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. 2021 Apr 26;6(2):100096. doi: 10.1016/j.esmoop.2021.100096

Table 3.

Existing areas of consensus and future actions to optimise AYA access to care and clinical trials

Areas of current consensus Historical AYA challenges Progress Outstanding issues Future actions
Availability of drugs and clinical trials Improve early access to new anticancer drugs for AYA.
Increase the number of early-phase trials.
Simplify the process of PIPs.a
Develop trials based on the molecular target and cancer type rather than age.		 Small number of diverse cancer types. Clinical trials focused on tumour type rather than molecular pathway exclude AYA.
Drug development in AYA and children is not as efficient as adult drug development.
PIPs can be waived if pharmaceutical companies believe that the disease is absent in AYA.		 ACCELERATEb initiative to favour mechanism-of-action trials, based on the biology of the disease.
ACCELERATE initiative to suppress article 11b of the European Paediatric Regulation.		 Companies can still apply for PIPs and not develop a drug in the child/adolescent population if the disease under study is non-existent in this population. They do not consider potential similar targets.
Drugs are being used off-label in adolescents with little safety or efficacy data.
Limited information about the biology of cancer in AYA and drug resistance.
  • •

    Develop drugs simultaneously across the whole age range of a disease or target pathway.

  • •

    Suppress article 11b.

  • •

    Do not issue waivers without scrutinising potential action in children and adolescents.

  • •

    Prospective data collection for off-label use.

  • •

    Identify new therapeutic targets for drug development.
Appropriateness of age eligibility criteria Arbitrary eligibility criteria should only exist where there is a biological rationale or safety concerns/evidence.
Improve access to drugs in early-phase trials.		 Many AYA fall between adult and paediatric trials and are excluded based on age eligibility criteria.
Pharmaceutical industry-sponsored trials predominately focus on older adults with a lower age limit of 18 years.		 ACCELERATE initiative to support the inclusion of adolescents aged ≥12 years in early adult phase I/II trials including first-in-class trials.
A number of joint paediatric/adult trials have been developed and have successfully recruited adolescents, and to some extent, young adults.		 The number of joint paediatric/adult trials developed has been small.
The lower age eligibility criterion of 18 years in trials has not been abolished, particularly in industry-sponsored registration trials. The upper age eligibility criterion in some paediatric trials remains.
Trials initiated by paediatric and adult oncology researchers in the same cancer type may overlap, creating confusion for the AYA.
Increased collaboration between adult and paediatric trialists is essential.
  • •

    Provide guidance to support paediatric and adult oncologists to work together.

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    Stop upper/lower age eligibility criteria being set in drug trials for cancers.

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    Support AYA recruitment into clinical trials which span both paediatric and adult populations.
Access to trials Relevant clinical trials should include AYA and AYA-appropriate care.
Adolescents ≥12 years of age should not be excluded from adult trials, based only on age criteria.		 Access to trials has been affected by the place of treatment (adult versus paediatric ward).
Limited access to adult early-phase trials.
Special skills required to obtain consent for AYA to participate in trials.		 Development of dedicated AYA hospitals and/or care networks.
Allows centralisation of care, AYA expertise and access to relevant trials.		 Access to specialist AYA care is not equitable.
No central AYA trials register.
Researchers tend to be trained in either the paediatric or adult setting and are unfamiliar with the process for consenting AYA into clinical trials.
  • •

    Establish a portal of available AYA trials and guidance on referrals to centres with open trials.

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    Develop a cohort of researchers competent at consenting AYA into clinical trials.
Enrolment into clinical trials Ensure young people and patient advocates are engaged in trial design.
Ensure research questions and endpoints are relevant to AYA needs.
Ensure patient information and consent processes are age appropriate.		 Involving young people in trial design can be resource intensive.
Traditional outcomes, such as survival, are required for regulatory approval. Some AYA cancers have excellent survival rates and trials on quality of life and late toxicities are paramount.		 Funding for patient and public involvement has been provided.
A number of patient groups are involved in clinical trial design.
Several studies have been successfully completed with quality of life and reducing treatment burden as primary endpoints.		 Limited awareness among patients and physicians regarding available clinical trials for AYA.
  • •

    Educate health care providers and other disciplines regarding the benefits of participating in clinical trials for AYA patients.

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    Engage patient advocates.

AYA, adolescents and young adults; PIP, paediatric investigation plan.

a

A PIP is a development plan aimed at ensuring that the necessary data are obtained through studies in children to support the authorisation of a medicine for children (https://www.ema.europa.eu/en/human-regulatory/research-development/paediatric-medicines/paediatric-investigation-plans).