Accelerating time to treatment for rare diseases with modern, patient-centric data

Accelerating time to treatment for rare diseases with modern, patient-centric data

By Peter Stark, Veeva Compass

Rare diseases, as a whole, are not rare at all. Every year, people’s lives are upended by a diagnosis of one of 10,000 identified rare conditions, which collectively affect one in 10 people, according to the Genetic and Rare Diseases Information Center at NIH. A fact sheet from Global Genes, a global nonprofit dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally, states because a majority are genetic and appear early, half of these patients are children, and many are not expected to reach their fifth birthday.

For patients left waiting, the stakes for new medicines are high. Yet, less than five percent of rare diseases have at least one approved treatment, the European Commission noted in February 2022 in Horizon, The EU Research and Innovation Magazine. Even when effective therapies and medicines exist, reaching the right patients is challenging. Survey data depicts a long and emotional journey for those awaiting a diagnosis. For adults, it can take up to five years, and half will receive a misdiagnosis, say researchers in an Orphanet Journal of Rare Diseases article titled, “The diagnostic odyssey: insights from parents of children living with an undiagnosed condition.”

Rare diseases challenge traditional ways of commercializing new medicines. With speed so critical to finding patients who aren’t always easily identified, traditional data can slow the process because it does not provide a complete view into the longitudinal patient journey. Once a medicine is approved, it is also crucial to seamlessly transition from trials to treatment given the smaller patient cohort, as the physicians who conduct the trials often become prescribers.

The good news is that the industry is making great progress in how medicines for rare diseases are brought to market. From new approaches to data to greater connectivity between medical and commercial teams, companies are breaking down silos and cutting the time to treatment. Along with richer healthcare data and effective medical education for physicians on symptoms and treatment options, these advances will help those undergoing complex patient journeys.

Data unlocks effective commercial execution

Data is core to understanding where the greatest patient needs are. Biopharmas need more complete and precise data that they can trust. Knowing specific HCPs for a particular indication to target and prioritize to find the patients in need is a game changer.

One enterprise biopharma launching a new treatment for a rare cancer needed to understand varying patient profiles – those newly diagnosed or treated – and isolate diagnosed patients and treatment providers. This meant finding patients already on market-leading therapies, but who still aren’t visible in traditional datasets. The company not only needed data for specific cancers, but also on health-related events for this particular patient population.

With Veeva Compass Patient, the brand found 34 percent more net new patients and identified a significant number of net new HCP targets compared to when using other traditional claims data sources. With unlimited access to the data, it is now being used to fuel an AI/ML engine to identify possible predictors to the disease diagnosis.

Data at speed, for faster patient starts

Biopharma companies are connecting technology and data to reach more of the right target patient populations and facilitate collaboration between functions that have not worked together before so they can identify and engage relevant HCPs. More than a third of all prescribed brands in the past year are found in medical claims, including in-office administered therapies, injections, and infusions. The dynamics have changed, and biopharma companies no longer just follow the prescription, they can now see the complete patient journey of prescriptions and procedures. The time to access data accelerated from weeks or a month to a day or even hours, as pharma and HCPs need to move fast to get the right medicine to the right patient.

With new patient-centric data for modern medicines, commercial leaders can now have complete, timely visibility into prescriptions dispensed at pharmacies, along with treatments administered in office settings – an industry shift never seen before.

With a complete data set of the complex patient journey now available, faster treatment starts can become a reality for rare disease patients.

Peter Stark is executive VP and general manager of Veeva Compass.