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Global Development of Anticancer Therapies for Rare Cancers, Pediatric Cancers, and Molecular Subtypes of Common Cancers.

Publication ,  Journal Article
Lyerly, HK; Ren, J; Canetta, R; Kim, GH; Nagai, S; Yamaguchi, T; Hatogai, K; Katayama, H; Da Rocha Dias, S; McManus, D; Soltys, K; Yang, Z ...
Published in: J Glob Oncol
December 2018

Advances in genetic sequencing and other diagnostic technologies have enabled the use of precision medicine in clinical cancer care, as well as the development of novel therapies that are targeted to specific molecular drivers of cancer. Developing these new agents and making them accessible to patients requires global clinical studies and regulatory review and approval by different national regulatory agencies. Whereas these global trials present challenges for drug developers who conduct them and regulatory agencies who oversee them, they also raise practical issues about patients with low-frequency cancers who need these therapies. A lack of uniform standards in both regulatory approval for marketing and reimbursement for approved agents across countries may make the newly developed agent either unavailable or inaccessible to patients in certain countries or regions, even if patients from those countries or regions participated in the clinical research that established the safety and efficacy of the agent. In an effort to further understand and address this need, we convened an international workshop in 2017 in North Bethesda, MD. After presentations of the individual regulatory pathways for marketing approval and reimbursement for individual nations, participants discussed expedited pathways and specific challenges for uncommon cancers. As a matter of justice, agents being developed for rare cancers, pediatric cancers, or uncommon molecular subsets of common cancers need a pragmatic, science-based regulatory policy framework to clearly specify the type and quantity of evidence needed to demonstrate efficacy from these trials and evidence to support accessibility.

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Published In

J Glob Oncol

DOI

EISSN

2378-9506

Publication Date

December 2018

Volume

4

Start / End Page

1 / 11

Location

United States

Related Subject Headings

  • Young Adult
  • Neoplasms
  • Humans
  • Child
  • Antineoplastic Agents
  • Adult
  • Adolescent
  • 4206 Public health
  • 3211 Oncology and carcinogenesis
 

Citation

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Lyerly, H. K., Ren, J., Canetta, R., Kim, G. H., Nagai, S., Yamaguchi, T., … Gross, T. (2018). Global Development of Anticancer Therapies for Rare Cancers, Pediatric Cancers, and Molecular Subtypes of Common Cancers. J Glob Oncol, 4, 1–11. https://doi.org/10.1200/JGO.18.00092
Lyerly, H Kim, Jun Ren, Renzo Canetta, Gi Hyun Kim, Sumimasa Nagai, Tomohiro Yamaguchi, Ken Hatogai, et al. “Global Development of Anticancer Therapies for Rare Cancers, Pediatric Cancers, and Molecular Subtypes of Common Cancers.J Glob Oncol 4 (December 2018): 1–11. https://doi.org/10.1200/JGO.18.00092.
Lyerly HK, Ren J, Canetta R, Kim GH, Nagai S, Yamaguchi T, et al. Global Development of Anticancer Therapies for Rare Cancers, Pediatric Cancers, and Molecular Subtypes of Common Cancers. J Glob Oncol. 2018 Dec;4:1–11.
Lyerly, H. Kim, et al. “Global Development of Anticancer Therapies for Rare Cancers, Pediatric Cancers, and Molecular Subtypes of Common Cancers.J Glob Oncol, vol. 4, Dec. 2018, pp. 1–11. Pubmed, doi:10.1200/JGO.18.00092.
Lyerly HK, Ren J, Canetta R, Kim GH, Nagai S, Yamaguchi T, Hatogai K, Katayama H, Da Rocha Dias S, McManus D, Soltys K, Yang Z, Olopade O, Goodman N, Reaman G, Gross T. Global Development of Anticancer Therapies for Rare Cancers, Pediatric Cancers, and Molecular Subtypes of Common Cancers. J Glob Oncol. 2018 Dec;4:1–11.

Published In

J Glob Oncol

DOI

EISSN

2378-9506

Publication Date

December 2018

Volume

4

Start / End Page

1 / 11

Location

United States

Related Subject Headings

  • Young Adult
  • Neoplasms
  • Humans
  • Child
  • Antineoplastic Agents
  • Adult
  • Adolescent
  • 4206 Public health
  • 3211 Oncology and carcinogenesis