Rare diseases are conditions with a low prevalence which can be severe, life-threatening, and disabling. Due to an inconsistency in defining discrete disease entities and variation in incidence across countries or demographics, estimates of the number of rare diseases likewise vary – with estimates between 5,000 and 8,000. Rare disease drug development tends to be conducted in smaller biotech companies. Rare disease contract research organizations (CROs) can play a role in helping these smaller biotech companies in the rare disease drug development process by running more efficient rare disease clinical trials. The past 40 years have seen an increase in investment in rare diseases from both “traditional” venture capital and private equity sources, and philanthropic, patient-led, and social-impact-based sources highlight the growing consensus that rare disease treatments may lead to significant societal benefit as well as the role of “moonshots” that have paved the way for more economically viable models for drug development.
In this article, we highlight CROs in San Francisco, California, from small startups to large global players, including an Asia Pacific CRO which has increased its presence in the U.S. to meet the rising demand for rare disease CRO services.
AllStripes is a healthcare technology company, co-founded in 2017 by CEO Nancy Yu and technology developer Onno Faber, dedicated to developing treatments for people with rare diseases. AllStripes created a technology platform to generate regulatory-ready evidence to accelerate rare disease research and drug development. The AllStripes patient application aims to help patients contribute to new treatment research by participating securely online from home. The CRO partners with rare disease patient advocacy groups and key opinion leaders to create their programs, and works with families, experts, academic institutions, and pharmaceutical companies to advance research. AllStripes advocates real-world evidence in drug development and builds tools that make research more inclusive for the global rare disease community. The CRO is committed to transparent data collection and sharing data across the life sciences continuum to advance clinical research. It is listed as one of the Top Fully Remote Biotech Companies 2023 by Built In, an online community for startups and tech companies. In 2021, AllStripes announced the completion of a $50 million Series B financing round with the goal of launching 100 rare disease research programs, expanding its global operational footprint, introducing tech and data automation enhancements, and investing in its team.
Headquartered in Burlington, North Carolina, LabCorp has a presence in San Francisco. The CRO leverages its analytical tools to access real-world LabCorp diagnostic lab result data, global Central Labs investigator performance data, and patient intelligence input to access, enroll and retain patients for rare disease clinical trials. It maintains relationships with leading advocacy groups and provides support and education for study teams, sites, patients, and their families. To facilitate the collection of key clinical data, LabCorp offers biomarker discovery/development, mobile health, virtual natural histories, and virtual rare disease clinical trials. Sponsors have access to 120 genetic counselors and more than 5,000 assays, including relevant biomarkers. LabCorp has supported more than 50 orphan indications and 103 rare disease studies globally. Understanding that the commercialization of orphan drugs has unique challenges, LabCorp generates real-world, value-based evidence to support market access strategies.
Novotech is an Asia Pacific (AP) CRO with a presence in Europe and the U.S. Since its expansion to San Francisco in 2017, Novotech has increased its presence in North America to meet the rising demand of U.S. biotech companies for CRO services in AP. It estimates that 30 million people are affected by rare diseases in the EU and in North America, while over 45 million suffer from a rare condition in Asia. In recent years, Asian countries have been implementing new legal frameworks to accelerate the registration and approval of rare disease drugs with new rules concerning health insurance coverage, expedited registration, and marketing exclusivity periods. Novotech is a full-service CRO with experience in enrolling rare disease patients. It offers expertise in site selection, access to patient registries, and global regulatory knowledge specifically for Rare Disease trials and Orphan diseases trials. Novotech has conducted over 20 trials in rare disease indications.
Vial is a next-generation CRO headquartered in San Francisco, with a mission to reimagine clinical trials. Vial is delivering faster, more efficient clinical trials that enable scientists to develop effective therapies to improve patients’ quality of life, slow disease progression, or even cure the disease.
Today, many clinical trials are highly inefficient due to misaligned incentives and antiquated processes like manual data collection on paper. Vial is working to change this through their technology platform that leverages connected systems and intuitive design to run global trials efficiently at scale. The platform integrates trial onboarding, patient enrollment, site communication, and data collection processes into one connected system, dramatically lowering costs for biotech sponsors.
The Vial Rare Disease CRO enables site startup in under 30 days, digitized, automated data capture through eSource, and accelerated enrollment via the recruitment platform. In addition, Vial offers sponsors fixed-pricing agreements (with no change orders) to align incentives, provide transparent budget visibility, have no unexpected charges or fees, and share accountability on commitments, ultimately building trust and deepening the relationship with sponsors. For more on the Vial Rare Disease CRO, click here.
Headquartered in Wilmington, North Carolina, PPD has a presence in San Francisco. The CRO’s rare disease clinical trial services include virtual rare disease trials, patient-centered rare disease research, and the PPD Rare Disease and Pediatrics Center of Excellence (COE), which focuses on rare and pediatric indications to shape trial design, strategy, and delivery. The CRO’s aim is to decrease burden and increase flexibility for patients and their families. Virtual rare disease trials enhance the patient experience with minimal delays and inconvenience while keeping patients engaged through a dedicated resource. PPD maintains relationships with patient advocacy groups, academic institutions, healthcare providers, and pharma industry groups to better understand the patient journey and help alleviate patient burden. PPD has dedicated rare disease expertise in neurology, metabolic, and neuromuscular clinical research development. In the past five years, PPD has worked with rare disease drug developers across all phases, indications, and therapeutic areas. PPD has supported 545 rare disease studies in 17,975 sites with 106,325 global patients across 101 countries.
With an understanding of the challenges specific to rare disease clinical development and commercialization, Syneos Health aims to offer innovative solutions to support rapid access to treatment options for patients with a rare disease. Syneos Health works with multiple stakeholders, including patients, advocacy groups, key opinion leaders, payers, and regulators. Through the Syneos Health consortia model, rare disease clinical trial services are provided through the Syneos Health Rare Disease Consortium. This includes access to its therapeutic expertise in specific rare disease indications, Pediatric Consortium, and partnerships with patient advocacy groups, highly specialized vendors, site advocacy groups, and rare disease organizations. In addition, sponsors can avail themselves of regulatory consulting, commercial consulting, real-world evidence, and biostatistics and data management services. In the past five years, Syneos Health has conducted over 350 projects in rare disease indications, including more than 30 real-world evidence projects.