By Ed Miseta, Chief Editor, Clinical LeaderFollow Me On Twitter @EdClinical
Recent years have seen unprecedented innovation in the clinical space. Precision medicine, cell and gene therapies, decentralized trials, real-world data, and the promise of artificial intelligence (AI) and machine learning (ML) are just a few of the reasons to be excited about the future of clinical research. But what can we expect to see in the next three years, and what are the challenges sponsor companies will need to overcome?
A webinar hosted by IBM Watson hoped to answer those questions. The discussion featured Lorraine Marchand, general manager of life sciences at IBM Watson Health; Nimita Limaye, research VP, Life Sciences R&D Strategy and Technology at IDC Health Insights; and Greg Cunningham, director of the RWE Center of Excellence at Eli Lilly and Company. The three shared insights into what we might expect to impact trials over the next three years.
In this article the panel discusses precision medicine and real-world data. In part 2 of this article the panel looks at the future of decentralized clinical trials.
The Growth Of Precision Medicine
The first game changer the panel discussed is the advancement of precision medicine. It has moved from exploring single gene mutations to performing research using combinations of genes. This change has the potential to bring better drug targets forward and get the best products to patients faster.
This has been playing out in the last decade in oncology real-world evidence, notes Cunningham. We've seen an evolution in precision medicine as we've built out the patient record. As we have done that, the marketplace has evolved rapidly, particularly for electronic medical record data and genomic data.
Pharma companies were happy to get their hands on electronic medical record data. When genetic test results were combined with that data, researchers gained the ability to look at a single mutation and develop better patient outcomes.
Where precision medicine will continue to evolve in 2022 and beyond is the growing use of genetic testing in oncology. This will provide the industry with more data about patients. With more genes at their disposal, researchers can look at groups of genes and the complex combinations of gene mutations. This has the potential to open the door for tools like artificial intelligence to help researchers analyze the complex number of permutations.
RWD Creates More Efficient Research
Next the panel discussed RWD and the ability to utilize it across several use cases from discovery and development to commercial. Limaye likes the prospect of being able to create a data exchange where researchers can bring together claims, clinical, EMR, and genomics data directly from patients to create an intelligent and digital patient health record. That record gives researchers the digital equivalent of a real-life patient which can be used as a natural history or synthetic control arm in randomized control clinical trials.
These data can allow drug developers to track patient response to drugs and look at outcomes after being exposed to new therapies. The promise of data and technology is using tools like AI to advance therapies and get them to patients faster. This will be done with better information and a much more efficient way to perform drug development and track and monitor outcomes in patients.
Big data has been a topic of discussion in pharma for years. The volume of clinical data is now growing exponentially. Approximately 30% of the world's datavolume is being generated by the healthcare industry and by 2025, the compound annual growth rate will hit 36%. That's 6% faster than manufacturing, 10% faster than financial services, and 11% faster than media & entertainment.
In addition to getting bigger, data is also getting broader. Researchers can not only look at a patients medical history but can now consider factors such as social determinants of health and behavioral data.
Since most EHRs do not include genomic data, researchers need the ability to look at patient data more holistically. Type 2 diabetes was one example discussed. Today, 40% to 70% of it is genetically inherited and there are over 500 different genetic loci which could be involved in causing the disease. The earlier strategy of looking at genetic risk scoring was single trait. That is now transitioning to multi-trait research with an integrated view that will drive a precision medicine strategy. This creates an interesting situation where drug discovery will continue to get more specific and focused towards an individual while also getting bigger and broader.
The Challenge Of RWD
With access to RWD, drug developers can benefit from data they may not have known existed. Although the data is rich and robust, it can be difficult to access. One of the biggest challenges the industry faces is data stored in silos. The panel notes data is stored in patient claims, electronic medical records, in lab apps, images, and genetic files on a smartphone. Having the technology to tap into those sources to identify quality data is the primary challenge.
The data must be de-identified for patient privacy, cleaned, curated to remove noise, and enriched, which means bringing together the various components that will be meaningful to drug development. That will allow researchers to have a patient record that is useful across pharma, from development through to commercial. An exchange would enable that exact process a platform where various entities can bring their data to have it linked, integrated, cleaned, and enriched, creating a data package that can be plugged into studies.
An important component of that exchange is the data being housed in a place where various third parties can feel comfortable bringing their data to match it with data from other third parties.
Cunningham cites lupus as an example of where pharma could benefit from such an exchange. I would like to have a complete data set of lupus, he says. Lupus is an autoimmune condition, and a quintessential data set could be used for a number of uses, such as preparing a Phase 1 trial, selecting patients, or understanding patient responses to different therapies when designing studies. Specific data sets could be created for each therapeutic area, and pharma companies need that hard work of bringing the data together removed.
