Regulations, Open Data and Healthcare Innovation: A Case of MSK-IMPACT and Its Implications for Better Cancer Care
Abstract
:Simple Summary
Abstract
1. Introduction
1.1. Next-Generation Sequencing (NGS) for Advanced Medicine
1.2. Regulatory Reforms for the Pharmaceutical Industry
1.3. Open Data and Healthcare Innovation
1.4. Purpose of the Study
2. Materials and Methods
2.1. The Case
2.2. Document-Based Analysis
2.3. Comparative Analysis
2.4. Interview-Based Analysis
3. Results
3.1. FDA’s Regulatory Reforms and Their Outcomes
3.2. The Contribution of Open Databases
3.3. An Early Application of the New Technologies
3.4. The Utilization Structure of MSK-IMPACT
3.5. Comparison between MSK-IMPACT and Other Panel Tests
4. Discussion
4.1. Implications of Regulatory Reforms to Corporate Activities
4.2. Characteristics of MSK-IMPACT
4.3. Recommendations for a Better Clinical Sequencing in Oncology
4.4. Study Limitations
5. Conclusions
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
- The Precision Medicine Initiative. Available online: https://obamawhitehouse.archives.gov/precision-medicine (accessed on 18 September 2020).
- Wetterstrand, K.A. DNA Sequencing Costs: Data from the NHGRI Genome Sequencing Program (GSP). Available online: https://www.genome.gov/about-genomics/fact-sheets/DNA-Sequencing-Costs-Data (accessed on 19 April 2021).
- The OECD Innovation Strategy: Getting a Head Start on Tomorrow. Available online: https://read.oecd-ilibrary.org/science-and-technology/the-oecd-innovation-strategy_9789264083479-en#page1 (accessed on 20 April 2021).
- Onodera, R.; Sengoku, S. Innovation process of mHealth: An overview of FDA-approved mobile medical applications. Int. J. Med Inform. 2018, 118, 65–71. [Google Scholar] [CrossRef]
- Coté, T.; Kelkar, A.; Xu, K.; Braun, M.M.; Phillips, M.I. Orphan products: An emerging trend in drug approvals. Nat. Rev. Drug Discov. 2010, 9, 84. [Google Scholar] [CrossRef] [Green Version]
- PUBLIC LAW 97-414. Available online: https://www.govinfo.gov/content/pkg/STATUTE-96/pdf/STATUTE-96-Pg2049.pdf (accessed on 8 July 2021).
- Food and Drug Administration Modernization Act of 1997. Available online: https://www.govinfo.gov/content/pkg/PLAW-105publ115/pdf/PLAW-105publ115.pdf (accessed on 8 July 2021).
- Accelerated Approval. Available online: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/accelerated-approval (accessed on 8 July 2021).
- PUBLIC LAW 112-144. Available online: https://www.govinfo.gov/content/pkg/PLAW-112publ144/pdf/PLAW-112publ144.pdf (accessed on 8 July 2021).
- Darrow, J.J.; Avorn, J.; Kesselheim, A.S. FDA Approval and Regulation of Pharmaceuticals, 1983–2018. Am. Med Assoc. 2020, 323, 164–176. [Google Scholar] [CrossRef]
- Darrow, J.J.; Avorn, J.; Kesselheim, A.S. New FDA Breakthrough-Drug Category—Implications for Patients. N. Engl. J. Med. 2014, 370, 1252–1258. [Google Scholar] [CrossRef] [Green Version]
- Golodner, L.F. The US Food and Drug Administration Modernization Act of 1997: Impact on consumers. Clin. Ther. 1998, 20, C20–C25. [Google Scholar] [CrossRef]
- Merrill, R.A. Modernizing the FDA: An Incremental Revolution. Health Aff. 1999, 18, 96–111. [Google Scholar] [CrossRef] [Green Version]
- Goble, J.A. The Potential Effect of the 21st Century Cures Act on Drug Development. J. Manag. Care Spec. Pharm. 2018, 24, 677–681. [Google Scholar] [CrossRef]
- Goodsell, D.S.; Zardecki, C.; Costanzo, L.D.; Duarte, J.M.; Hudson, B.P.; Persikova, I.; Segura, J.; Shao, C.; Voigt, M.; Westbrook, J.D.; et al. RCSB Protein Data Bank: Enabling biomedical research. Protein Soc. 2020, 29, 52–65. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Kahn, S.M. Next-generation sequencing for cancer drug development: The present and visions for the future. Pers. Med. 2014, 11, 139–142. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Patriquin, C.M. A Case Study of Next-Generation Sequencing Operationalization in an Oncology Companion Diagnostic Environment. Master’s Thesis, Harvard Extension School, Cambridge, MA, USA, May 2020. [Google Scholar]
- ClinicalTrials. Available online: https://clinicaltrials.gov/ (accessed on 19 January 2019).
