In a recent column in Medical Marketing & Media, the founder and president of a digital relationship marketing firm with a long history in assisting those searching for remedies for rare diseases offers advice on how pharmaceutical companies could profit from the experience of those working on so-called “orphan” drugs.
Siren Interactive president and founder Wendy White notes fully a third of the novel drugs approved by the Food and Drug Administration last year are orphan drugs, treating rare diseases which each affect no more than 200,000 people in the U.S. The almost 7,000 rare diseases, however, collectively affect about 10% of the U.S. population. Since 80% of the conditions are genetic in origin, they are very likely to affect children. Only about 5% have an FDA-approved therapy.
A frequent author and speaker on rare-drug issues, White begins by noting that fully a third of the new drugs approved by the Food and Drug Administration last year were for rare remedies, and more are in the pipeline for expedited review.
In fact, two of the nine novel orphan drugs cleared by the FDA in 2013 practically set speed records: Genentech’s chronic lymphocytic leukemia drug Gazyva won approval in just a little over six months, and Janssen’s mantle cell lymphoma drug Imbruvica cleared the agency in just four-and-a-half months.
That record of success is partly due, White notes, to government incentives for rare-drug remedies, and to their profitability (in a market estimated to hit $127 billion by 2018, their return on investment is 1.7% higher than for conventional drugs, White notes, citing a 2013 report.) But, she adds, credit should also go to lessons those drug makers have learned that the overall pharmaceutical industry would do well to heed.
White points to five lessons learned that have benefitted orphan drug makers. First, she notes, large drug firms sometimes make the mistake of viewing their operations as compartmentalized, seeing clinical trials as separate from their manufacturing, marketing and other operations.
But rare drug firms, on the other hand, have discovered that, in White’s words, “brand building begins long before there is a brand.” So it’s crucially important for pharma firms to engage with affected communities early on, while planning and running clinical trials. What they do there in areas such as sharing data, providing drugs for compassionate use, and speedily reimbursing trial participants will do much to impair or enhance their reputation.
Orphan drug makers have also learned the importance of using social media in reaching close-knit rare drug communities. By failing to reach out through these widely shared media to tell their story, uncommunicative drug firms virtually guarantee someone else will tell it for them.
To gain maximum support, rare drug makers have also become expert in providing personalized support to those affected by the diseases they research. Leveraging their information and resources, they get involved in creating collaborative ways to educate the community and the public, and to help deliver services. By extending their view beyond the laboratory, such efforts pay dividends by building trust and cooperation, especially in times of trouble (as an example, she cites Genzyme’s active involvement with patient communities as helping the company weather some past supply problems).
Finally, White adds, rare drug companies recognize that patients look for innovations that improve their care and quality of life, and working together with patients, support groups and other stakeholders is the most effective way to achieve those outcomes.