ABSTRACT
The innovative work (Research and development) pipeline is a huge cost for pharmaceutical Organizations. In spite of the requirement for more advancement, Research and development profitability has vegetated or decayed over various years.1-3 More present, the industry has not exhaustively evaluated the effect of new developments in pharmaceutical improvement and market get to particularly as far as basic achievement measurements, for example, clinical preliminary productivity, the probability of medication dispatch and patient access. To invigorate activity on this diagnostic issue, we accumulated and translated hard confirmation on the effect of chosen developments estimated against particular achievement measurements. The general objective of the investigation is to invigorate expansive dialog on how the business can utilize inventive methodologies in medicate advancement and market access to enhance proficiency, revive profitability and revitalize supportability.
It is unmistakable in openly evaluating the effect of the most encouraging advancements in sedate improvement on preliminary productivity and accomplishment in dispatch and getting model endorsement around the world. We recommend that it makes convincing, information-driven case for expediting the selection of new market get to forms for drugs. In particular, it demonstrates that the four developments assessed---adaptive trial designs, patient-centric trials, precision medicine trials and real-world data trials reliably convey in contrast to industry achievement touchstone.
- ASCO. Clinical trial design and methodology {Internet}. Alexandria (VA): American Society of Clinical Oncology. Available from: https://www.asco.org/research-progress/clinical-trials/clinicaltrial-resources/clinical-trial-design-and-methodology.Google Scholar
- FDA. Adaptive design clinical trials for drugs and biologics: draft guidance. Silver Spring (MD): US Food and Drug Administration, 2010. Available from: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM201790.pdf.Google Scholar
- CFDA. {Biostatistics guidelines for drug clinical trials}.Google Scholar
- FDA. FDA approves first cancer treatment for any solid tumor with a specific genetic feature {Internet}. Silver Spring (MD): US Food & Drug Administration. Available from:https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm560167.htm.Google Scholar
- Study of Pembrolizumab (MK-3475) in Participants With Advanced Solid Tumors (MK-3475-158/ KEYNOTE-158) {Internet}. Bethesda (MD): ClinicalTrials.gov. Available from: https://clinicaltrials.gov/ct2/show/NCT02628067.Google Scholar
- Investigational immunotherapy trials for solid tumors {Internet}. Kenilworth (NJ): Merck & Co, Inc. Available from: https://keynoteclinicaltrials.com/trials/solid-tumors.Google Scholar
- Ray, T. Basket study supported keytruda pan-cancer indication but challenges remain {Internet}. New York (NY): Genomewide. Available from: https://www.genomeweb.com/cancer/basket-studysupported-keytruda-pan- cancer-indication-challenges-remain.Google Scholar
- A study of vemurafenib in participants with BRAF V600 mutation-positive cancers {Internet}.Bethesda (MD): ClinicalTrials.gov. Available from: https://clinicaltrials.gov/ct2/show/NCT01524978.Google Scholar
- Hyman, M., Blay, J. Y., Chau, I., et al. VE-BASKET, a first-in-kind, phase II, histology-independent "basket" study of vemurafenib (VEM) in nonmelanoma solid tumors harboring BRAF V600 mutations (V600m). Journal of Clinical Oncology 32, No 15_suppl (May 2014) 2533--2533. 2014;32 ((suppl.)):2533.Google Scholar
- FDA announces first approval of targeted therapy based on basket study {Internet}. New York (NY): Memorial Sloan Kettering Cancer Center. Available from: https://www.mskcc.org/trending-topics/fda-announces-first-approval-targeted-therapy-based-basket-study.Google Scholar
- Ando, Y. Biostatistical review of new drug applications in Japan - current and future activity. Tokyo: Pharmaceuticals and Medical Devices Agency, 2015. Available from: https://www.pmda.go.jp/ files/000207303.pdf.Google Scholar
- ADDPLAN® {Internet}. Dublin: ICON. Available from: http://www.iconplc.com/innovation/addplan/.Google Scholar
- Treweek, S., Lockhart, P., Pitkethly, M., et al. Methods to improve recruitment to randomized controlled trials: Cochrane systematic review and meta-analysis. BMJ Open. 2013;3:e002360.Google Scholar
- Hoos, A., Anderson, J., Boutin, M., et al. Partnering with patients in the development and lifecycle of medicines: a call for action. Therapeutic Innovation & Regulatory Science.49(6):929--39, 2015Google ScholarCross Ref
