RareiTi VIEW

Managed Access: A Solution with Benefits for Everyone

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…

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It’s Not Big Data That Matters – it’s Broad Data

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…

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Rare Shouldn’t Mean Alone

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…

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Managed Access: A Missed Opportunity for Orphan Drug Developers and Patients

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…

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Rare Disease, COVID-19 and the Need for Mental Health Coaching

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…

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Keeping Drug Development Moving – through COVID-19 and Beyond

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…

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Real World Evidence as part of a Managed Access Program

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…

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Reducing Deaths from COVID-19: What Can We Learn from Rare Disease?

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…

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For the Cystic Fibrosis Community: Self Isolation is NOT the “New” Normal!

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…

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COVID-19 Response

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.

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