership benefits apply to biotechnology, pharmaceutical, medical device, genomics and diagnostics companies of all sizes, as well as to research universities and institutes, clinical research organizations, investors and service providers.

For more information on Biocom, please visit our website at www.biocom.org. Connect with us on LinkedIn, Facebook, and Twitter (@BIOCOMCA).