SHARING INSIGHTS ON HUMAN FACTORS DESIGN IN ORPHAN DISEASES
It was a privilege to speak in front of a large and esteemed audience at the sixth annual HFES International Symposium on Human Factors and Ergonomics in Health Care, made even more satisfying by the dynamic debate that followed my presentation.
Taking to the stage at any event always carries concern over the appropriateness of one’s presentation but any concerns I had quickly dissipated after I asked the audience to raise their hand if they’d experienced problems recruiting patients for studies into orphan diseases. When more than 50% of the audience raised their hands, I knew that what Beth Loring, Principal of Loring Human Factors LLC, and I had to present would resonate in the same way it had with clients we had engaged with.
HFE, or human factors and ergonomics, is a topic of great concern to many and this particular event has grown over recent years. I presented to a diverse group including large pharmaceutical companies, the FDA, academia, government, media, and others involved in the supply chain that supports the development of solutions in this sector.
As mentioned, the key focus of my presentation was to share the experience we have gained at Radius Innovation & Development in recruiting patients to conduct usability studies for ‘orphan diseases’, defined as diseases with less than 200,000 diagnoses. Currently, there are approximately 7,000 known orphan diseases affecting around 30 million Americans.
There are several reasons that the number of drugs and biologics for orphan diseases are rising. These include expedited processes, including fast tracking, and special designation as an orphan or breakthrough therapy. Other incentives include tax credits to offset clinical trial costs, special grants, and extended market exclusivity.
In my five years at Radius working on human factors research programs, we have worked on a number of cases and have learned many lessons on the way, which I was pleased to share along with the methodology we have built around such programs.
The challenges lie in the fact that standard recruiting methods are often unsuitable. We’ve conducted FDA validation studies where as many as 120 participants are required, the majority of participants can be drawn from a mix of nurses, caregivers, adult patients and juvenile patients, half of whom may be naive users and half experienced users. Since these diseases are rare, patients are also rare, and what’s more, patients may be unwilling or physically unable to participate. Hence a large database is needed together with creative recruiting methods.
I presented a human factors research case study to the audience based around a combination device that delivers a drug through subcutaneous infusion therapy to over 10,000 patients with a rare autoimmune disease. Certain strategies were employed as a result of the recruiting process being more difficult. These included testing in multiple locations, rather than a single city (four cities in this case), and extended recruiting timeline (8-12 weeks instead of 2-3) and an increased budget. To meet the more challenging recruitment process we employed multiple recruiting vendors to widen the database, rather than a single national recruiting company.
Based on our experience at Radius, I was able to share several overarching pieces of advice:
- Work with you FDA reviewer! Talk to them about you protocol, raise the project challenges with them as early as possible and notify them as you explore different options. They are able to provide guidance and suggestions.
- Recognize your challenges and attempt to address them early, using information from formative studies.
- Leverage the Drug Sales Organization Database to identify geographic areas with highest concentration of patients, as well as physicians treating patients with the disease. This will increase the probability of finding recruits, perhaps through physician outreach or patient referral.
- Consider working with local non-profit organizations or charities dedicated to specific diseases that may be able to provide links to blogs or online communities for patients and who may, with a donation, offer to post study details on their websites or patient blogs.
- Transform patients into an army of recruiters through referral incentives and through good old fashioned word-of-mouth. Many patients participate in local support groups; some diseases are hereditary and family members may be fellow sufferers.
And when all else fails, consider reusing formative study participants, or using surrogate patients with similar symptoms or disease characteristics, but as mentioned before, confirm with your FDA reviewer as early as possible.
Companies are becoming more aware of human factors research and more involved in the early stages of product development in the hopes of minimizing user errors rather than mitigating issue as an afterthought, where it is expensive and inefficient. Getting the human factors right first time is hugely valuable and the Radius HF team has a great deal of experience in this arena. Radius prides itself on its focus on the user experience as part of the ideation process of any product or ecosystem design; this is as important in this instance as in any other sector.
As with all innovation, the desire to compress the design, development, testing and approval processes and to get to market first is high. Getting the human factors research right first time is part of the process of compressing the time frame for product development.
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