A substantial change to the drug development landscape is currently underway. Regulatory authorities are starting to acknowledge pediatrics as one of the most underserved patient populations. This is reflected by the revelation that approximately 70% of every drug used in children has still never been clinically tested in children. Fortunately, that’s all starting to change.
As regulatory authorities continue to recognize the need for robust pediatric drug development, they are pushing for more thorough and proactive strategies to accomplish this. The RACE for Children Act, which went into effect in August 2020, is a good example. Under the RACE Act, any company developing a targeted cancer drug for adults must also evaluate their drug for children’s cancers. This regulation provides pediatric cancer patients and their families with more opportunities to participate in precision-based clinical trials and, ultimately, improve children’s therapeutic outcomes.
Moving forward, all drug developers may need to develop pediatric plans for their assets far earlier than they may have anticipated—regardless of therapeutic area. While many emerging biotech companies may have shied away from pediatric development and trials in the past, they will now need to consider pediatrics as part of their asset’s overarching clinical development plan.
Biotech and pharma alike must begin thinking about pediatric models while designing their preclinical programs, early clinical trials, and overall clinical development strategies. By developing and running safe, effective pediatric trials, biotech companies may be better able to position themselves in competitive markets and make lasting differences in the lives of pediatric patients and their families.
By developing and running safe, effective pediatric trials, biotech companies may be better able to position themselves in competitive markets and make lasting differences in the lives of pediatric patients and their families.
Overcoming pediatric drug development challenges.
Many challenges exist in pediatric drug development. Unfortunately, these challenges are often exacerbated for emerging biotech companies, who are usually smaller and operating with limited resources compared to larger pharma organizations. Because small and emerging biotech companies don’t always work on a global scale, they may lack the expertise and understanding of health authority regulations worldwide.
Emerging biotech companies generally have less experience in drug development than larger pharma organizations, making them more vulnerable to issues associated with both adult and pediatric drug development. As such, they may not fully appreciate the risks and benefits of thinking about pediatrics earlier in the drug development pipeline.
Partnering with a CRO that is experienced in pediatric clinical development can help emerging biotech meet these challenges head-on. Biotech companies should look for a CRO who understands the benefits and challenges of developing pediatric programs. This experienced and effective partner should also recognize that biotech companies may not be considering a full development approach, but rather looking to maximize the value of their asset for co-development or acquisition opportunities. Areas where value can be maximized may include pediatrics from an early stage, especially since most programs will require pediatric development in the future.
Addressing pediatric drug development myths.
Many myths surround pediatric drug development, especially regarding how these studies fit into a biotech’s development plan. Below, we address some common misconceptions and provide insight to help emerging organizations safely and effectively design and run pediatric trials.
Myth: Not including children in drug trials protects them.
For much of recent history, regulators and drug developers believed they were protecting children by keeping them out of clinical trials. This misconception stemmed from a lack of good, controlled studies throughout the mid-1900s, which led to some drugs having adverse effects on children. Based on these outcomes, regulations were established that made it difficult to conduct clinical trials in pediatric patients.
Limited pediatric studies led to the practice of off-label prescribing and, in turn, inadequate evidence on medication use and appropriate treatment for many pediatric conditions. Off-label prescribing occurs when little or no pediatric experience in controlled environments has been initiated or completed. Healthcare providers use “best guesses” from adult patient data when prescribing pediatric patients medicines, especially when related to dose.
This practice alone is risky as adequate dosages are difficult to determine without sufficient information regarding how a drug or treatment affects children and their conditions. At the same time, waiting until long-term safety data is well-known in an adult population delays access to therapies that could be potentially life-changing for sick children.
Not including children in drug trials means that children may receive off-label treatment without adequate understanding of dosage, which could lead to harm. Knowledge is power for prescribers and patients alike. A better understanding of the evolving healthcare landscape as it pertains to pediatrics ensures that all medicines are fully vetted in vulnerable populations.
Myth: Pediatric studies cost biotech companies more money.
Many emerging biotech companies believe that the upfront costs of running a pediatric trial simply aren’t worth it. The truth is that, depending on the nature of the program, all drug developers will ultimately need to implement a pediatric plan. Waiting to start until later down the development pipeline can increase costs and limit value at product launch.
On the contrary, early development in pediatric patients means earlier completion. Early completion of a pediatric study can maximize the target populations, and thus potentially maximize the value of an asset, extend patent life, and provide added value for all related programs. In addition, the inclusion of pediatric patient populations in adult phase IIb and phase III programs may reduce the overall pediatric sample size necessary, thus saving emerging biotech companies time and money.
Obtaining pediatric data on a drug or therapeutic also helps maximize the asset. Regulatory companies don’t want to simply de-risk. They reward companies that incorporate pediatric development early and effectively. Biotech companies that pursue pediatric development earlier will receive various incentives. One example of this is increased patent protection on assets post-approval, which can increase the overall asset value if the biotech develops the product alone or should a partnership be developed.
Developing a pediatric plan alongside an adult study allows biotech companies to show a better understanding of the drug development process and provides better opportunities for asset evaluation and value. Developing these studies simultaneously also helps patients. Data generated and examined in a controlled environment helps healthcare providers prescribe the right medicines and the right dosages for the right reasons.
Myth: Pediatric studies are more challenging than other types of clinical trials.
Pediatric studies are certainly not without their fair share of challenges. However, with the right partner, these challenges can be effectively addressed, mitigated, and often even avoided in the first place.
Emerging biotech companies should partner with a CRO that offers a full-service approach. For example, PRA has pediatric experts in all facets of clinical development, from regulatory and operations to therapeutic expertise and health outcomes. This broad, deep expertise, combined with our long history within the biotech space, allows us to customize our approach to meet the needs of any pediatric program. Through transparent, early conversations, we’re able to support and guide biotech companies through all aspects of the drug development process, including Pediatric Investigation Plans (PIPs), Pediatric Study Plan (PSP) development, discussions with regulators, protocol writing, and trial execution.
Collaboration is critical for successful pediatric development, which is why PRA established the Pediatric Site Network (PSN), a global partnership made up of internationally recognized pediatric centers of excellence and emerging centers with a large pediatric patient catchment. Each of these healthcare centers shares PRA’s commitment to bringing innovation to pediatric development and has been thoroughly vetted and personally visited by our pediatric executive leadership. The long-term relationships we’ve built and continue to establish with the PSN provide emerging biotech with relevant scientific feedback from global key opinion leaders (KOLs). Their expertise helps inform our protocol development and study design, as well as obtain realistic validation of standard of care in multiple regions.
Emerging biotech companies are uniquely positioned to run pediatric trials.
Emerging biotech has a unique advantage when it comes to pediatric clinical development. These smaller, more agile organizations are used to working with fewer resources and pivoting as needed. They’re also more willing to give innovative approaches a try—a necessity for designing and running pediatric trials. With the right partner, biotech companies can address common and unexpected pediatric trial challenges to bring life-changing products that are safe and effective for children to market.
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