A convergence of trends — including the continuously growing focus on drug development for rare diseases, the rising percentage of biologic drug candidates, and the expansion of decentralized trials — is creating opportunities for more efficient trials that are less burdensome for patients and their families. With a limited number of rare disease patients, clinically relevant data is invaluable. Thanks to innovative and flexible protocols, we are developing new ways to enable us to gather critical trial data to support new drug submissions.
Growing Interest and Investment in Rare Disease Research
Over the last 20 years, the focus of the pharmaceutical industry has shifted away from blockbuster drugs treating patient populations of hundreds of thousands or millions to more specific treatments that target subtypes and increasingly rare diseases with smaller, more dispersed patient populations (right drug for the right patient at the right time).1
Simultaneously, there has been a shift in protocols for clinical trials due to a growing percentage of new drug candidates being biologics, including advanced antibody–drug conjugates, bispecific antibodies, cell and gene therapies, and other novel medicines — a growing proportion of which are focused on treating rare diseases.
The growth of gene therapies coupled with a greater focus on rare diseases has also coincided with the introduction of remote digital tools that, in part, enable the decentralization of clinical trials.2 When the pandemic intensified, decentralization became critical for the execution of nearly all clinical trials. As a result, the industry witnessed further adoption and acceleration of hybrid and fully decentralized or virtual study models. This paradigm shift suits the rare disease space well, as patient populations with rare diseases are typically both small and geographically dispersed, making site-based trials for these studies challenging.
As it stands today, digital and decentralized solutions have made it possible for more patients to participate regardless of location. Notably, we are also seeing an uptick in use of decentralized approaches within rare disease pediatric trials, including those with highly vulnerable populations.
Flexibility for Pediatric Patients and Their Loved Ones
Bringing children into a clinic has many challenges, and for children with rare diseases it can be even more difficult. However, with the widespread availability of decentralized options, hybrid approaches are possible which include a mixture of in-clinic and at-home endpoints, supported by bespoke digital platforms and novel trial design solutions.
Patient feedback has indicated that parents prefer to interact with their physicians face-to-face on some occasions, particularly early in treatment, but are amenable to remote interactions outside of milestone visits. Combining home-health nursing visits with periodic on-site physician evaluations can offer a truly patient-centric approach. This gives patients and their families maximum flexibility for clinical trial participation, in that they can opt for what best suits their schedule or lifestyle.
Patient Experience is Paramount to Success
We believe that it is crucial to do everything possible to optimize the patient experience, especially for the rarest of indications. Regardless of how a study is set up — with the patient visiting a local clinic, or the central investigator site or a physician or nurse making an in-home visit — that interaction must occur without delay and with minimal inconvenience to the patient. A white-glove service, such as support of a study concierge, is another way study teams can elevate the research experience while ensuring patients remain engaged and samples are collected on time and handled appropriately, with all critical data collected
Furthermore, the PPD patient concierge service acts as a dedicated resource for patients and their families. These trained individuals serve as a guide through all aspects of trial participation. This single point of contact helps organize everything from travel to reimbursement of expenses and coordination of home health nurse visits. The concierge can also provide reminders about appointments and patient-reported outcomes (PROs) requirements with the goal of making trial participation as easy as possible, while ensuring key data is collected. This is significant because many rare disease patients can spend years before receiving their correct diagnosis. Without steadfast engagement while on a trial, there is a stronger chance of dropout.
A Network to Support All Stages of Research
Significant regulatory expertise and proactive regulatory engagement are essential to the success of modern protocol designs. We help by augmenting the existing expertise of biotechs and pharma organizations to provide more robust knowledge across all regulatory aspects. Our established relationships with regulatory agencies enable us to engage frequently around new concepts and opportunities. We also work proactively with global regulatory bodies on each clinical study we support to ensure that the trial protocols meet their expectations and that the data generated will support product registrations.
Similarly, partnerships with vendors that understand the specific needs of rare disease patients participating in decentralized clinical trials can provide sponsors with a necessary edge and help them select the best solution for their trial. To improve patient recruitment, we also work closely with advocacy groups and others active in the rare disease community.
The Partner of Choice
Rare disease study sponsors are passionate about their programs and extremely knowledgeable about the disease(s) they are hoping to treat or cure. They seek an equally devoted CRO partner that can facilitate other aspects of the study process with the highest level of quality. These clients benefit from PPD custom solutions, particularly when it comes to patient recruitment and engagement in multiple countries (rare disease patients are often difficult to find and must be approached and managed differently from those in a study involving a large cohort of patients with a more common disease) and treatment of patients in non-traditional settings.
More than Just Incremental Changes to Our Offering
In the rare disease space, our therapeutic knowledge, combined with our leading digital and decentralized solutions, supports the seamless design and execution of innovative protocol designs. Our experience allows us to take less of a “bolt on” approach and instead leverage the full spectrum of tools, vendors, systems, and platforms to produce bespoke solutions tailored to patient and research needs, ideally with the PPD team being engaged during protocol development to ensure study design incorporates patient-centric solutions.
For instance, in the United States, we partner with remote clinical staff, including nurses, nurse practitioners, phlebotomists, and qualified physicians who visit patients’ homes to conduct assessments from basic physical exams to evaluations that require highly specialized techniques. For rare disease studies, this solution is another mechanism for taking the trial to the patient.
Our teams have also been instrumental in deploying mobile sites within recreational vehicles. This offering has been used extensively during the COVID-19 pandemic to recruit patients in remote settings or to reach those where an outbreak may be taking place.
Our patient-centered approach to innovation is helping increase the benefits of decentralization in rare disease clinical trials. In partnership with our dedicated centers of excellence, we continue to explore new ways to support this goal by offering a broad range of options for rare disease patients, including those that extend beyond conventional trial approaches.
Rare disease study sponsors are passionate about their programs and extremely knowledgeable about the disease(s) they are hoping to treat or cure. They seek an equally devoted CRO partner that can facilitate other aspects of the study process with the highest level of quality.
Our patient-centered approach to innovation is helping increase the benefits of decentralization in rare disease clinical trials. In partnership with our dedicated centers of excellence, we continue to explore new ways to support this goal by offering a broad range of options for rare disease patients, including those that extend beyond conventional trial approaches.
References
- Kakkar, Ashish Kumar & Neha Dahiya. “The evolving drug development landscape: from blockbusters to niche busters in the orphan drug space.” Drug Dev. Res. 75: 321–324 (2014).
- Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. U.S. Food and Drug Administration. 15 Jan. 2019.