How do you motivate patients to adopt a new medicine and comply with its demanding dosing schedule, when free care has been available for 20 years? SmithSolve made it easy for patients to transition to commercial therapy. Working with patient organizations, we developed and shared educational tools on the disease, access programs and compliance. The clinical benefits of treatment are now crystal clear to this rare disease community, and product adoption exceeded expectations.


In patients with cystinosis, crystals accumulate in the eyes, causing vision problems such as light sensitivity, corneal damage and blurred vision. Treatment consists of eye drops taken every waking hour, previously available only through a free clinical trial that spanned two decades. With the launch of Sigma-Tau’s new product, patients with this rare disease finally gained access to an approved treatment. SmithSolve was tasked with easing the transition for patients to commercial therapy and encouraging them to comply with the dosing schedule



Our work included:

  • Worked with Sigma-Tau, the National Institutes of Health, the Cystinosis Research Foundation, the Cystinosis Research Network and other patient advocacy groups to spread the word about the product launch
  • Prepared launch updates for Sigma-Tau to present at patient meetings
  • Created an access infographic showing the steps to secure access
  • Developed news releases and fact sheets on the disease and its treatment
  • Briefed patient organizations and equipping them with educational tools to share through websites, newsletters and meetings
  • Produced a video on compliance issues that featured interviews with physicians, caregivers and patients for peer-to-peer education


  • Exceeded product sales expectations as clinical trial patients transitioned to commercial supply
  • Alerted all targeted patient organizations’ members, shared educational tools and posted access resources
  • Secured praise from patient organizationsu for bringing news and tools directly to the community
  • Generated nearly 1,200 views of the video in the first three weeks in this community of 500 patients
  • Exceeded Cystaran sales expectations as patients adopted and continued on therapy

Living with Cystinosis: A Closer Look



“You fit it into your life. You fit the disease into your life and make it work. Whatever that means is up to you as an individual and family, but you have to fit the disease into your life. Do not let it run your life. This is exciting and a wonderful thing to be able to see the progress and live the progress. We really are. Well, she is.”

Marybeth Krummenacker
Caregiver and advocate