- Risk Evaluation and Mitigation Strategies|REMS. Available online: https://www.fda.gov/drugs/drug-safety-and-availability/risk-evaluation-and-mitigation-strategies-rems (accessed on 14 July 2020).
- Platt, R.; Brown, J.S.; Robb, M.; McClellan, M.; Ball, R.; Nguyen, M.D.; Sherman, R.E. The FDA Sentinel Initiative—An Evolving National Resource. N. Engl. J. Med. 2018, 379, 2091–2093. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- 21st Century Cures Act. Available online: https://www.fda.gov/regulatory-information/selected-amendments-fdc-act/21st-century-cures-act (accessed on 8 July 2021).
- Hillebrenner, E. Third Party Review: FDA Perspective. Available online: https://www.amdm.org/uploads/8/3/9/2/8392851/hillebrenner_3p_case_study.pdf (accessed on 25 June 2020).
- Public Workshop—Optimizing FDA’s Regulatory Oversight of Next Generation Sequencing Diagnostic Tests Public Workshop. Available online: https://wayback.archive-it.org/7993/20170406155937/https://www.fda.gov/MedicalDevices/NewsEvents/WorkshopsConferences/ucm427296.htm (accessed on 23 September 2020).
- Public Workshop—Standards Based Approach to Analytical Performance Evaluation of Next Generation Sequencing In Vitro Diagnostic Tests. Available online: https://wayback.archive-it.org/7993/20170111165845/http://www.fda.gov/MedicalDevices/NewsEvents/WorkshopsConferences/ucm459449.htm (accessed on 23 September 2020).
- Webinar—Next Generation Sequencing (NGS) Draft Guidances: Implications for Patients and Providers—27 July 2016. Available online: https://www.fda.gov/medical-devices/workshops-conferences-medical-devices/webinar-next-generation-sequencing-ngs-draft-guidances-implications-patients-and-providers-july-27 (accessed on 23 September 2020).
- Use of Public Human Genetic Variant Databases to Support Clinical Validity for Genetic and Genomic-Based In Vitro Diagnostics. Available online: https://www.fda.gov/media/99200/download (accessed on 23 September 2020).
- dbSNP Celebrates 20 Years! Available online: https://ncbiinsights.ncbi.nlm.nih.gov/2019/10/07/dbsnp-celebrates-20-years/ (accessed on 14 September 2020).
- Empowering GWAS for a New Era of Discovery. Available online: https://jp.illumina.com/content/dam/illumina-marketing/documents/products/technotes/technote_empower_gwas.pdf (accessed on 14 September 2020).
- PubMed. Available online: https://pubmed.ncbi.nlm.nih.gov/ (accessed on 7 October 2019).
- Participating in the Harvard PGP. Available online: https://pgp.med.harvard.edu/participate (accessed on 16 September 2020).
- McVean, G.A. An integrated map of genetic variation from 1092 human genomes. Nature 2012, 491, 56–65. [Google Scholar]
- Mitsubishi Research Institute, Inc. Heisei Niju-Nana Nendo Kokunaigai ni Okeru Idenshi Shinryo no Jittai Chosa Houkokusyo—Kenkyu Iryomen ni Oite Yuyoh na Data Sharing Kiban no Kento. Available online: https://www.amed.go.jp/content/000004863.pdf (accessed on 17 September 2020). (In Japanese).