Data Assembly And Analysis
Currently, drug developers spend 80% of their time assembling data and 20% of their time analyzing it. The situation must be flipped so that 80% of the time is spent performing analysis. The panel recommends rethinking how health records are created. The healthcare and life science industries require the ability to easily put data together. That comes back to investing in data standards everyone can agree upon. With the right standards and technology, the industry can spend its time improving lives as opposed to assembling data.
The FDA has indicated it is aware and supportive of the fact that pharma needs use RWD in drug discovery. The industry now needs to create the interoperability, standards, and methods to ensure that data can be included in regulatory submissions. This evolution may be akin to the critical path initiative. When the FDA embraced the idea of the critical path and allowing more in silico modeling of clinical trial design and development, it took the industry almost 10 years to adopt and apply the guidance.
The FDA has said it recognizes the importance of RWD, but that acknowledgement has resulted in few approvals. Looking at the use of synthetic control arms and RWD in regulatory submissions over the last five years shows just 10 submissions and all were in oncology. Only one was a successful submission, and the rest were rejected because of lack of completeness of the data.
Those numbers should tell the industry the FDA is not going to dictate how to get to approvals. The industry is going to have to figure out the interoperability and how to apply the standards. Regulators are always going to require quality data. Industry will need to enrich the data and create the cohort that is going to be equivalent to a patient in the real world.
In part 2 of this article, the panel discusses the role of technology in clinical trials, how decentralized trials will continue to evolve, what capabilities sponsor companies will need, and whether decentralized trials might offer cost benefits to companies.
Original post:
The Next Three Years Of Clinical Trials DCTs RWE And Beyond - Clinical Leader
- BENITEC BIOPHARMA INC. Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - Marketscreener.com - February 13th, 2023 [February 13th, 2023]
- CENTOGENE to Participate in Upcoming Conferences in February in the Lead Up to Rare Disease Day - Marketscreener.com - February 7th, 2023 [February 7th, 2023]
- Gene | Definition, Structure, Expression, & Facts | Britannica - January 27th, 2023 [January 27th, 2023]
- New gene therapy delivers treatment directly to brain - January 27th, 2023 [January 27th, 2023]
- Indian Pharma Congress: Gene-cell therapy, preventive medicine future of health care, says expert - Economic Times - January 25th, 2023 [January 25th, 2023]
- A blood test that identifies people at higher risk of miscarriage? Thats the goal of this award-winning Rutgers med student. - The Philadelphia... - January 19th, 2023 [January 19th, 2023]
- Gene Therapy: Genes As Medicine | Pfizer - January 6th, 2023 [January 6th, 2023]
- How Genomics will ensure a risk-free and beneficial treatment for good health and well-being - The Financial Express - December 28th, 2022 [December 28th, 2022]
- Regenerative Medicine Advanced Therapy Designation | FDA - December 18th, 2022 [December 18th, 2022]
- 3576 - Gene ResultCXCL8 C-X-C motif chemokine ligand 8 [ (human)] - November 23rd, 2022 [November 23rd, 2022]
- Study identifies new gene that drives colon cancer - EurekAlert - October 17th, 2022 [October 17th, 2022]
- Updated Stroke Gene Panels: Rapid evolution of knowledge on monogenic causes of stroke | European Journal of Human Genetics - Nature.com - October 17th, 2022 [October 17th, 2022]
- The challenges of translating CRISPR to the clinic - Labiotech.eu - October 17th, 2022 [October 17th, 2022]
- Editas Medicine Presents Preclinical Data on EDIT-103 for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa at the European Society of Gene... - October 17th, 2022 [October 17th, 2022]
- 'We have to find a way': FDA seeks solutions to aid bespoke gene therapy - BioPharma Dive - October 17th, 2022 [October 17th, 2022]
- Mathematical model could bring us closer to effective stem cell therapies - Michigan Medicine - October 17th, 2022 [October 17th, 2022]
- Approval, Commercialization Highlighted at Cell & Gene Meeting on the Mesa - Genetic Engineering & Biotechnology News - October 17th, 2022 [October 17th, 2022]
- CANbridge-UMass Chan Medical School Gene Therapy Research in Oral Presentation at the European Society of Gene and Cell Therapy (ESGCT) 29th Annual... - October 17th, 2022 [October 17th, 2022]
