Researchers conducting clinical trials for rare diseases typically face two types of recruitment issues. The first one pertains to logistics and administration. Because about half of rare disease patients are children, getting them enrolled requires significant effort and collaboration with parents and caregivers. In addition to granting consent for a potentially life-changing treatment with uncertain outcomes, adults may need to take time off work, travel to clinical sites, or even relocate altogether. As patients are not assured of experiencing an improvement in their condition, they and their families may not always perceive participation in clinical trials as worth the effort.

The second recruitment problem pertains to ensuring ethnic diversity and inclusivity within the patient pool, as different groups can exhibit different reactions to the same treatment. Inclusive clinical trials are therefore most promising at generating universal, broadly applicable findings. However, industry sponsored clinical trials for rare diseases in the US fall short from meeting this standard—despite the country’s multiethnic composition. Caucasian males are currently overrepresented in rare disease patient pools, as women and ethnic minorities often encounter biases and barriers when enrolling in clinical trials at investigator sites.

“Logistics and the lack of ethnic representation are the main factors driving patient recruitment roadblocks in rare disease clinical trials,” says Dr. Rajasimha. “Biopharmaceutical companies and clinical trial investigators must get creative in enrolling and engaging participants in the context of these obstacles. They need to look beyond the US and EU for investigator sites for enrolling diverse patients.”

The promise of patient advocacy groups
Patient advocacy groups (PAGs) have been traditionally called upon by clinical investigators to assist in the recruitment for rare disease clinical trials.

PAGs can facilitate connections between both parties, enabling researchers to access a broader patient pool. On the patients’ side, these groups, often comprising individuals with firsthand experiences, can help patients understand the importance of participating in clinical trials and contributing to the advancement of medical research. For instance, PAGs can organize community events and workshops, creating environments where patients feel comfortable discussing and considering clinical trial participation.

Beyond acting as intermediaries between researchers and patients, PAGs possess a distinct advantage over the former in the recruitment of clinical trial participants. PAGs excel in providing emotional support and guidance to patients, helping them cope with their disorder with a level of empathy that researchers cannot replicate. Such support can prove invaluable in assisting patients through periods of hardship, such as the phases of a clinical study. A PAG may be the difference between a participant continuing with a study or dropping out of it.

Finally, PAGs may include healthcare professionals and other specialists among their members. This collective expertise positions these groups to actively participate in the design and implementation of clinical trials, leading to more patient-friendly protocols. For example, PAGs can devise strategies to minimize clinic visits, improve communication channels between researchers and participants, and implement culturally sensitive procedures. By enhancing the appeal of clinical trials from a patient’s standpoint, PAGs provide yet another avenue for contributing to patient recruitment and retention.

According to a study conducted in genes research, 91% of PAGs surveyed have assisted in participant recruitment, and 56% contributed to study design.⁴

Three modern strategies to maximize engagement with patient advocacy groups
While the idea of leveraging PAGs to bolster patient recruitment efforts is well understood, making the most out of this collaboration is more complicated. Cooperation between researchers and PAGs is commonplace, but the results have not always been satisfactory. Research indicates that only 68% of surveyed PAGs had a positive experience working with researchers during clinical trials; 12% regretted their experience altogether.⁵

“It is not uncommon for PAGs to distrust biopharmaceutical companies,” says Rajasimha. “The former’s prioritization of patient well-being and the latter’s focus on medical advancement can result in fundamentally divergent interests.”

Given that collaboration between these entities is necessary but challenging, researchers and the companies they work for should explore new approaches to foster trust with PAGs. Creative and attuned to emerging technological trends, these strategies should empower researchers to present their best selves when engaging with PAGs in the foreseeable future.

●    Set up clinical trial sites abroad: Including investigator sites in multiple countries with known prevalence of the rare disease is one of the best ways to boost diversity of patients in a clinical trial but will require collaboration with international PAGs. Such trials not only have to be compliant with local regulations and can be potentially expensive, but they directly benefit more patients and help sponsor achieve their DEIA goals to satisfy regulatory expectations.

However, organizing clinical research in other countries is more than just showing commitment to foreign rare disease patients and their advocacy groups. By having access to ethnically diverse patient pools, multinational studies address the homogeneity issue prevalent within clinical trial cohorts in the US and/or EU.

A particularly suitable country for hosting clinical trial sites for US biopharmaceutical firms is India. Despite the country representing one-third of global rare disease cases and the Indian diaspora constituting a sizable proportion of the American population, participants of Indian origin accounted for merely 1% in US-based rare disease clinical trials.⁶

●    Integrate new, AI-powered clinical management tools: Even when researchers and PAGs are committed to working together, engagement and communication can be problematic when both parties are collaborating from disparate geographical locations. Routine activities, including coordinating and hosting in-person meetings and events, documentation, and record-keeping, and ensuring PAGs are well-versed with the specifics of the clinical trial process, suddenly become veritable setbacks.

Modern clinical management tools, inspired by the popularity of remote patient access, can align researchers and PAGs more closely. Features such as omni-channel communication, eDocumentation, automated workflows, and process modularization effectively address most operational barriers while elevating the scalability of clinical trials to unprecedented levels.

