When I think of rare diseases, I remember the story of a young boy from Auburn, US. He was suffering from an almost unheard of immunodeficiency condition called Job Syndrome. Despite his medical condition, The Star reported how he passionately pursued bike racing – taking a gamma globulin injection every 21 days to build antibodies that his body did not naturally produce.
One in one million people are affected by this rare condition, and we are yet to find a cure. Till date, only a couple of hundred cases have been reported. Monsters and Critics, in August 2017, published a similar story of Lucy Wies who is part of the first human trial that aims to find a cure for Job Syndrome. The clinicians at the National Institutes of Health, US, are working with Lucy to find a way to treat this rare condition soon.
These stories highlight a crucial point—creating awareness of rare diseases to find the right treatment. In this regard, the patient’s medical history plays a significant role in providing the right direction for pharma R&D. However, clinicians have noted gaps in accessing patient data, which is primarily attributed to complex regulatory standards, low engagement levels, and hesitance to adopt emerging technologies.
Reaching out to a wider community
From Facebook and Instagram to YouTube, and a variety of other platforms, now is the time to leverage social media to spread information across numerous channels. Around the world, people with common concerns already connect online and offline with each other to share their experiences, reach out for support, and often help each other with different solutions. Data from this community can be extremely useful for clinicians, researchers, and industry leaders to define a roadmap that will help reach better patient outcomes faster.
For pharma, the Orphan Drug Act (ODA) has incentivized drug development by creating scope for meaningful industrial involvement and continuing research even after patents have expired. Digital marketing and technology companies are using social media to help researchers connect with patients to engage them in clinical trials. Their efforts have already yielded fruit—shortening the recruitment schedules and finding more patients from rural areas and minority groups. Employing digital technologies allows clinicians to focus on what matters the most, providing patient care right at the start of the trial.
Raising Rare Disease Awareness
User-generated-content (UGC) on social media channels can provide insights gleaned from these shared experiences. Further, digital technologies like analytics can help break down these huge volumes of data into easily consumable reports. By its capacity to enable accessibility, social media can help pharma players enable patient engagement that goes beyond clinical testing and survey feedback. Integrating wearable devices with data servers can help researchers understand the status of patient health in real-time. Adding a social media layer here can help patients alert near ones about any adverse events. Moreover, this can provide a statistically validated report on the treatment’s success that will generate further interest in the trial.
Mayo Clinic has demonstrated a good success story of using social media in recruiting patients with Spontaneous Coronary Artery Dissection (a rare disease). Researchers at Mayo Clinic were able to recruit, within 1 week, the required number of 12 patients surviving SCAD. In a second paper published in JAMA by Mayo Clinic, the researchers discussed that through the use of social media they had been able to enroll more than 400 patients to the Mayo Clinic SCAD Registry, which began as a patient-driven initiative to raise awareness.
The list of success stories continue to grow with more and more clinical research players involving social and digital media technologies as an integral part of clinical trial plan. This includes posting ads on mainstream sites—primarily Facebook—about upcoming trials to interactively engage with patients. All of this can be done within the existing regulatory framework while one can easily measure the success and compare the cost of the campaigns.
In his research paper, Wolfgang Eglmeier has cited McAnulty’s work, which evaluated the cost for the 31 randomized patients in a movement disorder trial. The costs per randomized patient attracted to the trial by radio advertisements were $17,222, compared with $3961 per patient randomized by search engine marketing.
In the field of rare disease drug development, the future will belong to those who will adopt the power of digital marketing techniques. Glimpses of which can be seen from the way some service providers, that manage online communities, are contributing towards the patient engagement landscape.
The strategic implementation of such campaigns results in faster drug development which may mean cost effective treatment for patients with rare disease.
How else can clinicians and researchers leverage emerging technologies to ensure better outcomes while treating orphan diseases? Tell us in the comments below.