Five things big pharma can learn from the rare-disease community

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Wendy White, founder and president, Siren Interactive
Wendy White, founder and president, Siren Interactive

Orphan drugs are a success: One-third of 27 novel new drugs approved by the FDA in 2013 were orphans. They are more likely to be expedited: Genentech's Gazyva, for chronic lymphocytic leukemia, was approved in 6.3 months, and Janssen's Imbruvica , for mantle cell lymphoma, in 4.5 months. They are profitable: with 1.7% greater ROI than non-orphans*, the worldwide market is projected to reach $127 billion by 2018.

Why? Government incentives help. More importantly, companies in the rare disease space have learned lessons big pharma can leverage.

1. Patient Engagement Starts in Clinical Trials

Internally, you may have different divisions responsible for research, manufacturing marketing and med affairs, but to patients and caregivers, it's one company. Your reputation is built on every encounter, and brand building begins long before there is a brand, with the patient experience in clinical trials. Participants care about your stance on compassionate use, your willingness to share data, and how quickly they are reimbursed for their expenses.


Click on the image at the left to view Journey into the Unknown: The Search for Rare Diagnosis, an infographic that is Siren Interactive's latest effort in an educational series on rare disease diagnosis and the resources available for those seeking a diagnosis.

2. Silence Isn't Safe

There's a reason so many social-media firsts occur in this space—the strength of rare-disease communities and their hunger to connect and learn from each other. Patient opinions are powerful and will be shared, positive or negative. Patients go online to compare experiences, evaluate guidance of their physicians, learn from mistakes of others and weigh their options. Social media is essential. If you don't tell your story, others will tell it for you.

3. Support Must Go Beyond the Brand

Just as orphan-drug companies are leading the way in personalized medicine, the same is true in personalized support. They excel at providing disease information and resources, and supporting access, delivery and education. They don't claim to be the authority on what it's like to live with a rare disease; they ask patients. They engage them in creating resources that will be useful to others. Treat patients with respect and it will be returned.

4. Don't Wait for a Crisis

Companies that are loyal supporters, and maintain healthy relationships by working to understand and meet the needs of patients and caregivers, will find that the community will stick with them if and when a problem occurs.

When virus contamination forced Genzyme to close a facility that produced treatments for Fabry and Gaucher disease, the resulting shortage created a health risk for many patients. However, the close, transparent relationship the company had forged with the patient communities enabled Genzyme to weather the storm.

5. True Innovation Begins with Outcomes

Process improvement is not the same as innovation. From the patient's viewpoint, innovation only matters if it leads to better care and improved quality of life. Where these goals are being achieved, it's because communities and companies have learned that the more stakeholders involved, the better the outcome.

Rare-disease communities are experts at collaboration. As changes in the healthcare environment bring new challenges and technology provides new opportunities, they turn to their partners to help them advocate and innovate. What you can learn from them is this: The only way we'll get there is together.

*Orphan Drug Report 2013, EvaluatePharma

Wendy White is founder and president, Siren Interactive

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