Innovation in the area of oncology has taken off at a historic pace. This is represented by the increase in available treatment options and revenue generated from therapies currently marketed across cancer conditions. This momentum has gained speed in recent years as innovative cancer treatment candidates have progressed through development pipelines of both established and emerging biopharmaceutical companies. Now private industry and the FDA are relying more on big data, specifically multi-source, point-of-care, real-world data (RWD) to power clinical and economic real-world evidence (RWE) outcomes. We are experiencing more powerful, streamlined ways to bring innovations and advances to patients who need them at the point of care.
The Cancer R&D Wave
The last decade has seen oncology drugs grow to dominate as the largest sector of the research and development (R&D) focus of the pharmaceutical industry. In 2018, nearly $150 billion was spent on anti-cancer medicines, with a record 15 new oncology therapies launched for 17 indications.1 In 2019, the proportion of pharmaceutical R&D dedicated to cancer topped one-third of the pipeline, up from a quarter in 2010.2 Based on this trajectory, the global oncology market is predicted to be valued between $200 and $230 billion by 2023. The compounded annual growth rate is estimated to grow from 11% to 14% between 2019 and 2023.1
The profound momentum in oncology places that much more pressure on pharmaceutical sponsors to speed products through drug development and also provide sufficient evidence that meets the safety and efficacy standards needed for regulatory rigor and health technology assessment review. It also underscores an unprecedented need for comparative effectiveness evidence between competing therapies that relies on data from real-world practice patterns and utilization.
The Era of Breakthrough Cancer Therapies
Patients today have unprecedented access to the most innovative, life-extending cancer therapies. But many still lack critical access mainly due to the industry having limited data on product effectiveness or lack of FDA regulatory approval for new indications or label expansions. However, one reason for record breaking expansion to access is that the FDA is granting cancer drugs a breakthrough designation that expedites their development if they demonstrate a substantial benefit over the available standard of care. Between 2012 and 2017, 43% of approved oncology therapies received this designation.3
The R&D Challenges of Oncology
While there is a recent surge in pipeline activities, oncology still remains one of the most difficult and complex R&D sectors in pharmaceuticals. Randomized clinical trials face a significant risk of failure—partly due to a sharp increase in complexity—and greater time needed for drug development. The drop in composite success rate for phase transitions (from 11.7% in 2017 to 8.0% in 2018) and longer clinical trial durations compared to other disease areas (3.2 years to 1.8 years, respectively) can attest to this.1
Another obstacle is identifying appropriate patient volunteers to participate in investigational studies and candidates for such targeted therapies, such as biomarker matching. Retaining those patients over the course of the study is just as important as recruitment goals and should be monitored closely.
FDA’s Evolving Review Process
Despite these challenges, therapies in oncology have the potential to impact the lives of so many patients and their families with limited options, especially those in underserved communities who might face challenges in access to care and payment for treatment. The FDA has modernized and streamlined the drug approval structures and processes in an attempt to address the need for more expedited drug evaluation reviews and patient feedback inclusion. This includes having special designations for review and approval, such as Fast Tracking, Breakthrough Therapy, Orphan Drug, Accelerated Approval, and Priority Review. The FDA has also unified post-market pharmacovigilance surveillance systems and made enhancements that incorporate more patient engagement and feedback into the drug development process.
Where Real-World Data Comes In
In 2016, the FDA signed the 21st Century Cures Act into law, and this has a great impact on the availability of oncolytic agents in the marketplace. The objective of the law is to help accelerate medical product development and bring innovations and advances to patients who need them faster. A significant part of this initiative is to enhance the ability to modernize clinical trial design and speed up the development and review process of novel medical products through the use of data that exists beyond clinical trials (that is, RWD), which can then be curated and structured into RWE that can aid in clinical decisions.
The Larger Role of Real-World Data and Evidence
Today, competition in the pharmaceutical space is not just based on the strength of R&D pipelines and treatment portfolios. Its strength also depends on the outcomes and demonstrated results that provide benefits in a patient-centered and appropriate manner. This is the measurement of effectiveness that determines how well a therapy works, and not just if it works or efficacy. The opportunity to make an impact is also more constrained as older treatments mature past patent protection and newer products come onto market at greater efficiency. This puts greater urgency on the ability to demonstrate the evidence of drug efficacy and make cost-effective choices.
The challenge with RWD is that data are often deeply fragmented and have varied data points across the healthcare spectrum. They show that simply having access to big data does not necessarily translate into value as it is utilized. Instead, translating these data into meaningful formats for research is how it fulfills an essential need. Overcoming the largest hurdles to creating real-world evidence demands navigating and structuring complex, unstructured, and in many cases non-digital source data.
As the driving force behind RWE, RWD serve a crucial role in informing real value-based health decisions. The richness in clinical data contained in medical records provides a more accurate, detailed reflection of a patient’s health status and outcomes within a given time period. The messy, herculean challenge of structuring these data into useful data sets is where Ciox Real World Data comes in.
A Powerful Platform With Research-Grade Information
Ciox Real World Data provides real-world clinical source data that are fit for medical research. The Ciox DataFit Platform™ transforms the medical research experience by design. Through expert data model designers, full custody of the data supply chain, and artificial intelligence/cloud native technologies, the platform provides a continuously customizable digital sandbox that’s filled with the richest, most relevant cohorts by assembling all available structured, unstructured, and nonstandard patient data.
The Ciox DataFit Platform™ aggregates, structures, and normalizes patient information into clean, analyzable data that allow our data designers and curators to continually review and ensure that source data translate into research-grade information.
Real-World Data’s Impact in Oncology
The importance that RWE carries for oncology is tremendous. It enables cancer drug developers to take the necessary steps that increase their chances of success by accessing a broad range of data on patient treatment patterns, drug combinations and health outcomes. This level of access to curated, reliable real-world data sources also enables intermediate end points that better inform decisions faster in the development lifecycle.
And most importantly, by streamlining oncology drug development with fewer financial and procedural hurdles while at the same time bolstering access to clinical data with greater insight, we give patients and their families who are fighting cancer more options. It comes down to giving them a greater chance to more fully live their lives.
1. IQVIA Inc. Global Oncology Trends 2019: Therapeutics, Clinical Development and Health System Implications Institute Report. Published May 30, 2019. Accessed November 16, 2020. https://www.iqvia.com/insights/the-iqvia-institute/reports/global-oncology-trends-2019 2. Lloyd I. Pharma R&D Annual Review 2019. Published February 2019. Accessed November 16, 2020. https://pharmaintelligence.informa.com/resources/product-content/pharma-rnd-annual-review-2019 3. Shepshelovich D, Amir E. Expedited approval of cancer drugs without randomized controlled trials: too good to be true? Oncotarget. 2018;9(57):30942-30943. doi:10.18632/oncotarget.25799