Situational Analysis — The Challenges Orphan Manufacturers Face
By definition, a rare disease includes fewer than 200,000 diagnosed patients, yet many of these diseases have only hundreds to a few thousand patients. Industry reports indicate there are approximately 7,000 identified rare diseases. Although many of these diseases have no therapies, the trend is improving owing to government incentives to pharmaceutical manufacturers to invest in these rare diseases. In fact, of the 44 new products approved by the FDA in 2015, 42% received orphan drug designation for rare diseases (see “Impact of the Orphan Drug Tax Credit on Treatments for Rare Diseases, Prepared for the Biotechnology Industry Organization and the National Organization for Rare Disorders,” June 2015, at https://rarediseases.org/assets/files/white-papers/2015-06-17.nord-bio-ey-odtc). Product approval is only the first hurdle. The commercial challenge for manufacturers is to find healthcare providers who manage these rare diseases and locate patients in need of orphan medications.
There are also many other challenges facing the orphan drug category:
- Multiple healthcare providers and specialties may be participating in a patient’s care.
- Patients often seek multiple opinions before gaining a proper diagnosis.
- Patients are geographically dispersed across the country.
- Many specialists are often not aware of new treatment options.
- Resourcing a field sales force is difficult and expensive.
Some of these patients have been afflicted with a serious but non-fatal condition for years. Others may die if they aren’t located and introduced to therapy within the first year. By locating patients stricken with such rare diseases and connecting them with the therapies they need, the result can improve a patient’s quality of life or even save that life. The obvious gap in this process is the cost-effective way to canvas the complex healthcare marketplace in search of physicians who diagnose, treat, and manage these rare patients.
TrialCard understands the complexity of finding patients with rare diseases. It takes time to learn about the brand and the disease state community. TrialCard deploys highly skilled pharmaceutical clinical specialist teams who are empowered to canvas healthcare systems in search of the healthcare professionals who manage these patients and connect them with the orphan drug manufacturer. The teams serve as healthcare detectives within complex systems.
To be successful, the detective model must include the following:
- The right team members with an incredible will and drive to save patients’ lives.
- A customized call flow that helps team members navigate offices and healthcare systems.
- Specific skills to listen and inform healthcare professionals to create deep physician engagement.
- The ability to act as a patient advocate and instill a spirit of polite persistence.
- State-of-the-art technology to track progress and report findings.
TrialCard approaches each and every opportunity from the standpoint of true partnership. The Case Study below illustrates how TrialCard configured a customized approach to meet brand, patient, and prescriber needs.
Case Study: Rare Kidney Disease
TrialCard was asked to search for patients with a rare kidney disease, which typically required them to have 10 to 20 or more surgeries in a lifetime. As with all genetic disorders, the condition has no cure, but there are available therapies that can help prevent the need for surgery, providing a meaningful quality-of-life improvement. Patient diagnosis of this condition is difficult, requiring a greater knowledge of science to successfully engage the healthcare professionals on the other end of the telephone. TrialCard customized a dual-model program, utilizing both healthcare service representatives and pharmaceutical clinical specialists. Healthcare service representatives were given a script and call flow, while the clinical specialists were given a list of leads in healthcare systems. Both teams were trained on TrialCard’s “Detective Model” and underwent a continuous team-sharing program to accelerate results.
In the first five months of the program, the TrialCard teams as a group identified more than 90 qualified leads of physicians who had patients presenting symptoms of this disease in their practice. Through the vetting process, several of those patients were connected to therapy. In this Lead Generation Program, the pharmaceutical clinical specialists found 72% of the patients, while the healthcare service representatives found 28%. TrialCard delivered patient counts six times higher than original client expectations. Notable in this program was the success of both teams in reaching a nurse or physician in between 30% and 50% of the calls made. These results show a more cost-effective orphan drug lead generation effort than traditional field-force initiatives, mass-marketing campaigns, or traditional event lead generation.
Finding physicians who treat rare diseases and identifying patients under their care that would be strong candidates for orphan drug therapies is by no means an easy task. It’s one, however, that the pharmaceutical clinical specialist teams at TrialCard have repeatedly proved successful in accomplishing. In doing the “detective work” to find and connect patients, providers, and therapies, TrialCard is proud to help deliver quality-of-life improvements to patients.