- Alexandrov, L.B.; Nik-Zainal, S.; Stratton, M.R.; Wedge, D.C.; Aparicio, S.A.J.R.; Behjati, S.; Biankin, A.V.; Bignell, G.R.; Bolli, N.; Borg, A.; et al. Signatures of mutational processes in human cancer. Nature 2013, 500, 415–421. [Google Scholar] [CrossRef] [Green Version]
- Gargis, A.S.; Kalman, L.; Berry, M.W.; Bick, D.P.; Dimmock, D.P.; Hambuch, T.; Lu, F.; Lyon, E.; Voelkerding, K.V.; Zehnbauer, B.A.; et al. Assuring the quality of next-generation sequencing in clinical laboratory practice. Nat. Biotechnol. 2012, 30, 1033–1036. [Google Scholar] [CrossRef] [PubMed]
- FDA Approves First Companion Diagnostic Test to Simultaneously Screen for Multiple Non-Small Cell Lung Cancer Therapies. Available online: https://thermofisher.mediaroom.com/2017-06-22-FDA-Approves-First-Companion-Diagnostic-Test-to-Simultaneously-Screen-for-Multiple-Non-Small-Cell-Lung-Cancer-Therapies (accessed on 24 September 2020).
- FDA Unveils a Streamlined Path for the Authorization of Tumor Profiling Tests Alongside Its Latest Product Action. Available online: https://www.fda.gov/news-events/press-announcements/fda-unveils-streamlined-path-authorization-tumor-profiling-tests-alongside-its-latest-product-action (accessed on 24 September 2020).
- FDA Approves Foundation Medicine’s FoundationOne CDx™, the First and Only Comprehensive Genomic Profiling Test for All Solid Tumors Incorporating Multiple Companion Diagnostics. Available online: https://www.foundationmedicine.com/press-releases/f2b20698-10bd-4ac9-a5e5-c80c398a57b5 (accessed on 24 September 2020).
- Fda Fact Sheet Cdrh’s Approach to Tumor Profiling Next Generation Sequencing Tests. Available online: https://www.fda.gov/media/109050/download (accessed on 24 September 2020).
- Memorial Sloan Kettering Cancer Center. Available online: https://health.usnews.com/best-hospitals/area/ny/memorial-sloan-kettering-cancer-center-6213060 (accessed on 7 October 2020).
- cBioPortal for Cancer Genomics. Available online: https://www.cbioportal.org/ (accessed on 8 October 2020).
- cBioPortal. Available online: https://github.com/cBioPortal/ (accessed on 8 October 2020).
- Hyman, D.M.; Solit, D.B.; Arcila, M.E.; Cheng, D.; Sabbatini, P.; Baselga, J.; Berger, M.F.; Ladanyi, M. Precision medicine at Memorial Sloan Kettering Cancer Center: Clinical next-generation sequencing enabling next-generation targeted therapy trials. Drug Discov. Today 2015, 20, 1422–1428. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Cancer Genomics. Available online: https://www.thehyve.nl/focus-areas/cancer-genomics (accessed on 3 June 2021).
- Chakravarty, D.; Gao, J.; Phillips, S.; Kundra, R.; Zhang, H.; Wang, J.; Rudolph, J.E.; Yaeger, R.; Soumerai, T.; Nissan, M.H.; et al. OncoKB: A Precision Oncology Knowledge Base. JCO Precis. Oncol. 2017. [Google Scholar] [CrossRef] [PubMed]
- Early Drug Development Service: Phase I Clinical Trials Program. Available online: https://www.mskcc.org/departments/division-solid-tumor-oncology/early-drug-development-service-phase-clinical-trials (accessed on 16 October 2020).
- MSK-IMPACT: A Targeted Test for Mutations in Both Rare and Common Cancers. Available online: https://www.mskcc.org/msk-impact (accessed on 20 November 2020).
- Eubank, M.H.; Hyman, D.M.; Kanakamedala, A.D.; Gardos, S.M.; Wills, J.M.; Stetson, P.D. Automated eligibility screening and monitoring for genotype-driven precision oncology trials. J. Am. Med. Inform. Assoc. 2016, 23, 777–781. [Google Scholar] [CrossRef] [Green Version]
- Zehir, A.; Benayed, R.; Shah, R.H.; Syed, A.; Middha, S.; Kim, H.R.; Srinivasan, P.; Gao, J.; Chakravarty, D.; Delvin, S.M.; et al. Mutational landscape of metastatic cancer revealed from prospective clinical sequencing of 10,000 patients. Nat. Med. 2017, 23, 703–713. [Google Scholar] [CrossRef]
- MSK Program Focuses on Speeding Up Development of New Leukemia Treatments. Available online: https://www.mskcc.org/news/msk-program-focuses-speeding-up-new-leukemia-treatments (accessed on 20 November 2020).
