CBI Scene Blog

INFOGRAPHIC: Seizing a Rare Opportunity- A Current Snapshot of the Rare Disease Community

Posted by Logan Fleck on Sep 25, 2014 9:00:00 AM


People living with rare diseases face unique obstacles that stand in the way of their treatment. Because a rare disease affects less than 200,000 Americans, these diseases can go largely unrecognized – even amongst health care providers. Technological developments and the expansion of online communities are helping to raise awareness by providing new opportunities for networking and valuable resources for patients and professionals alike.

Tracking down participants for clinical trials can be difficult and trials often occur far from patients’ homes, but drug developers are beginning to use online sharing and social media to spread the word to target communities. Individuals within these communities are also spreading the word and helping to increase involvement in trials where new treatments are in high demand. Because only about 400 approved treatments exist for rare diseases, there is pressure for new medicines to be produced quickly and most development timelines are condensed. The drugs that meet FDA criteria are released and provide great relief to a small market. However, the shortened timeline can increase the opportunity for error to occur outside of the trial and limits the amount of time for observations to ensure a drug’s maximum efficacy.

Despite difficulties in drug development, communicating remotely with treatment providers has become much simpler and can help keep patients adherent to their treatment programs. Coupon and co-pay assistance programs also increase adherence by providing funds to cover therapy. These programs fall under the category of PAPs, or Patient Assistance and Access Programs, which offer a network of services that aid patients who are presented with boundaries that are prohibitive to their care. Some offerings, such as ancillary services, aim to localize treatment by helping patients to access important components like diagnostic testing from specialists like radiologists, physical and nutritional therapy, and nursing home care. Other offerings make local information available across the globe. HUB services keep healthcare providers and treatment professionals connected to patients and their charts and treatment information when they receive services at many locations.

Advocacy groups are also taking a global approach to connect all concerned with opportunities to facilitate research, shape public policy, build relationships, and raise funds. Social media has been particularly effective for leveraging involvement and awareness, with campaigns like the ALS Ice Bucket Challenge increasing donations drastically. The challenge raised 77.2 million dollars more during a month-long period in 2014 than the same period in 2013 before the social media sensation began.

The Internet proffers boundless opportunities to those who are afflicted with rare diseases by uniting individuals from around the world. Patients, their treatment providers, Health Care Professionals (or HCPs), patient organizations, and Key Opinion Leaders (KOLs) are all hosted online in diverse communities that offer support and expert opinions. Sometimes these communities simply offer a forum where a member’s challenges are understood and do not go unnoticed. By using new technology and online resources, advice and encouragement from experts can reach those who may not be fortunate enough to have access to such offerings close to home.

To find out more about how you can provide support those living with rare diseases in your community or online, join us at NORD's 2014 Rare Diseases & Orphan Products Breakthrough Summit, taking place October 21-22, 2014 in Alexandria, VA.

NORD 2014

Topics: Patient Access