ACCELERATE-ing access to drugs for adolescents with cancer
medwireNews spoke to Miranda Fidler, from the International Agency for Research on Cancer in Lyon, France, and Simon Davies, Executive Director of the charity Teen Cancer America, about the proposal by the ACCELERATE working group to improve adolescent patients’ access to new cancer drugs, examining the wider implications and potential barriers to implementation.
What are your thoughts on the ACCELERATE initiative?
Fidler: Currently in the United States, adolescents and young adults (AYAs) with cancer are under-represented in clinical trials. Consequently, it remains unclear in many situations what treatment protocols offer this population the best chance of cure, with extrapolations often being made from pediatric or adult settings. As there has been some evidence that survival improvements in AYAs has lagged behind those of both children and older adults , increasing the availability of and participation in clinical trials in this age group is of paramount importance. Initiatives such as ACCELERATE are thus critical for raising awareness, better understanding the complexities associated with this topic, and gaining endorsements for adolescent strategies.
Is the timing right for such an initiative?
Davies: This is an excellent initiative. I was CEO of the Teenage Cancer Trust and Chair of Cancer 52 in the UK before coming out here in 2013 to lead Teen Cancer America. As far back as then we were advocating for this and I have continued to do so here in the USA. Young people have suffered and may not have survived because they were not given access to adult clinical trials. Both industry and regulators should be proactive in ensuring that any current exclusion is eradicated. It will be shameful if this continued.
What are the implications for patients and physicians?
Fidler: There are many implications of this initiative for adolescent cancer patients. For example, it would accelerate the access to new drugs and potentially provide data to inform future drug development. Further, increasing the number of available trials would potentially increase the participation of adolescents in clinical trials, which could in return provide invaluable information for identifying effective treatment protocols and risk-stratification.
With regard to clinicians, a joint network between pediatric and adult oncologists could strengthen the knowledge base in both settings, which in return could result in better access to treatment and improved outcomes for patients.
Are there wider implications?
Davies: Learning about the impact of trials on adolescents can benefit the scientific community's knowledge about a drug and its interaction with a much broader age range. This will provide valuable research outcomes and information that will help adult patients. Particularly the young adults that are close in age to the adolescents.
What issues do you anticipate with joint adolescent–adult trials?
Fidler: Work from the US Centers of Disease Control and Prevention  has suggested that there are distinct issues affecting clinical trial enrollment among adolescents with cancer, which will continue to exist even if adolescent–adult joint trials are launched; two of the most important issues are that (i) many adolescents with cancer are not referred to institutions where clinical trials are offered, and (ii) psychosocial barriers impede adolescents with cancer from enrolling in clinical trials. Further work to increase access to studies and improve the understandability of clinical trials for these individuals is therefore still needed.
While it remains unclear whether adolescent–adult joint clinical trials will increase the participation rate among adolescents, they would increase the number of clinical trials available to individuals with cancer in this age group, which is currently a huge barrier and thus a good first step forward.
What are the potential barriers to launching such trials?
Davies: There are many potential barriers as so much change is needed:
Regulators: The first hurdle. We need to move away from the traditional pediatric and adult divide and concentrate on biology. The fact that setting up pediatric trials can be more complex and expensive is a disincentive. Plus only certain centers can run them. There needs to be much more flexibility and incentives to collaborate.
Policy makers: We have to get away from always thinking of the pediatric and adult health communities as entirely separate. There needs to be policies established by national and international societies and any stakeholder body that influences practice and sets standards to ensure “cross fertilization” and collaboration between pediatric and adult oncology fields.
For example, a 17-year 10-month old patient on a pediatric ward with acute lymphoblastic leukemia will receive the pediatric protocol. However, the 18-year 1-month old patient with the exact same disease and stage could end up on an adult ward with the adult protocol that is proven to have 25% worse outcomes for young adults than the pediatric protocol. This is outrageous and policy makers have it within their grasp to require change.
Pharma: As said above, opening pediatric trials can be expensive and difficult because of the regulatory system. Pharmaceutical companies need to recognize the importance and invest more. And if regulators make it easier then they will do more. Furthermore, there need to be more financial incentives to do research and trials in rare cancers. There is too much investment in “me too” drugs for common cancers because they make profit. The rare cancer community is left behind.
Academic and community hospitals: It is one thing to have a clinical trial but it is something else to set it up. Many community hospitals do not have the resource or expertise to manage clinical trials and yet the majority of cancer patients are treated in community hospitals. There needs to be far more collaboration between academic and community settings. Most adults are treated in community hospitals and they have so many more patients that are eligible but it is academic centers that have the expertise. Collaboration could bring rewards for both types of institution and of course the patents.
Health professionals: Collaboration, communication, and cooperation are critical. The professional pediatric and adult cancer communities need to come together more at a scientific, clinical and organizational level.
Insurance companies (at least in the US): They should appreciate the importance of access to clinical trials and should support this gold standard for as many customers as possible. They have it in their power to make a huge impact by insisting on access to clinical trials for their customers. This will be profitable for them because improved outcomes and survival will bring down health costs.
Patients: They need to be better informed and have their voice heard in this story. They should be empowered to influence and engage.
When can we expect to see such trials?
Davies: The answer to this should be yesterday! As soon as the regulators such as the EMA and FDA allow this to happen, both of them should also direct and incentivize research into rare cancers that affect the young. Then there will be progress.
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- Adolescent and Young Adult Oncology Progress Review Group. Closing the gap: Research and care imperatives for adolescents and young adults with cancer. https://www.cancer.gov/types/aya/research/ayao-august-2006.pdf. Accessed 12 April 2017.
- Tai E, Beaupin L, Bleyer A. Clinical trial enrollment among adolescents with cancer: supplement overview. Pediatrics 2014; 133: S85–S90.