- Clinical Trial Shows Promise of “Basket Studies” for Cancer Drugs. Available online: https://www.mskcc.org/news/clinical-trial-shows-promise-basket-studies-drugs (accessed on 10 December 2020).
- Kim, G.; McKee, A.E.; Ning, Y.M.; Hazarika, M.; Theoret, M.; Johnson, J.R.; Xu, Q.C.; Tang, S.; Sridhara, R.; Jiang, X.; et al. FDA Approval Summary: Vemurafenib for Treatment of Unresectable or Metastatic Melanoma with the BRAFV600E Mutation. Clin. Cancer Res. 2014, 20, 4994–5000. [Google Scholar] [CrossRef] [Green Version]
- Hyman, D.M.; Puzanov, I.; Subbiah, V.; Faris, J.E.; Chau, I.; Blay, J.Y.; Wolf, J.; Raje, N.S.; Diamond, E.L.; Hollebecque, A.; et al. Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations. N. Engl. J. Med. 2015, 373, 726–736. [Google Scholar] [CrossRef]
- Patricia, O.A.; Kwitkowski, V.; Luo, L.; Shen, Y.L.; Subramaniam, S.; Shord, S.; Goldberg, K.B.; McKee, A.E.; Kaminskas, E.; Farrell, A.; et al. FDA Approval Summary: Vemurafenib for the Treatment of Patients with Erdheim-Chester Disease with the BRAFV600 Mutation. Oncologist 2018, 23, 1520–1524. [Google Scholar]
- FDA Approves First Treatment for Certain Patients with Erdheim-Chester Disease, a Rare Blood Cancer. Available online: https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-certain-patients-erdheim-chester-disease-rare-blood-cancer#:~:text=The%20U.S.%20Food%20and%20Drug%20Administration%20today%20expanded,a%20specific%20genetic%20mutation%20known%20as%20BRAF%20V600 (accessed on 10 December 2020).
- List of Cleared or Approved Companion Diagnostic Devices (In Vitro and Imaging Tools). Available online: https://www.fda.gov/medical-devices/vitro-diagnostics/list-cleared-or-approved-companion-diagnostic-devices-vitro-and-imaging-tools (accessed on 18 December 2020).
- Genomic Data Solutions. Available online: https://www.foundationmedicine.com/service/genomic-data-solutions (accessed on 25 November 2020).
- FoundationOne®CDx Technical Information. Available online: https://info.foundationmedicine.com/hubfs/FMI%20Labels/FoundationOne_CDx_Label_Technical_Info.pdf (accessed on 3 June 2021).
- Evaluation of Automatic Class III Designation for Msk-Impact (Integrated Mutation Profiling of Actionable Cancer Targets) Decision Summary. Available online: https://www.accessdata.fda.gov/cdrh_docs/reviews/den170058.pdf (accessed on 3 June 2021).
- Oncomine™ Dx Target Test Part I: Sample Preparation and Quantification User Guide. Available online: https://www.accessdata.fda.gov/cdrh_docs/pdf16/p160045c.pdf (accessed on 25 April 2021).
- Summary of Safety and Effectiveness Data (SSED). Available online: https://www.accessdata.fda.gov/cdrh_docs/pdf16/p160045b.pdf (accessed on 3 June 2021).
- Abou-Alfa, G.K.; Sahai, V.; Hollebecque, A.; Vaccaro, G.; Melisi, D.; Al-Rajabi, R.; Paulson, A.S.; Borad, M.J.; Gallinson, D.; Murphy, A.G.; et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: A multicentre, open-label, phase 2 study. Lancet Oncol. 2020, 21, 671–684. [Google Scholar] [CrossRef]
- FDA Approves First Targeted Treatment for Patients with Cholangiocarcinoma, a Cancer of Bile Ducts. Available online: https://www.fda.gov/news-events/press-announcements/fda-approves-first-targeted-treatment-patients-cholangiocarcinoma-cancer-bile-ducts (accessed on 8 February 2021).
