Horizon Pharma focuses primarily on the research, development and commercialization of medicines that treat rare diseases, which are diseases that impact fewer than 200,000 people in the United States. Because so few individuals are affected by a single rare disease, working to better understand the nature of the specific disease as well as developing appropriate biomarkers, diagnostics and treatments is often very challenging and requires a network of academic, government and private physicians and scientists to work together alongside those few patients impacted by the disease.
Ninety-five percent of the more than 7,000 different rare diseases do not yet have an FDA-approved treatment. However, it is not sustainable or scalable to approach discovery one medicine or one disease at a time nor is it feasible for every institution to build the capabilities needed to be successful. Horizon Pharma works tirelessly to ensure that the patient perspective is the primary force driving the drug development process and regulatory decision-making.
As a company dedicated to meeting unmet medical needs, specifically in the rare disease space, Horizon Pharma understands and embraces the importance of increasing research efforts to develop therapies for some of the most vulnerable patient populations. “For us, it’s personal in that we work every day to put patients and their families first in every decision that we make including our goal to bring innovative therapies to market,” said Shao-Lee Lin, M.D., Ph.D., executive vice president, head of research and development and chief scientific officer, Horizon Pharma plc.
A common challenge for researchers and developers of rare disease therapies, Dr. Lin explained, is the small patient populations affected by these diseases. Improvements to the regulatory system/framework will be important to keep pace with scientific developments. For example, the ability to use novel methods in clinical trials, such as real world evidence and patient-focused drug development, may help get new therapies to patients faster.
“A robust research enterprise and strong investment environment affects everyone in the biomedical ecosystem,” noted Dr. Lin. “Guaranteeing well-funded federal research agencies and ensuring that policies are in place that encourage investment in the biopharmaceutical industry will allow innovative therapies to reach the patients that need them most.”
As Horizon Pharma actively seeks to address unmet needs in challenging and rare diseases, the company pursues opportunities to work across industry, academia and institutions to advance research and elevate the importance of funding to further innovative research.
“A major challenge in advocacy is making sure your voice is heard,” Dr. Lin said. “Because of this, it’s important to work with other organizations that share a common goal.” Horizon Pharma has partnered with a number of organizations, such as NORD and Global Genes, to advocate on behalf of the rare disease community. “We work with a number of umbrella organizations, such as Research!America and the Alliance for a Stronger FDA, to advocate for robust funding of America’s research enterprise,” Dr. Lin added.
For more information about Horizon Pharma, visit www.horizonpharma.com.