Achondroplasia is a very rare disease, with fewer than 20,000 cases per year in the United States. It is a disorder of bone growth that prevents the changing of cartilage, particularly in the long bones of the arms and legs to bone. It is characterised by dwarfism, limited range of motion at the elbows, large head size (macrocephaly), small fingers, and normal intelligence.

The client was conducting a a pharmaco-epidemiologic study to characterise the impacts and burden of Achondroplasia across the lifespan. The study wasfailing to meet enrollment goals and feedback from sites was that patientswere not interested in participating in the registry. The sponsor engaged ICONto uncover patient insights related to recruitment challenges, barriers. They specifically sought to understand what would motivate patient to participate in the research, and to apply those patient perspectives to address the enrollment and retention challenges.

ICON conducted extensive secondary research including social listening, review of existing study materials, and identification of global patient advocacy groups (PAGs) supporting the Achondroplasia community.

Building upon learnings from secondary research we completed global in-depth interviews with KOLs, PAGs and patients understand the journeys and needs of this patient population. Additional research objectives were to:

• Identify key moments in time to send outreach materials to patients
• Pressure test existing materials from a PAG and patient POV
• Identify barriers to enrollment related to recent protocol amendments requiring re-consent
• Uncover ways to motivate patients to remain engaged in the study long-term

Our research identified a common theme among patients in the Achondroplasia community, namely that this is a condition that is not central to their definition of self. Patients reported that they did not want to be continually reminded that they have this condition. They also reported that materials were not only failing to engage them, but in many cases operational problems resulted in the wrong materials being sent to patients. For example, adult patients received coloring books meant for young children.

We also identified two key PAG challenges. First, patients were not engaged with PAGs as they entered adulthood. Second, the sponsor needed to engage directly with PAGs to help drive awareness.

As a result of these robust patient insights, we worked closely with ICON teams to develop a patient outreach strategy that leveraged lexicon, themes and imagery directly from conversations with patients.

We recommended and collaborated on the development of a social media plan to reach a hard to reach age group within this population, patients from ages 20-40.

We developed high-value partnerships with global PAGs supporting the patient population. In partnership with these groups we developed strategies that PAGs could leverage to drive awareness among patients and health care providers.

Although the Sponsor chose initially not to move ahead with the recommended strategies and materials, failure to meet enrollment goals resulted in the sponsor re-engaging with ICON for additional assistance more than one year later.