Skip to content
Visit our COVID-19 Response Center for all our updates Read more

Nareda Mills, President of Ashfield Patient Solutions US and Sam Falsetti, PhD, Head of Medical Strategy and Product Innovation at Cambridge BioMarketing talk to Pharmaceutical Commerce on the topic of patient support for the rare disease community.

The following is an expert from Pharmaceutical Commerce, read the full article here.

Diagnosing and treating a rare disease is difficult on a good day. Length of time to diagnosis (often more than five years), lack of therapies (there are effective therapies for less than five percent of 7,000 known rare diseases) and small patient populations all lead to significant challenges for the healthcare industry in rare disease management and support of these patient populations.  Even more so than those suffering with common illnesses, rare disease patients are as unique as the disease with which they suffer, making patient support even more challenging. Not only do they require a more personalized approach, but the industry must also cope with the fact that these patients often self-educate about their condition, putting them ahead of the curve in understanding their illness. Beyond education, patients’ deep knowledge of their condition makes them prime advocates for their community’s needs, such as assistance with FDA approvals for orphan drugs, fundraisers for clinical trials, and more.

Meeting the challenge of rare-disease patient support

The touch points in addressing rare disease are the same as in common illness, just the complexity of messaging and degree of importance each touch point takes may vary greatly. The industry must not just support, but educate the patients and their caregivers, the health professionals tending to these patients, and other key stakeholders such as patient HUB service providers and payor groups. Payor groups may not have developed protocols for coverage of these rare diseases and navigating these challenges can be very daunting without support.

This is a tall order when diagnosis remains an issue due to inadequate information and lack of awareness. Forty percent of rare disease patients are misdiagnosed at least once. Primary care physicians, and even specialists, often do not link the symptoms they are witnessing to a rare disease.

In order to raise awareness and initiate diagnoses of rare diseases, the industry should engage in multichannel awareness campaigns providing comprehensive educational support as early as Phase II development of medicines targeting a rare disease. In so doing, pharma also becomes a valued contributor to the community and gains its trust.  It should be working in partnership with appropriate advocacy groups to better understand the issues faced by rare disease patients and then conduct online patient and HCP awareness campaigns and social media listening efforts. Outbound contact centers can target appropriate HCP groups and identify those most likely to have potential rare disease patients. Once identified, posters benefiting rare disease patients can be placed in appropriate physician offices and follow-up emails sent to these HCPs. It is critical to enable knowledge sharing, networking and collaboration between HCPs in order to best serve this patient population. Developing expert-led rare disease medical education programs and providing online and face-to-face networking opportunities can lead to the coordination of multidisciplinary care often necessary in treating rare disease and also more quickly bring to light new treatments as they are developed.

Read the full article.

Receive all of our latest blog entries straight to your inbox!