Simone, Joseph V. MD
JOSEPH V. SIMONE, MD
Almost 10 years ago I wrote my first column for Oncology Times: “Pediatric Oncology, Quo Vadis?” (9/25/03). Despite that pretentious title, I attempted to celebrate the enormous success that has been achieved in pediatric cancer care, and provided what I thought were some of the reasons for that success. The latter included a biological advantage that many, if not most, pediatric cancers were sensitive to chemotherapy and radiation.
I also credited the structure of care based mainly in multidisciplinary centers, the widespread participation in clinical trials, and the collaborative and cooperative spirit among pediatric oncologists. And I believed that the relatively small number of patients and the institutional base for care meant that pediatric oncology was not a lucrative sub-specialty, thus making institutional employment the norm instead of fee-for-service private practice and collaboration rather than competition among providers.
I believed then and now that the standards developed for chemotherapy, radiation, surgery, and pathology to comply with clinical trials systematized the infrastructure of care for all children with cancer, whether treated by a protocol or not, so that all benefitted from the prospective installation of best practices.
But I also raised the issue of Quo Vadis? Where are you going? I thought the merger of the two pediatric cooperative groups, the Children's Cancer Group and the Pediatric Oncology Group, was a bad idea, and I said so then and continue to believe so now. The rationale for the merger was greater efficiency, lowering cost, more patients on trials, and speedier protocol development. None of those goals came to pass, however, and several negative consequences emerged.
My opposition was based mainly on three key issues:
1. Having two groups made it possible to have collegial competition and peer review of each other's work.
2. The merger effectively cut in half the number of leadership opportunities for younger investigators, a key track for those mainly running protocols, and not labs, to get ahead academically and to gain leadership experience. Furthermore, the old timers who head committees rarely volunteer to step down, so stagnation and sclerosis were a major risk.
3. I did not believe that the merger would make a more efficient and productive organization. Mergers, whether in business or academia, often fail, or at best, result in one group dominating the other. According to several of those involved, that is what happened.
Pediatricians in academia also have an underlying handicap. Pediatric departments in most medical schools are financially poor, do not enrich the school or its hospital, and thus get little in the way of resources and attention. In contrast, pediatric departments largely based in and supported by freestanding children's hospitals tend to have more resources and have broader and deeper portfolios because most have excellent philanthropic support and a stronger business environment. Even these freestanding hospital departments are at risk in a rapidly changing economic environment.
ASPHO, the American Society of Pediatric Hematology/Oncology, has been working hard to remedy some of these problems. ASPHO has an excellent webinar series for preparing members for leadership and job opportunities as well as for providing connections to others in the field.
But the members face strong headwinds. In addition to often working in financially weak departments, there is a relatively small membership that is at the lower end of the pay scale. Also, pediatric oncology has been largely disregarded by the pharmaceutical industry, from which the larger cancer organizations earn considerable sums of money for exhibits at their meetings and donations for young investigators.
I was asked to lecture at the Annual ASPHO meeting, held in Miami in April. I was one of three speakers in the Presidential Symposium on the future of pediatric oncology. Charles Mulligan from St. Jude Children's Research Hospital spoke about the amazing advances in the molecular characterization of childhood leukemia and other tumors. Peter Adamson, from the Children's Hospital of Pennsylvania, and also the current chair of the Children's Oncology Group, gave a stimulating talk on the dangers of excessive risk aversion in the search for better therapies.
My own talk was on the future of pediatric oncology, and I speculated on some key factors that I believe will have an impact on its future, for good or ill:
Strengths and Advantages
- Parents will take their child anywhere to obtain excellent care (adult cancer patients often seek a convenient location first).
- Parents are more willing to enroll their child in a clinical trial, and pediatric oncologists are more committed to clinical trials than medical oncologists.
- The pediatric culture is more user-friendly and often provides more services for the patient and family.
- Pediatric oncology has a great record of success.
- Well-integrated multi-disciplinary care is the norm.
- Survivorship programs began in pediatric oncology and are steadily growing.
- Philanthropic funds are obtained more easily for children than for adults with cancer.
- No major new therapeutic agents for ALL (and several other tumors) have been developed in 30 years, and drug companies have no interest in pediatric tumors because of their relative rarity and a scant profit margin.
- Therapy remains too complex, too expensive, and too toxic.
- The research model of cancer therapy is misaligned with the current emphasis on prospective payment and value-driven health care. And insurers may become more aggressive in directing patients and their families to specific programs for care.
- New therapeutic agents like imatinib are very expensive and in the prospective payment strategies may not be obtainable.
- There is no access to modern cancer care for children in most of the world.
- The causes of pediatric cancers are largely unknown, and few systematic studies are under way to answer these critical questions.
- Therapy for many pediatric cancers is mature; revolutionary approaches are less likely because of the risk to those cured by current therapy.
- The COG structure remains inefficient and antithetical to the “nimble” program necessary to identify and implement innovative therapy.
- Molecular characterization of pediatric tumors is expanding rapidly.
- Small molecules like imatinib are not curative thus far, but in children with cancer they may turn out to be excellent in combination with conventional agents, especially for sustaining remissions, and perhaps for use intrathecally because of their favorable toxicity profiles.
- The structure of pediatric oncology makes continued empirical progress possible, since a large fraction of patients are treated according to clinical trials.
- There are too few physician scientists in pediatric oncology and even those often receive training in fundamental discovery science that emulates the last generation of mentors. Even fewer are trained in health care delivery science and clinical effectiveness.
- Too few pediatric oncologists are trained and mentored for leadership roles.
- Pediatric departments in most medical schools are money losers, and so often receive little support.
- The future of biomedical research funding by the NIH is uncertain and likely to decline (Emanuel: JAMA 2013;309:1589-1590).
Pediatric oncology has been remarkably successful in the management of children with cancer. It is deeply rewarding to work for the sake of stricken children around the world. I hope it continues its success and thrives for its own sake, but also for the broader oncology profession, which can learn a lot about excellence in cancer care from pediatric oncology.
But like most of medicine today, it faces an uncertain future. The current generation of leaders must consider these factors and develop strategies to meet those challenges.