Interview: The Family Journey of Early Access Care

Caregivers and their families are at the center of what we do at Early Access Care. Working with Patients and Patient advocates is at the heart of developing real world programs that support patient access to investigational unlicensed medicines. Often, it is the patient or caregiver who informs and educates on rare diseases and how a treatment may directly impact them. Increasingly, patient advocates, both patient and caregivers, are spearheading the movement of increased awareness, and urgency to find biomedical solutions for the treatment of rare diseases.

Patient advocates are actively sharing the need for urgency, hope and action. Early Access Care actively supports compassionate use in rare disease medicines. Our staff come from backgrounds of clinical development of investigational molecules, operationalizing clinical trials, managing regulatory interactions, and characterizing the safety profile of compounds. At Early Access Care, our staff have a nurturing approach to both investigational medicine and patient. Our world is in the middle, bridging patient to investigational medicine. Our Team members support patients and caregivers, viewing each as their own loved one. Relationships grow with patient and their families, one patient at a time. Our team has been supporting one such family for five years and watched the miracle of a new biological medicine and the science behind it, bring new hope, save the life of a child, and change the reality for one family.

The mother and father of that child have generously shared the details of their family journey, the behind-the-scenes story and insights. While we have many chats with the mother, one thing that recently came to light was the communication sent by the pharmaceutical company to the physician, in 2019, informing the physician that his patient was approved for treatment and someone from the Early Access Care team would be reaching out shortly with all the details. And one week later the first infusion was being administered. At Early Access Care we act with urgency for each request. This was he case for this little baby. Every day was critical to stop further decline. The mother, Petra, is joining Early Access Care as part of a panel, to share in her family journey, what they learned, and the treatment that changed their lives.

We’re going to share more of Petra’s story about the day of the diagnosis of the rare disease with few treatment options. The treating physician had an idea, he said there are no guarantees, but there might be a chance that the pharmaceutical company would be willing to provide the biologic medicine through compassionate use. If you are able to join the conference, Operationalize Early Access Care programmes Summit Europe, in London October 8, 2024.

We are honored to deliver a session on the patient journey at the Europe EAP Summit to hear about the remarkable 5-year journey of one family. Join Early Access Care, LLC at the Summit to hear their story. We will continue to bring you the interview with Petra following the conference.