- FDA Approves Gilotrif® (Afatinib) as New Oral Treatment Option for Patients with Squamous Cell Carcinoma of the Lung. Available online: https://www.boehringer-ingelheim.us/press-release/fda-approves-gilotrifr-afatinib-new-oral-treatment-option-patients-squamous-cell (accessed on 14 February 2021).
- Soria, J.C.; Felip, E.; Cobo, M.; Lu, S.; Syrigos, K.; Lee, K.H.; Goker, E.; Georgoulias, V.; Li, W.; Isla, D.; et al. Afatinib versus erlotinib as second-line treatment of patients with advanced squamous cell carcinoma of the lung (LUX-Lung 8): An open-label randomised controlled phase 3 trial. Lancet Oncol. 2015, 16, 897–907. [Google Scholar] [CrossRef] [Green Version]
- NDA/BLA Multi-Disciplinary Review and Evaluation NDA 212725 Rozlytrek (Entrectinib). Available online: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212725Orig1s000,%20212726Orig1s000MultidisciplineR.pdf (accessed on 1 February 2021).
- FDA Approves Entrectinib for NTRK Solid Tumors and ROS-1 NSCLC. Available online: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-entrectinib-ntrk-solid-tumors-and-ros-1-nsclc#:~:text=On%20August%2015%2C%202019%2C%20the%20Food%20and%20Drug,following%20treatment%20or%20have%20no%20satisfactory%20standard%20th (accessed on 15 August 2019).
- Prescribing Information of KEYTRUDA. Available online: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/125514s071s090lbl.pdf (accessed on 1 February 2021).
- FDA Approves Pembrolizumab for Adults and Children with TMB-H Solid Tumors. Available online: https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-pembrolizumab-adults-and-children-tmb-h-solid-tumors (accessed on 8 February 2021).
- Arora, S.; Balasubramaniam, S.; Zhang, H.; Berman, T.; Narayan, P.; Suzman, D.; Bloomquist, E.; Tang, S.; Gong, Y.; Sridhara, R.; et al. FDA Approval Summary: Olaparib Monotherapy or in Combination with Bevacizumab for the Maintenance Treatment of Patients with Advanced Ovarian Cancer. Oncologist 2021, 26, e164–e172. [Google Scholar] [CrossRef]
- FDA Approved Olaparib (LYNPARZA, AstraZeneca Pharmaceuticals LP). Available online: https://www.fda.gov/drugs/fda-approved-olaparib-lynparza-astrazeneca-pharmaceuticals-lp-maintenance-treatment-adult-patients (accessed on 18 February 2021).
- Prescribing Information of TABRECTA. Available online: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213591s000lbl.pdf (accessed on 8 February 2021).
- FDA Approves First Targeted Therapy to Treat Aggressive Form of Lung Cancer. Available online: https://www.fda.gov/news-events/press-announcements/fda-approves-first-targeted-therapy-treat-aggressive-form-lung-cancer (accessed on 8 February 2021).
- De Bono, J.; Mateo, J.; Fizazi, K.; Saad, F.; Shore, N.; Sandhu, S.; Chi, K.N.; Sartor, O.; Agarwal, N.; Olmos, D.; et al. Olaparib for Metastatic Castration-Resistant Prostate Cancer. N. Engl. J. Med. 2020, 382, 2091–2102. [Google Scholar] [CrossRef]
- FDA Approves Olaparib for HRR Gene-Mutated Metastatic Castration-Resistant Prostate Cancer. Available online: https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-olaparib-hrr-gene-mutated-metastatic-castration-resistant-prostate-cancer#:~:text=On%20May%2019%2C%202020%2C%20the,(mCRPC)%2C%20who%20have%20progressed (accessed on 18 February 2021).
