Medidata Blog
Accelerating Rare Disease Clinical Development by Enhancing Patient Engagement in Clinical Trials
More than 7,000 rare diseases affect over 400 million people globally and almost 30 million in the United States. Estimates indicate that nearly 1 in 20 individuals across the world will be affected by a rare disease at some point in their lives. These numbers are staggering.
There has been exciting progress over recent years; for example, in 2021 over 30 drugs were approved by the FDA for the treatment of rare diseases such as Duchenne muscular dystrophy, Alagille syndrome, Late-onset Pompe disease, and certain types of non-small cell lung cancer (NSCLC). However, rare diseases still present a vast area of unmet medical need. Despite the burgeoning scale of this need and various orphan designation incentives, there are only about 550 FDA-approved orphan drugs and medical devices available for treating rare diseases.
This is not surprising given some of the key challenges associated with rare disease clinical research, including:
- Rare disease patient recruitment
- Patient retention
- Small patient populations that can increase the risk of incomplete study data
- Limited clinical trial data for analytical purposes
- Limited understanding of the disease
- Limited access to rare disease specialists
- Ill-defined clinical targets and endpoints
- Rare disease clinical trial execution
Such challenges are further exacerbated by the geographic spread of both patients and medical experts, often requiring complex multinational collaborations. These obstacles have traditionally dissuaded investment in rare disease drug development since they slow the path to approval and add substantially to drug development costs.
However, the biopharmaceutical industry has been increasing its focus on developing novel rare disease therapies, thanks to the advent of novel clinical trial technologies and methodologies for supporting rare disease clinical development. Modern solutions that support rare disease clinical drug development include novel approaches to increasing patient engagement in clinical trials.
Drug developers must incorporate patients’ voices and their expert insights into the rare disease clinical development process as early as possible to maximize the impact of clinical trials. This is done by nurturing connections and communication between rare disease patient groups, medical and scientific experts, drug developers, and patient advocacy groups. Importantly, patient advocacy groups understand the common priorities of the rare disease patient community, and generally have a big-picture view of what is important to a specific patient group.
Advocacy groups typically have a great deal of expertise when it comes to engaging academics and drug developers throughout a therapy’s lifecycle. Collectively, these interactions not only foster patient engagement, which translates into enhanced recruitment and retention, but also foster the inclusion of patient voices, which enrich diversity, encourage knowledge sharing, and facilitate patient access to leading medical experts.
When patient engagement in clinical trials is enhanced and a highly-positive experience is created, both rare disease patients and drug developers benefit—patients from the accelerated development of novel therapies, and drug developers from the connection with the rare disease patients they need for their clinical studies. It is key for drug developers to be sensitive to both representativeness and diversity in their collaborations with patient communities. Through early engagement with members of the patient community, including patient advocacy groups, drug developers can position their rare disease programs for success.
Watch this video featuring rare disease experts on patient engagement in clinical development.