Managed access programs – also known as compassionate use, early access, and named-patient programs- have clear benefits for patients and physicians. These programs provide an alternative regulatory pathway to help patients access potentially life-altering drugs when they are ineligible for clinical trials or the drug has not been approved in their home country. Historically, the…Read More
Big data will never be an option for rare disease research. By definition, rare diseases affect around 620 people per million. Ultra-rare diseases may affect as few as 1 in a million. When you have a tiny population, scattered across the globe, how can you hope to find enough clinical trial participants to demonstrate efficacy…Read More
A study published in the Journal of Genetic Counseling provided evidence for something every rare disease patient and caregiver has learned in the school of experience. Alone in a Crowd? Parents of Children with Rare Diseases Experience of Navigating the Healthcare System 1 reported on the challenges rare disease parents face once their children are…Read More
Product launch planning has changed since my days of working in big pharma. Ten years ago, pharmaceutical marketing professionals focused on ensuring patients could access therapy early, even if they didn’t meet the clinical trial criteria. Early access to therapy, pre-launch and pre-license, offers benefits for all stakeholders. For patients, access to therapy can slow…Read More
In one way or another, the pandemic has impacted nearly everyone on the planet. But some groups have suffered more than others. Because COVID-19 preys on those with underlying health conditions, people with rare diseases have had to take extraordinary precautions to avoid contracting the illness. The threat of becoming infected has added tremendous stress…Read More
Along with its direct impact on human health and survival, COVID-19 is indirectly impacting health by causing many companies to hit the pause button on clinical trials. Hundreds of studies have been suspended, and many more are pushing out their start dates. The reasons are many: Site closures Risk of infection from visiting a medical…Read More
The lack of treatment for COVID-19 , which resulted in a decision by the FDA to allow expanded access and compassionate use for remdesivir and Emergency Use Authorization (EUA) for chloroquine, has brought renewed attention to these methods of getting treatment to patients with life-threatening illnesses when there is no approved drug. The FDA’s response…Read More
With so much pressure on health systems across the world in dealing with the urgency of the COVID-19 pandemic it is reassuring to know that a major health system like the NHS has the ability and agility to tap into alternative care pathways with home healthcare models to protect more lives, particularly those already coping…Read More
The COVID-19 outbreak has been exposing many of the existing cracks in the foundations of our healthcare system. But it is not just the lack of equipment, shortage of healthcare workers and hospital beds, and a missing plan for pandemics that are hampering our collective response. Some patients are dying not because of the virus…Read More
RareiTi and CysticFibrosis.com have come together during the COVID-19 pandemic, to find out directly from CF patients and their caregivers what impact the pandemic is having on them. We wondered what differences they are feeling and what we all can learn from this vulnerable rare disease community, who are already use to self-isolation and physical…Read More
The COVID-19 pandemic is having significant impact on clinical trials globally. Many trials face suspension or closure due to clinical priorities and the impact of social distancing and quarantine.
Clinical trial patients with a rare condition face great challenges; being part of a clinical trial when living with a rare condition can often be the only lifeline available, offering the chance to access novel therapies that could improve life enormously. During the pandemic, not being able to access novel therapies could have a major negative impact on health, disease progression and mortality. In addition, progress toward an approved therapy should not have to be completely stalled because of a lack of ability to collect relevant data.