- Drilon, A.; Laetsch, T.W.; Kummar, S.; DuBois, S.G.; Lassen, U.N.; Demetri, G.D.; Nathenson, M.; Doebele, R.C.; Farago, A.F.; Pappo, A.S.; et al. Efficacy of Larotrectinib in TRK Fusion–Positive Cancers in Adults and Children. N. Engl. J. Med. 2018, 378, 731–739. [Google Scholar] [CrossRef] [PubMed]
- FDA Approves Larotrectinib for Solid Tumors with NTRK Gene Fusions. Available online: https://www.fda.gov/drugs/fda-approves-larotrectinib-solid-tumors-ntrk-gene-fusions (accessed on 8 February 2021).
- Cheng, M.L.; Berger, M.F.; Hyman, D.M.; Solit, D.B. Clinical tumour sequencing for precision oncology: Time for a universal strategy. Nat. Rev. Cancer 2018, 18, 527–528. [Google Scholar] [CrossRef] [PubMed]
- FDA Approves Neratinib for Metastatic HER2-Positive Breast Cancer. Available online: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-neratinib-metastatic-her2-positive-breast-cancer (accessed on 18 February 2021).
- O’Reilly, E.M.; Hechtman, J.H. Tumour response to TRK inhibition in a patient with pancreatic adenocarcinoma harbouring an NTRK gene fusion. Ann. Oncol. 2019, 30, viii36–viii40. [Google Scholar] [CrossRef] [Green Version]
- Prescribing Information of GAVRETO. Available online: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213721s000lbl.pdf (accessed on 8 February 2021).
- FDA Approves Pralsetinib for Lung Cancer with RET Gene Fusions. Available online: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pralsetinib-lung-cancer-ret-gene-fusions (accessed on 8 February 2021).
- Odogwu, L.; Mathieu, L.; Blumenthal, G.; Larkins, E.; Goldberg, K.B.; Griffin, N.; Bijwaard, K.; Lee, E.Y.; Philip, R.; Jian, X.; et al. FDA Approval Summary: Dabrafenib and Trametinib for the Treatment of Metastatic Non-Small Cell Lung Cancers Harboring BRAF V600E Mutations. Oncologist 2018, 23, 740–745. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Cortellis. Available online: https://www.cortellis.com/intelligence/home.do (accessed on 24 April 2021).
- Application Number: 213721Orig1s000 Administrative and Correspondence Documents. Available online: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213721Orig1s000AdminCorres.pdf (accessed on 25 April 2021).
- Application Number: 213736Orig1s000 Multi-Discipline Review. Available online: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213736Orig1s000MultidisciplineR.pdf (accessed on 25 April 2021).
- Application Number: 213591Orig1s000 Administrative and Correspondence Documents. Available online: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213591Orig1s000AdminCorres.pdf (accessed on 25 April 2021).
- Application Number: 210861Orig1s000 211710Orig1s000 MULTI-Discipline Review. Available online: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/210861Orig1s000_211710Orig1s000MultidisciplineR.pdf (accessed on 25 April 2021).
- Mirnezami, R.; Nicholson, J.; Darzi, A. Preparing for Precision Medicine. N. Engl. J. Med. 2012, 366, 489–491. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Chakradhar, S. Tumor sequencing takes off, but insurance reimbursement lags. Nat. Med. 2014, 20, 1220–1221. [Google Scholar] [CrossRef]
- Makino, T.; Lim, Y.; Kodama, K. Strategic R&D transactions in personalized drug development. Drug Discov. Today 2018, 23, 1334–1339. [Google Scholar] [PubMed]
- Sengoku, S.; Sakurai, M.; Yashiro, Y. Japan’s regulatory framework: Seeking to provide impetus to the commercialization of regenerative medicine products. Cell Gene Ther. Insights 2015, 1, 83–92. [Google Scholar] [CrossRef]
Category | Stakeholders/Catalysts | Major Roles in Cancer Care Innovation |
---|---|---|
Regulatory authority | U.S. Food and Drug Administration (FDA) | Take responsibilities to set out and periodically reform pharmaceutical and medical devices regulations |