- JPRN. Japan primary registries network {Internet}. Available from: http://rctportal.niph.go.jp/.Google Scholar
- PCORI. About our research {Internet}. Washington (DC): Patient-Centered Outcomes Research Institute. Available from: https://www.pcori.org/research-results/about-our-research.Google Scholar
- PCORI. PCORI methodology standards {Internet}. Washington (DC): Patient-Centered Outcomes Research Institute. Available from: https://www.pcori.org/research-results/about-our-research/research-methodology/pcori-methodology-standards.Google Scholar
- CTTI. Project: patient groups & clinical trials {Internet}. Durham (NC): Clinical Trials Transofrmation Initiative. Available from: https://www.ctti-clinicaltrials.org/projects/patient-groups-clinical-trials.Google Scholar
- Burrows, A. The future of clinical trials: industry voices {Internet}. London: Knect365. Available from:https://knect365.com/clinical-trials-innovation/article/0e134709--42bc-4c70-a271-dead8fd43612/industry-voices-future-clinical-trials-673533.Google Scholar
- FDA. Biomarker qualification program {Internet}. Silver Spring (MD): US Food and Drug Administration. Available from: https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ DrugDevelopmentToolsQualificationProgram/BiomarkerQualificationProgram/default.htm.Google Scholar
- The precision medicine initiative {Internet}. Bethesda (MD): Precision Medicine Initiative. Available from: https://syndication.nih.gov/multimedia/pmi/infographics/pmi-infographic.pdf.Google Scholar
- Liu, P. China initiative would pour billions into precision medicine {Internet}. Available from: http://www.bioworld.com/content/china-initiative-would-pour-billions-precision-medicine-0.Google Scholar
- Cyranoski, D. China embraces precision medicine on a massive scale {Internet}. Available from: https://www.nature.com/news/china-embraces-precision-medicine-on-a-massive-scale-1.19108.Google Scholar
- Sherman, R. E., Anderson, S. A., Dal Pan G. J., et al. Real-world evidence: what is it and what can it tell us? New England Journal of Medicine. 375(23):2293--7, 2016Google ScholarCross Ref
- Sun, X. F., Tan, J., Tang, L., et al. Real-world evidence: experience and lessons from China. BMJ. (360):j5262; 2018Google ScholarCross Ref
- PMDA. "Rational Medicine" Initiative. Tokyo: Pharmaceuticals and Medicines Devices Agency, 2017.Available from: https://www.pmda.go.jp/files/000216304.pdf.Google Scholar
- FDA. Use of real-world evidence to support regulatory decision-making for medical devices. Silver Spring (MD): US Food and Drug Administration. Available from: https://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm513027.pdf.Google Scholar
- CDER SBIA Chronicles. Real-world data and evidence in drug development. Silver Spring (MD): US Food and Drug Administration, 2017. Available from: https://www.fda.gov/downloads/drugs/developmentapprovalprocess/smallbusinessassistance/ucm572939.pdf.Google Scholar
- EMA. Public summary of opinion on orphan designation: eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. London: European Medicines Agency, 2015. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2009/10/WC500006161.pdf.Google Scholar
- Zimmermann, M. Soliris case: PNH indication update based on data from the global registry. London: European Medicines Agency, 2016. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Presentation/2016/12/WC500217727.pdf.Google Scholar
- Woodcock, A. and Leather, D. The Salford lung study. London: European Medicines Agency, 2016. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Presentation/2016/12/WC500218600.pdf.Google Scholar
- New J. P., Diar, B. N., Leather, D., et al. Obtaining real-world evidence: the Salford lung study. BMJ Thorax, 69(12):1152--4: 2015Google ScholarCross Ref
- Lewis, J. R. R., Kerridge, I. and Lipworth, W. Use of Real-World Data for the Research, Development, and Evaluation of Oncology Precision Medicines. JCO Precision Oncology. 2017(1):1--11.Google ScholarCross Ref
- Curran, G. M., Bauer, M., Mittman, B., et al. Effectiveness-implementation hybrid designs: combining elements of clinical effectiveness and implementation research to enhance public health impact.Med Care. 2012;50(3):217--26.Google Scholar
- Makady, A., Ham, R. T., de Boer, A., et al. Policies for Use of Real-World Data in Health technology assessment (HTA): A Comparative Study of Six HTA Agencies. The value in Health.20(4):520--32.Google Scholar
Index Terms
- Momentous Innovations in the Prospective Method of Drug Development
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