- Depression Treatment: How Genetic Testing Can Help Find the Right Medication - Dunya News - October 17th, 2022 [October 17th, 2022]
- The Risk-Reward Proposition for CGT Clinical Trials - Applied Clinical Trials Online - October 17th, 2022 [October 17th, 2022]
- Precision Medicine Could Get Even More Precise With Allarity Therapeutics Next-Generation Diagnostics - Benzinga - October 17th, 2022 [October 17th, 2022]
- Decibel Therapeutics Receives FDA Clearance of IND Application for DB-OTO, a Gene Therapy Product Candidate Designed to Provide Hearing to Individuals... - October 17th, 2022 [October 17th, 2022]
- Time for your medicine: unlocking the power of our body clocks - The Guardian - October 17th, 2022 [October 17th, 2022]
- Replay establishes distinguished Scientific Advisory Board of genomic medicine and cell therapy experts - Yahoo Finance - October 17th, 2022 [October 17th, 2022]
- Scientists Reappraise the Role of Zombie Cells That Anti-aging Medicine Has Sought to Eliminate - Neuroscience News - October 17th, 2022 [October 17th, 2022]
- Forge Biologics Announces Updated Positive Clinical Data in RESKUE, a Novel Phase 1/2 Gene Therapy Trial for Patients with Krabbe Disease - Business... - October 17th, 2022 [October 17th, 2022]
- Gene Expression Signatures Are Analyzed for Biomarkers of Response in HCC - Targeted Oncology - October 17th, 2022 [October 17th, 2022]
- NHS England World-first national genetic testing service to deliver rapid life-saving checks for babies and kids - NHS England - October 17th, 2022 [October 17th, 2022]
- The proteinprotein relationship that could mend a broken heart - RegMedNet - October 17th, 2022 [October 17th, 2022]
- Study finds microprotein correlated to Alzheimers risk - Daily Trojan Online - October 11th, 2022 [October 11th, 2022]
- Passage Bio Announces Appointment of William Chou, M.D. as Chief Executive Officer - Yahoo Finance - October 11th, 2022 [October 11th, 2022]
- Gene Therapy Rapidly Improves Night Vision in Adults with Congenital Blindness - Newswise - October 11th, 2022 [October 11th, 2022]
- Scientists Discover Protein Partners that Could Heal Heart Muscle | Newsroom - UNC Health and UNC School of Medicine - October 11th, 2022 [October 11th, 2022]
- The Pros and Cons of Lentiviral and Adeno-Associated Viral Vectors - The Medicine Maker - October 11th, 2022 [October 11th, 2022]
- Insights & Outcomes: Foreign DNA, quantum potholes and relapsing fever - Yale News - October 11th, 2022 [October 11th, 2022]
- Expediting IND applications with drug master files - BioPharma Dive - October 11th, 2022 [October 11th, 2022]
- UNC School of Medicine Awarded $3 Million to Lead Study to Reduce PTSD Frequency, Severity | Newsroom - UNC Health and UNC School of Medicine - October 11th, 2022 [October 11th, 2022]
- Lineage to Present at Alliance for Regenerative Medicine 2022 Cell & Gene Meeting on the Mesa - businesswire.com - October 8th, 2022 [October 8th, 2022]
- The Next Crispr Gene Editing IPO Could Be Near - Henry Herald - October 8th, 2022 [October 8th, 2022]
- 10-year CRISPR anniversary: How gene editing revolutionized medicine, and what lies ahead - Genetic Literacy Project - October 8th, 2022 [October 8th, 2022]
- Blood from a baby at birth can be gene sequenced to prevent diseases - USA TODAY - October 8th, 2022 [October 8th, 2022]
- What doctors wish patients knew about breast-cancer prevention - American Medical Association - October 8th, 2022 [October 8th, 2022]
- Growth in Cell and Gene Therapy Market - Pharmaceutical Technology Magazine - October 8th, 2022 [October 8th, 2022]
- Gene Editing Service Market 2022 : Top Players to Reflect Impressive Growth Rate till 2029: Caribou Biosciences, CRISPR Therapeutics, Merck KGaA,... - October 8th, 2022 [October 8th, 2022]
- Tip Sheet: $78 million to support new precision oncology institute, update on experimental gene therapy for herpes and the launch of Fred Hutch's new... - October 8th, 2022 [October 8th, 2022]
- Cell and Gene Therapy: Rewriting the Future of Medicine - Technology Networks - October 2nd, 2022 [October 2nd, 2022]
- Growth in Cell and Gene Therapy Market - BioPharm International - October 2nd, 2022 [October 2nd, 2022]
- CRISPR Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX130 for the Treatment of Cutaneous T-Cell... - October 2nd, 2022 [October 2nd, 2022]
- BioMarin Resubmits Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Severe Hemophilia A to the FDA - PR... - October 2nd, 2022 [October 2nd, 2022]
- Fighting Breast and Ovarian Cancer With a Lupus Antibody - Yale School of Medicine - October 2nd, 2022 [October 2nd, 2022]