Assay developer | Foundation Medicine, Inc., Life Technologies Corporation, Memorial Sloan Kettering Cancer Center (MSKCC) | Develop and commercialize tumor profiling tests |
Developer of public data sharing platform | National Center for Biotechnology Information (NCBI), National Human Genome Research Institute | Establish and provide access to publicly accessible open data through international research programs/projects |
International research program/project | Cancer Genome Atlas, Genome Reference Consortium, International HapMap Project, Personal Genome Project, 1000 Genome Project | Help researchers obtain genetic information from cancer patients |
Drug manufacturer | Pharmaceutical companies (i.e., Roche Holding AG, Basel, Switzerland) | Develop new drugs and/or add new indications to the existing drugs on the basis of the use of tumor profiling tests |
Healthcare institution | Hospitals providing healthcare services (i.e., MSKCC) | Provide cancer patients with opportunities for cancer care and clinical trials |
Direct beneficiary of healthcare innovation | Cancer patients | Provide genetic data and use newly developed cancer therapies through clinical trials |
Item | FoundationOne CDx [37,55,56,57] | MSK-IMPACT [36,45,46,58] | Oncomine Dx Target Test [35,55,59,60] |
---|---|---|---|
Developer | Foundation Medicine, Inc. | Memorial Sloan Kettering Cancer Center (MSKCC) | Life Technologies Corporation |
Date of FDA approval as IVD | 30 November 2017 | 15 November 2017 | 22 June 2017 |
Specimen type | FFPE tumor tissue | FFPE tumor tissue and patient-matched blood/normal tissue as a normal control | FFPE tumor tissue |
Number of genes covered | 324 | 468 | 23 |
Biomarker | SNVs, Indels, CNVs, gene rearrangements, TMB, MSI and HRD | SNVs, Indels, CNVs, Promoter mutation (TERT), Gene rearrangements, TMB and MSI | SNVs, Deletions and Fusion gene (ROS1) |
FDA approval for CDx | Granted for diagnosis of breast cancer, cholangiocarcinoma, colon/rectum cancer, non-small cell lung cancer (NSCLC), malignant melanoma, ovary cancer, prostate cancer, and solid cancer | None | Granted for diagnosis of non-small cell lung cancer (NSCLC) |
Availability and functions of data management and/or sharing platform for new drug development | Allows access to open data on cancer patients through FoundationInsights, a cloud-based data platform. Provides access to FoundationCore through FoundationInsights, a knowledgebase with data obtained from cancer patients | Facilitates research for the development of new therapies through cBioPortal for Cancer Genomics (open database) Provides and updates clinical data obtained from cancer patients through OncoKB (knowledgebase) Allows patients to access Phase 1 clinical trials for solid tumors identified by the DARWIN Cohort Management System Manages study cohorts for clinical trials on a timely basis | Analyzes and reports sequencing data through the Torrent Suit Dx Software, which works on Google Chrome browser Allows sequencing results and reports to be automatically archived to an external server |
Product Name | New Drugs | Expanded Additional Indications to Existing Drugs | ||||||
---|---|---|---|---|---|---|---|---|
Drug Name | Biomarker | Indication/Therapy Type | Status | Drug Name | Biomarker | Indication/Therapy Type | Status | |
FoundationOne CDx | PEMAZYRE® (pemigatinib) [61,62] | FGFR2 | Cholangiocarcinoma/Monotherapy | Approved (April 2020) | GILOTRIF® (afatinib) [63,64] | EGFR | Squamous cell carcinoma (lung)/Monotherapy | Approved (April 2016) |
ROZLYTREK® (entrectinib) [65,66] | NTRK | Solid tumors/Monotherapy | Approved (August 2019) | KEYTRUDA® (pembrolizumab) [67,68] | TMB | TMB-H solid tumors/Monotherapy | Approved (June 2020) | |
