Real-World Clinical Profile, Treatment Patterns And Patient-Reported Outcomes for Thyroid Cancer in Japan

Abstract

Aim: To provide a real-world snapshot of the clinical profile, management, and patient-reported outcomes (PRO) for advanced medullary and papillary thyroid cancer prior to the availability of rearranged during transfection (RET) inhibitors in Japan. Materials & methods: Physicians completed patient-record forms for eligible patients seen during routine clinical practice. Physicians were also surveyed about their routine practice and patients were asked to provide PRO data. Results:RET testing patterns varied by hospital type; no therapeutic relevance was a commonly cited reason to not carry out testing. Multikinase inhibitors were the main systemic therapies prescribed, although timing to start multikinase inhibitors varied; adverse events were reported as challenges. PROs revealed high disease/treatment burden. Conclusion: More effective and less toxic systemic treatment targeting genomic alterations is needed to improve long-term outcomes of thyroid cancer.

Plain Language Summary

Thyroid cancer in Japan: which treatments are used in every-day clinical practice & the impact of the disease & treatments on people with this condition

This survey, conducted in Japan in 2020, included doctors who treat thyroid cancer and their patients. It is called a real-world survey because it provides information such as the types of tests and treatments used for thyroid cancer management in everyday clinical practice. The survey focused on two types of thyroid cancer: papillary thyroid cancer (PTC), a common type, and medullary thyroid cancer (MTC), an uncommon type. About 10–20% of people with PTC and most people with MTC have alterations in a gene called RET, which caused the cancer. Laboratory tests can identify these gene alterations, fusions (joining the parts of two different genes) or mutations (changes to a gene’s DNA sequence) and results can help guide treatment decisions. The survey showed that testing for RET gene alterations was less than optimal and varied by the type of hospital/center. Common reasons provided by doctors for not testing for RET alterations were, “no therapeutic relevance for patient management” and “specific targeted therapies not available”. However, the survey was conducted before the availability in Japan of the treatment selpercatinib, which selectively targets/inhibits tumors with RET alterations. Most patients in the survey, including those with RET alterations, received treatment with a type of inhibitor called multikinase inhibitors, as per available guidelines. Doctors considered side effects due to inhibition of multiple targets by multikinase inhibitors to be among areas for improvement needed. People with PTC and MTC also reported substantial burdens (i.e., negative impact on their lives) from the disease/treatment. The researchers concluded that barriers to RET testing need to be overcome, and more effective and less toxic treatments targeting gene alterations are needed to improve long-term outcomes.

Keywords::

• Japan
• management
• real-world
• thyroid cancer

Supplementary data

To view the supplementary data that accompany this paper please visit the journal website at: www.tandfonline.com/doi/suppl/10.2217/fon-2022-1297

Author contributions

N Fukuda and U Kiiskinen contributed to the conception of the project. K Nakamura, U Kiiskinen, I Sanderson, A Rider and K Lewis were involved in the study design. I Sanderson, A Rider and K Lewis were involved in acquisition of the data and I Sanderson analyzed the data. All authors contributed to interpreting the data, drafting the manuscript and have approved the final version for submission.

Financial & competing interests disclosure

This study was funded by Eli Lilly and Company. N Fukuda has received personal fees from Eisai and Eli-Lilly Japan outside the submitted work. Y Tanizawa, K Nakamura, Y Okada, G Segall, U Kiiskinen and N Fasnacht are employees of Eli Lilly and Company with stock options. I Sanderson, A Rider and K Lewis are employees of Adelphi Real World Ltd. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

The authors would like to acknowledge Patrick Moore and Greg Plosker (Rx Communications, Mold, UK) for medical writing assistance with the preparation of this manuscript, which was funded by Eli Lilly and Company.

Ethical conduct of research

A complete description of the methods of the survey has been previously published and validated [10–12]. Using a check box, patients provided informed consent for use of their anonymized and aggregated data for research and publication in scientific journals. Data were collected in such a way that patients and physicians could not be identified directly; all data were aggregated and de-identified before receipt.

This research also obtained ethics approval from the Western Institutional Review Board, study protocol number #AG8757.

Data collection was undertaken in line with European Pharmaceutical Marketing Research Association guidelines [13] and as such it does not require ethics committee approval. Each survey was performed in full accordance with relevant legislation at the time of data collection, including the Health Information Technology for Economic and Clinical Health Act legislation [14].

Data sharing statement

The dataset supporting the conclusions of this article are included within the article (and its additional files).