Their diseases may be rare, but feelings of isolation are all too common among the relatively small numbers of patients suffering from orphan diseases. To help these groups connect with others, specialty pharma companies are launching online hubs tuned to the needs of the orphan community.

“We consider each of these patients to be as important as a patient with a much more common disease,” said Will Roberts, VP of corporate communications for ViroPharma, which markets the drug Cinryze. The biologic was the first product approved by the FDA for patients with the rare disease hereditary angioedema (HAE), a genetic disorder marked by attacks of uncontrolled swelling in various parts of the body including the hands, feet, face and throat.

Prior to approval of Cinryze in late 2008, Roberts said that patients with HAE largely felt alone. The firm launched a support program last year, Ryze Above, designed to dovetail with the services of the HAE Association of America, a patient advocacy organization based in Honolulu.

HAE afflicts only about 6-10,000 patients in the US, yet three products are approved for the disease, and more are in development. All three are supported by branded and unbranded educational websites, and while some of the marketers acknowledge the significance of social networking for these patients, they are awaiting FDA regulation on the topic. To help increase awareness of the need for earlier diagnosis, research and access to treatments, some of the companies are planning events around Rare Diseases Day this Monday, Feb. 28.

Cinryze is the only HAE drug approved for prophylactic use, as opposed to acute. Patients infuse the biologic every three to four days to prevent attacks, and Roberts said the goal of the Ryze Above program is to “build a sustainable relationship” with them and to offer emotional and other kinds of support.

“If we do our jobs with Cinryze, we are allowing these patients to have a normalized life without the fear of attacks,” he said. “We’ve designed this community to create ways for patients to share their experiences to make sure they are well represented, well taken care of and that they have all the information they need to maintain a normal lifestyle.”

Therese Heimbold, assistant director for Cinryze marketing, who conceived of the Ryze Above program, said it can “inspire and motivate Cinryze patients through stories from real patients, one-on-one patient support and relevant HAE resources.”

The program was designed by agencies Dudnyk and Cadient. Branded site, by Cadient, offers a therapy tracker, electronic reminders for doctor appointments, tips on living with disease, access to a journal to record attacks, and travel kits. It doesn’t have a social media component yet.

“Social media is a very important platform for ViroPharma programs; it’s something we will consider for this program, as well,” said Roberts, adding that the firm is awaiting FDA guidelines on the issue.

ViroPharma competes for attention against online HAE resources launched by the other two approved drugs in this disease state—IV infusion drug Berinert, for treating acute attacks in the face and abdomen, and Dyax’s Kalbitor, which is approved for acute attacks in all locations and is the only HAE drug that can be injected under the skin.

Berinert’s marketer, specialty company CSL Behring, offers personalized interactive tools through an unbranded site, Launched in 2007, the site’s resources include a journal patients can use to keep track of HAE attacks, as well as doctor appointments, medication, diet and exercise. A “Family Tree” section aims to help patients understand how HAE presents in families.

The Berinert Expert Network, a support program CSL Behring started last year, helps patients secure access to the drug and offers additional resources via a 24/7 phone line. Patients, caregivers and physicians do not need to register to access the information provided by either the Berinert Expert Network or

Dyax has developed two websites relevant to the HAE community—, a non-branded disease website designed to educate patients and create awareness, and Patients can register to receive newsletters, but this is required for only one feature—submitting an HAE-related question to an expert on

To keep content fresh, visitors are encouraged to submit questions. Personal success stories from Kalbitor patients are also featured on
“The main idea is to make the sites personal, applicable and interactive for our users,” said Paul Fletcher, director of marketing at Dyax. “Ultimately, we try to get insights from our users as to what information they would like to receive, and then we strive to create programs to make it available to them.”

He said Dyax is exploring several interactive features based on their needs, including social media as a means to communicate with patients and caregivers. “Of course FDA guidance will help inform these initiatives.”

Dyax has also used abruptive print advertising to draw attention to the condition and to online resources. Its journal ad targeting allergists and immunologists, designed by GSW Worldwide, won an MM&M Gold Award last year for depicting what an HAE attacks feels like.

ViroPharma requires patients to register in order to access online features but has not released the exact number of those who have. At the end of 2010, the firm said that more than 600 patients were taking Cinryze.

To drive patients to, ViroPharma uses personalized emails, direct mail and SEO, said Roberts. The reason for putting features behind a firewall has to do with demographics and the specific needs of patients, he said, as well as patient privacy. Care coordinators have access to patients’ phone numbers.

ViroPharma said the most heavily used part of its site is the section where patients can listen to other Cinryze patients share experiences. Updates to this section are planned for this year. Said Roberts: “We do know [users] identify with these stories and want to make sure that part of the site is as up-to-date and fresh as it can be.”

In the US HAE pipeline are Shire’s Firazyr, in Phase III testing, and Pharming’s Rhucin, which has been filed for approval. Both are already on the market in Europe and are designed to treat acute angioedema attacks caused by C1-inhibitor deficiency.