ROS1 | Non-small cell lung cancer (NSCLC)/Monotherapy | LYNPARZA® (olaparib) | BRCA1/2 | Ovarian cancer/Monotherapy [69,70] | Approved (December 2018) | |||
TABRECTA™ (capmatinib) [71,72] | Mutation relating to MET exon 14 skipping | Non-small cell lung cancer (NSCLC)/Monotherapy | Approved (May 2020) | HRR genes | mCRPC/Monotherapy [73,74] | Approved (May 2020) | ||
VITRAKVI® (larotrectinib) [75,76] | NTRK | Solid tumors/Monotherapy | Approved (November 2018) | ZELBORAF® (vemulafenib) [52,54] | BRAF V600 | Erdheim-Chester disease (ECD)/Monotherapy | Approved (November 2017) | |
MSK-IMPACT | AZD5363 (capivasertib) [18,77] | AKT1/2/3 | Multiple indications (breast cancer, prostate cancer, solid tumors, etc.)/Either monotherapy or combination | Phase 1~ (as of April 2021) | NERLYNX® (neratinib) with XELODA® (capecitabine) [77,78] | HER2 | Breast cancer/Combination | Approved (February 2020) |
LOXO-195 (selitrectinib) [18,79] | NTRK | Solid tumors (with resistance to Larotrectinib)/Monotherapy | Phase 1/2 (as of April 2021) | - | - | - | - | |
VITRAKVI® (larotrectinib) [75,76] | NTRK | Solid tumors/Monotherapy | Approved (November 2018) | - | - | - | - | |
Oncomine Dx Target Test | GAVRETO™ (pralsetinib) [80,81] | RET | Non-small cell lung cancer (NSCLC)/Monotherapy | Approved (September 2020) | TAFINLAR® (dabrafenib) with MEKINIST® (trametinib) [82] | BRAF V600 E | Non-small cell lung cancer (NSCLC)/Combination | Approved (June 2017) |
Drug Information | Expedited Approval Programs [83] | ||||||||
---|---|---|---|---|---|---|---|---|---|
Drug Name (Generic Name) | Assay Used for Clinical Trials | Indication | IND Submission Date | Approval Date | Orphan Drug | Fast Track | Breakthrough Therapy | Priority Review | Accelerated Approval |
GAVRETO™ (pralsetinib) | Oncomine Dx Target Test | Non-small cell lung cancer (NSCLC) | August 2019 [84] | September 2020 [81] | Yes | No | Yes | Yes | Yes |
PEMAZYRE® (pemigatinib) | FoundationOne CDx | Cholangiocarcinoma | January 2018 [85] | April. 2020 [62] | Yes | No | Yes | Yes | Yes |
ROZLYTREK® (entrectinib) | FoundationOne CDx | Solid tumors | February 2014 [83] | August 2019 [66] | Yes | No | Yes | Yes | Yes |
Non-small cell lung cancer (NSCLC) | May 2017 [65] | August 2019 [66] | Yes | No | No | Yes | Yes | ||
TABRECTA™ (capmatinib) | FoundationOne CDx | Non-small cell lung cancer (NSCLC) | January 2015 [86] | May 2020 [72] | Yes | No | Yes | No | Yes |
VITRAKVI® (larotrectinib) | FoundationOne CDx, MSK-IMPACT™ | Solid tumors | February 2014 [87] | November 2018 [76] | Yes | No | Yes | Yes | Yes |
Publisher’s Note: MDPI stays neutral with regard to jurisdictional claims in published maps and institutional affiliations. |
© 2021 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
Share and Cite
Jibiki, T.; Nishimura, H.; Sengoku, S.; Kodama, K. Regulations, Open Data and Healthcare Innovation: A Case of MSK-IMPACT and Its Implications for Better Cancer Care. Cancers 2021, 13, 3448. https://doi.org/10.3390/cancers13143448
Jibiki T, Nishimura H, Sengoku S, Kodama K. Regulations, Open Data and Healthcare Innovation: A Case of MSK-IMPACT and Its Implications for Better Cancer Care. Cancers. 2021; 13(14):3448. https://doi.org/10.3390/cancers13143448
Chicago/Turabian StyleJibiki, Takaharu, Hayato Nishimura, Shintaro Sengoku, and Kota Kodama. 2021. "Regulations, Open Data and Healthcare Innovation: A Case of MSK-IMPACT and Its Implications for Better Cancer Care" Cancers 13, no. 14: 3448. https://doi.org/10.3390/cancers13143448
APA StyleJibiki, T., Nishimura, H., Sengoku, S., & Kodama, K. (2021). Regulations, Open Data and Healthcare Innovation: A Case of MSK-IMPACT and Its Implications for Better Cancer Care. Cancers, 13(14), 3448. https://doi.org/10.3390/cancers13143448