Real progress happens where unmet medical need is greatest and patient impact can be clearly measured. Pediatric rare cancers exemplify these criteria and have therefore generated continued investment, even in a capital-constrained environment.
M&A and IPOs may be picking up, but the fact remains that the biotech markets have remained cautious and capital-constrained since the bursting of the pandemic bubble collapsed the investor base. The capital environment has forced companies to be more deliberate about where to invest time and money, as investors are more selective than in the past, timelines are under pressure, and the bar for clinical evidence has never been higher.
Even in this environment, regulators and investors show continued interest in rare cancers, particularly in pediatric populations.
Having launched nine products spanning oncology, surgery, HIV, pulmonary disease, cardiovascular disease and diabetes in my over 25 years at Amgen, Johnson & Johnson and Boehringer Ingelheim, I have seen significant changes in biotech’s approach to science, finance and regulation. One theme that has remained consistent is that real progress happens where unmet medical need is greatest and patient impact can be clearly measured.
These criteria define the rare pediatric cancer space and have shaped how I approach my work as CEO of OS Therapies, a clinical-stage company focused on developing immunotherapies for aggressive and rare cancers.
From what I’ve seen, investment in rare pediatric cancers isn’t driven by novelty or optimism alone. It reflects a broader shift in what constitutes real value in drug development, with programs targeting crowded therapeutic areas or offering only modest improvements on the standard of care struggling to stand out in the current tight funding environment.
Rare disease programs tend to operate under a different set of assumptions. These diseases often have limited or no effective treatment options, small patient populations and, in many cases, poor survival outcomes. Programs seem to stand out when there is a clear biological rationale and meaningful, measurable clinical benefit; this is especially true in pediatric oncology.
Tailored drugs, tailored regulation
Childhood cancers are biologically distinct from adult cancers, yet many chemotherapy treatments used today were adapted from adult indications. As a result, pediatric patients with rare cancers often face limited benefits alongside significant and debilitating side effects.
Programs developed specifically for these patients are addressing a long-standing gap in care. For example, at OS Therapies, our work focuses on stimulating the patient’s own immune system to recognize and fight circulating cancer cells and solid tumors with significantly fewer side effects than chemotherapy, allowing us to treat the many patients who experience dose-limiting chemotherapy side effects with little documented benefit. This type of therapy has been encouraged through FDA’s strong interest in regenerative medicine innovation, especially in rare pediatric diseases.
The FDA has spent decades developing programs and pathways to support development in rare diseases and pediatric populations. Designations such as Orphan Drug, Fast Track, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT) and priority review are intended to reflect a different balance of risk and benefit when patient need is great and options are limited. These programs don’t lower standards, but they do recognize that flexibility and speed can matter when the stakes are high and placebo-controlled trials are difficult to enroll.
The Rare Pediatric Disease Priority Review Voucher program has become especially relevant in recent years. While the market value of these vouchers has fluctuated over time, their strategic importance has remained largely consistent for small companies looking to address rare childhood diseases.
When Congress narrowly passed a spending bill in early February that included renewal of the pediatric rare disease priority review voucher, it ended a nail-biting year-plus for the rare disease field. The program, which grants qualifying drug sponsors a voucher for expedited FDA review that can be sold, had lapsed at the end of 2024, imperiling more than $4 billion in reinvestment, according to the Rare Disease Company Coalition.
That estimate highlights how much the industry depends on priority review vouchers (PRVs). From a development perspective, PRVs help address some of the challenges of working in small, complex patient populations. From an investor perspective, they represent a meaningful, objective source of value that changes the dynamic of how a program is evaluated. From a policy standpoint, they are intended to encourage investment in pediatric rare diseases that might otherwise be unattractive to investors.
A return to fundamentals
Investors today have little patience for vague narratives or loosely defined endpoints. Instead, there’s a growing emphasis on thoughtful trial design, early regulatory engagement and clinically meaningful outcomes. In rare pediatric oncology, that can often mean seeing predictive biomarkers correlate with survival benefit.
Overall, this has been a positive shift. It creates stronger incentives for teams to develop a deep understanding of the biology of their disease, design studies around outcomes that matter to patients and engage early with regulators. It also encourages thoughtful decisions around allocation of limited resources, which ultimately benefits the broader biotech space.
Rare disease oncology is not an easy path. These programs are complex, the work can be emotionally demanding and the patient population is small. Trials are difficult to run, and there’s very little margin for error. But it’s also an area in which patient need, regulatory support and investor discipline come together far more synchronously than in other areas of biotech.
The continued focus on rare disease and pediatric oncology appears less like a passing trend and more like a return to fundamentals. When incentives are structured thoughtfully, and science is paired with unmet need, progress tends to follow, even in uncertain markets.
In an industry that often gravitates toward scale, some of the most meaningful advances still come from tackling the hardest problems first. Rare pediatric cancers are among those problems. Seeing continued engagement from regulators and investors, even amid broader market volatility, gives me reason for optimism.