- This gene therapy company is testing new tech to 'switch off' diabetes and obesity with a pill - Euronews - October 2nd, 2022 [October 2nd, 2022]
- Tenaya Therapeutics to Participate in Inaugural Hypertrophic Cardiomyopathy Medical Societys 2022 Scientific Sessions - Yahoo Finance - October 2nd, 2022 [October 2nd, 2022]
- Risk of Alzheimer's dementia may be predicted with help of new tool Washington University School of Medicine in St. Louis - Washington University... - October 2nd, 2022 [October 2nd, 2022]
- Tiny Sea Creature's Genes Shed Light on Evolution of Immunity - UPMC - October 2nd, 2022 [October 2nd, 2022]
- Who will get the call from Stockholm? It's time for STAT's 2022 Nobel Prize predictions - STAT - October 2nd, 2022 [October 2nd, 2022]
- Excision BioTherapeutics Awarded California Institute for Regenerative Medicine (CIRM) Grant to Support Ongoing Phase 1/2 Trial Evaluating EBT-101 as... - October 2nd, 2022 [October 2nd, 2022]
- NeuroVoices: Emma Ciafaloni, MD, on the Vast Expansion of Innovative Approaches to Duchenne Muscular Dystrophy - Neurology Live - October 2nd, 2022 [October 2nd, 2022]
- COVID mRNA Jabs and Testing Kicked Off This Industry of Drug Development: Here's What You Need to Know - The Epoch Times - October 2nd, 2022 [October 2nd, 2022]
- Kidney resident macrophages have distinct subpopulations and occupy distinct microenvironments - University of Alabama at Birmingham - October 2nd, 2022 [October 2nd, 2022]
- Nobel Prize for medicine: the full list of winners - The National - October 2nd, 2022 [October 2nd, 2022]
- The surprising link between circadian disruption and cancer may have to do with temperature - EurekAlert - October 2nd, 2022 [October 2nd, 2022]
- The global live cell imaging market is expected to grow at a CAGR of 8.44% during 2022-2027 - Yahoo Finance - October 2nd, 2022 [October 2nd, 2022]
- Chroma Medicine Announces Formation of Scientific Advisory Board of Global Experts in Gene Editing and Cell and Gene Therapy - PR Newswire - September 20th, 2022 [September 20th, 2022]
- Ring Therapeutics Announces Issuance of U.S. Patent for its Anellovector Compositions - Yahoo Finance - September 20th, 2022 [September 20th, 2022]
- Cholesterol gene mutation: Why would a healthy 27-year-old have severe heart problems? - 69News WFMZ-TV - September 20th, 2022 [September 20th, 2022]
- Gene Therapy for Severe Hemophilia B Could Be More Cost Effective Than Current Treatments - Managed Healthcare Executive - September 20th, 2022 [September 20th, 2022]
- AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis... - September 20th, 2022 [September 20th, 2022]
- The Biggest CGT Breakthroughs Through the Eyes of Our 2022 Power List - The Medicine Maker - September 20th, 2022 [September 20th, 2022]
- Leading Virus Researcher to Chair UVA's Department of Microbiology, Immunology and Cancer Biology - UVA Health Newsroom - September 20th, 2022 [September 20th, 2022]
- Work remains on Tay-Sachs and other Ashkenazi genetic disorders J. - The Jewish News of Northern California - September 20th, 2022 [September 20th, 2022]
- Study Shows Genetic Link to Moving to the Beat of Music - Newswise - September 20th, 2022 [September 20th, 2022]
- Viewpoint: In the post Roe v Wade world, what changes should a biology textbook writer make to address the medical repercussions of Dobbs? - Genetic... - September 20th, 2022 [September 20th, 2022]
- Alnylam Receives Approval in Europe for AMVUTTRA (vutrisiran) for the Treatment of Hereditary Transthyretin-mediated (hATTR) Amyloidosis in Adult... - September 20th, 2022 [September 20th, 2022]
- CSL flexes gene therapy muscle with latest drug - Sydney Morning Herald - September 20th, 2022 [September 20th, 2022]
- The MIT Press releases new book on the science of the heart from cardiac expert Dr. Sian Harding - EurekAlert - September 20th, 2022 [September 20th, 2022]
- Global Pharmaceutical Contract Manufacturing Market is projected to reach a market value of US$329.7 Billion in 2032: Visiongain Reports Ltd - Yahoo... - September 20th, 2022 [September 20th, 2022]
- Alzheon Reports Industry-Leading Biomarker, Brain Preservation and Clinical Effects Following 12 Months of Treatment in Phase 2 Trial of Oral ALZ-801... - September 20th, 2022 [September 20th, 2022]
- Do You Have Lung Cancer With An EGFR Mutation? If So, The Drug Tagrisso Might Be Right For You Based On New Results From A 'Practice Changing' Trial -... - September 20th, 2022 [September 20th, 2022]
- Getting rid of unwanted transformed cells: Possible new directions in cancer therapy - EurekAlert - September 20th, 2022 [September 20th, 2022]
- Sven Kili on reconvening with the ISCT - The Medicine Maker - September 14th, 2022 [September 14th, 2022]