Raremark, an online patient community geared toward people diagnosed with rare diseases, has introduced its Xperiome knowledge platform. The technology is intended to harness patient insights and data to bring novel therapies to market more rapidly.
Jeremy Edwards, Raremark CEO, told Outsourcing-Pharma that developing treatments for rare diseases is vital. An estimated 30m patients in the US alone are impacted by such conditions, with about 7,000 rare diseases found in the country, and only a few hundred of them currently have available treatments.
“Research is absolutely paramount for rare diseases, in most cases the disease is genetic and having effective treatments can vastly improve the length and quality of life for patients,” Edwards explained. “We know from speaking to our myasthenia gravis community – a neuromuscular disease that causes muscle weakness – that the treatment options available at the moment come with nasty side effects and in some cases make people feel worse than before they’d taken the medicine, so any research that can help improve their lives will be incredibly helpful."
Still, Edwards added, there is hope for many patients dealing with rare diseases.
“Luckily, for myasthenia gravis, there’s a few clinical trials taking place where researchers are hoping to find a better treatment for this disease,” he said. “In fact, we’re helping with a few of those.”
Additionally, Edwards said, increasing the amount of research performed regarding rare diseases, the more chances researchers have to help patients lead better lives. What’s more, he told us, listening to patient experience is important to that progress.
“Patients and carers sometimes know more about their disease than their doctor, because they are the ones combing through journals and dense medical literature, and learning from others through online patient platforms like Raremark or by setting up a Facebook group,” he said. “We have found that the biggest driver of change in rare disease is families.”
Tapping into Raremark’s network of more than 11,000 active rare-disease patients and caregivers, Xperiome is designed to bring together those affected by a range of rare conditions, including idiopathic pulmonary fibrosis, myasthenia gravis, and urea cycle disorder. By utilizing the platform, pharmaceutical companies reportedly will be better able to recruit and retain patients from populations that can be hard to reach, improve go-to-market strategies and evaluate the effectiveness of new treatments in a real-world setting.
Edwards added that enabling pharma companies to forge deeper connections with patients and better understand the rare diseases being targeted.
“It’s absolutely critical that pharma builds a strong and co-beneficial relationship with rare disease communities right from early phase development so that they can understand the rare disease and experiences of living with the condition fully – this will then help strengthen the impact of their clinical research and vastly improve treatment satisfaction,” he said.
According to the company, the Xperiome platform can inform pharma companies throughout drug development and commercialization, enabling them to connect with the right patients at the right time, complete missing datasets and unlock real-world insights.