A notable clinical management platform tailored for modern clinical trials, including for rare diseases, is the Jeeva eClinical SaaS system. Utilizing AI for automation, remote screening, and home-based trial participation, the software can play a crucial role in assisting researchers and PAGs with patient recruitment and engagement.

●    Involve nonprofits: Nonprofits facilitate partnerships between industry and PAGs, amplifying impact and driving even more positive outcomes. Often situated at the center of the rare disease community, nonprofits comprehend the values and priorities of different PAGs and can advise researchers on approaching the latter effectively. Bringing a nonprofit onboard also adds legitimacy to the partnership, cultivating trust from both sides.

Nonprofits offer concrete resources that can aid engagement with PAGs as well. Grant programs allow PAGs to organize outreach campaigns and sustain prolonged recruitment drives for clinical trials. In the event a PAG encounters a knowledge gap hindering collaboration with investigators, a nonprofit can provide training through conferences, webinars, and workshops.    

“A nonprofit organization I founded, the Indo US Organization for Rare Diseases (IndoUSrare), has a long history of bridging geographic silos and gaps between industry partners, researchers, and patient advocacy groups,” says Rajasimha, CEO. “A few months ago, we organized our inaugural Indo US Bridging RARE summit at George Mason University, where we flew in patient advocates and connected them with global experts.”

Together, these strategies should permit researchers to establish more fruitful relationships with PAGs. Conducting inclusive modern clinical trials with international sites signals dedication to the patients globally and expands the available patient pool for researchers. Adopting sophisticated clinical management tools solves important organizational and operational conundrums that arise during these unions. Serving as force multipliers, nonprofits can add considerable value to these endeavors as well.

Collaborate across borders with confidence
Researchers’ struggle to recruit enough eligible patients within a limited time frame for clinical trials remains one of the greatest challenges blocking medical progress in this field. Engaging patient advocacy groups (PAGs) early on during the clinical trial process has demonstrated success in galvanizing timely patient recruitment and retention. Nonetheless, successful teamwork between researchers and PAGs is far from guaranteed—a troubling aspect given that rare diseases impact millions.

Perhaps sponsors of orphan drugs have much to gain by considering alternative frameworks of patient engagement in clinical trials. Establishing clinical trials in selected countries for the purposes of diversity and inclusion, integrating AI-powered modern clinical trial management platforms, and bringing in nonprofits as a third party are all part of winning strategies.

“For various reasons, including the political environment, the incentives for the biopharmaceutical industry to invest in rare disease research and orphan drug development are declining,” says Rajasimha. “Going forward, the quality of collaboration between industry, clinical researchers, and patient advocacy groups will play a magnified role; cross-border cooperation among all stakeholders will be essential.”

“We are excited to collaborate in the Bullpen with Dr. Rajasimha and team IndoUSrare during the 42nd annual JP Morgan Healthcare Investors Week Rare Disease event at the Golden Gate Yacht Club.” says Christiaan Engstrom, Founder of Bullpen.

About IndoUSrare:
IndoUSrare is a humanitarian nonprofit 501(c)(3) tax-exempt public charity organization based in the United States. Founder and Executive Chairman Dr. Harsha Rajasimha, who lost a child to a rare disease in 2012, has been a rare disease advocate for more than 10 years. To address the unmet needs of diverse patients with rare diseases globally, the leadership team comprised of experienced professionals from research, advocacy, regulatory, and drug development seeks to build cross-border collaborations connecting stakeholders of rare diseases in low- and middle-income regions such as India, with their counterparts and clinical researchers in the United States to improve the diversity of clinical trial participants, accelerate research and development, and improve equitable access to life-saving therapies to diverse populations of rare disease patients. Visit https://indousrare.org.

References:
1.    NORD. “Rare Disease Database.” NORD, rarediseases.org/rare-diseases/. Accessed 19 Dec. 2023.
2.    NORD. “Rare Disease Day: Frequently Asked Questions.” NORD, rarediseases.org/wp-content/uploads/2019/01/RDD-FAQ-2019.pdf. Accessed 19 Dec. 2023.
3.    Desai, Mira. “Recruitment and retention of participants in clinical studies: Critical issues and challenges.” Perspect Clin Res, ncbi.nlm.nih.gov/pmc/articles/PMC7342339/#ref7. Accessed 19 Dec. 2023.
4.    Landy, David C. et al. “How disease advocacy organization participate in clinical research: a survey of genetic organizations.” Genetics in Medicine, sciencedirect.com/science/article/pii/S1098360021041629. Accessed 20 Dec. 2023.
5.    Ibid. Accessed 20 Dec. 2023.
6.    Chakraborty, Monoswi et al. “Rare disease patients in India are rarely involved in international orphan drug trials.” PLOS Global Public Health, journals.plos.org/globalpublichealth/article?id=10.1371/journal.pgph.0000890. Accessed 20 Dec. 2023.
7.    Ibid. Accessed 20 Dec. 2023.

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