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Rare disease is a complicated but rewarding space for drug developers of all sizes. Most rare diseases have no approved treatment options, making clinical research the only care option for many of these patients. Due to this high unmet medical need, rare patient communities are often ready and willing to not only participate in clinical research, but help shape it to ensure it meets the needs of patients. This provides emerging biotech organizations with the opportunity to work closely with patients and their advocates on potential new treatments.

The Center for Rare Diseases
The Center for Rare Diseases

Additionally, the rare disease space typically has less competition, which gives smaller biotech companies the opportunity to be first-to-market. The regulatory path required for these studies is often clearer and shorter, and the clinical development costs are typically lower because these studies require less participants and have a higher probability of approval.

However, these studies are not without their challenges—both for the patients and their loved ones as well as the drug developers working on life-changing treatments and therapies.

Finding patients for a rare disease study can be difficult, especially as the majority of these disease populations are relatively small and geographically dispersed around the globe. This makes site selection, enrollment, and retention especially challenging. In order to truly meet patients where they are, sponsors must implement innovative solutions, technologies, and strategies for engaging patients within rare disease studies. Unfortunately, small to mid-sized biotech organizations who’ve never worked in this space (or with a particular rare disease) before may have less resources and tools to leverage.

Fortunately, they don’t have to do it alone. Contract research organizations (CROs) with extensive experience in the rare disease space and a trusted history of working with small biotech can help efficiently design and conduct safe and effective rare disease studies.

When choosing a rare disease partner, small biotech should consider the following:

What type of experience does the CRO have?

First and foremost, look for a CRO who has experience working with small biotech companies and understands the unique pressures and nuances within this dynamic space.

Experience within particular therapeutic areas and, ideally, within the specific disease state you will be working in is important—but a successful track record of working with small pharma and biotech organizations is critical. Emerging biotech must be flexible, agile, and responsive as they are often trying to do more with fewer resources. A CRO partner must be able to adapt to meet their unique needs and provide customized solutions to ensure study success.

It’s also important to look at a CRO’s ability to engage with patients. Do they have experience working with patient advocacy organizations (PAOs) and patient advocates? These relationships are vital in developing a feasible study strategy that works for both the sponsor and the patients, as they provide real patient insights and data. A CRO partner must be able to guide biotech on how and when to engage with PAOs and how to build authentic long-term relationships with these constituents.

One way PRA Health Sciences is working to better integrate rare disease patients into the clinical development lifecycle is with the Rare Diseases Advisory Committee (RDAC). This independent and autonomous volunteer advisory group works with our Center for Rare Diseases to provide insight into how we can make clinical trials more effective and accessible to rare disease patients. The RDAC helps us to incorporate the patient voice early in the clinical development lifecycle and serves as a sounding board to ensure our organization is truly patient-focused.

Global reach will also benefit small biotech companies working in the rare disease space. A CRO with experience conducting global studies can help you find the patients and the run your trials wherever you need to be—whether that’s around the world or from your patients’ very own mobile devices. A CRO should also be able to leverage vast data resources to provide visibility into patients and historical comparators, as well as have experience developing endpoints and protocol designs. A CRO must be able to provide country-specific solutions and local expertise to navigate regulatory pathways, especially within the rare disease space where patients are often recruited from a variety of geographies.

How flexible is the CRO?

Many emerging biotech are often new to working in the rare disease spaces. To stay competitive, they must move faster and think differently than larger, more established organizations already working in the same space. A partner that can adapt to their evolving needs is essential—especially when it comes to rare disease studies where customized and innovative approaches are the norm. A CRO partner must adapt their processes and workflow to accommodate the unique environment within which a biotech is operating.

“A CRO must go beyond operational excellence and become a trusted advisor and expert consultant to the sponsor’s development team,” PRA’s Solomon Babani, Senior Vice President and General Partner of Scientific & Medical Affairs, explains.

The last thing an emerging biotech wants is to become lost in the shuffle or stuck in a rigid partnership. Just as they must be nimble, so, too, should their partner. CROs that are overly process-oriented likely won’t be able to provide the support these agile companies require. Rather, it’s important for emerging organizations to partner with a CRO that packages their expansive resources in a customizable and fluid way.

Does the CRO have any tools that can give our research a leg up?

A patient-focused paradigm that leverages technology can provide broad access to patients wherever they may be. This is especially important with geographically disparate rare disease patients.

For example, incorporating telemedicine and the use of wearables into a study allows for remote and continuous data collection. Not only does this boost enrollment and patient retention, it can also lower the overall study costs while speeding up overall development timelines. An effective CRO partner should be able to provide a variety of tools like these to facilitate decentralized, hybrid, and virtual study designs that are customized to meet each sponsor’s unique needs.

Of course, having the technology is not the same as optimizing use. “This is not about throwing a piece of technology at a study and expecting perfection,” PRA’s Brandon Early, Vice President of Project Delivery, says. “You need to know the problem you are trying to solve and how to use the technology to solve it and more.”

Emerging biotech must not only consider the technology a CRO provides, but the expertise and guidance behind that technology. When it comes to clinical trial design, there’s no one-size-fits-all solution that will work for all sponsors and all trials. That’s why PRA builds customized digital health solutions that are determined by the specific disease state, patient population, and sponsor’s targets. Our experts leverage medical informatics to guide study design, predict enrollment rates, and analyze the competitive trial landscape to ensure every sponsor has exactly what they need to succeed.

How does the CRO incorporate the patient voice into their studies?

Only a very small percentage of eligible patients enroll in clinical trials, yet their participation is critical and required for developing new therapies. Often, this is due to poor clinical trial design. Even today, many studies are still designed without thinking about what is realistic for patients and their families.

“Studies that are designed to be more patient-focused will be easier to enroll and retain patients and result in a more diverse and representative patient population,” Scott Schliebner, Senior Vice President at PRA’s Center for Rare Diseases explains. “Clinical development is overdue to adopt a more consumer-focused approach and to better consider the end user—the patient.”

Incorporating the patient voice early in the study planning phase is key to improving study design, accelerating enrollment, and boosting patient retention overall. Small biotech companies should engage early, often, and consistently with PAOs as these organizations know their disease and their patients better than anyone else. Patient organizations know who their patients are, where they live, and what their healthcare experience is like. They can help guide biotech companies in study design, endpoint selection, eligibility criteria, and investigator selection. They can also assist with developing registries and natural history studies, conducting study feasibility, and supporting study branding, awareness, and patient recruitment.

A CRO with proven experience working with patient advocacy groups can help emerging biotech develop open, authentic, long-term relationships that are mutually beneficial to both the sponsor and the patients.

How soon can the CRO start engaging with our program?

There is a myriad of factors that can impact a rare disease clinical program. Working with a partner who has experienced most of them and created solutions to overcome these challenges is important. The right CRO can support an overall drug development plan, help design and develop clinical studies, and create an overall patient community engagement plan to support the success of your rare disease study. The sooner they’re strategically incorporated, the better.

At PRA, we’ve worked with many emerging organizations across all phases of development, incorporating a variety of approaches and strategies. We know what works and what doesn’t because we’ve done it before. Our goal is to save sponsors time, money, and frustration by providing custom solutions based off of learned outcomes and experience—not best guesses. From external support in funding and asset valuation to development and regulatory strategies, we’re here to help emerging biotech bring life-changing rare disease treatments to fruition.

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