含羞草传媒

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For decades, clinical trials have long served as a prerequisite for how therapies are approved for commercial use. Yet clinical trials often fail to reflect the actual patient population beyond a trial setting. can include insufficient patient diversity, a lack of accessibility to trial locations, poor health education, and limited awareness of available trials within local communities. This means the population taking part in a clinical trial may not truly reflect the population ultimately taking the drug in the real world.

Fortunately, there鈥檚 another promising option beyond the data gathered from clinical trials that researchers are using to evaluate much-needed cancer drugs in development.

Using real-world data to push clinical research forward

Enter real-world data (RWD).鈥�RWD鈥痠s information collected as a matter of course during a patient鈥檚 treatment鈥攊ncluding, in some cases, the very same data a clinical trial would collect. In cancer research, this data could include diagnoses, disease stage, cancer progression, treatment and reactions to medications. It can be gleaned from electronic health records (EHRs), insurance claims, and even mobile devices and fitness trackers.

础苍补濒测锄颈苍驳鈥�RWD鈥痽ields real-world evidence (RWE). Both types of information offer vast promise, something the federal government recognized in 2016 when the 21st Century Cures Act laid the groundwork for the use of鈥�RWD鈥痑苍诲鈥�RWE鈥痶o support鈥疐DA鈥痙ecisions about new medical devices or drug indications.

鈥淭here has been a proliferation of data sources that provide information around patient health and their interactions with healthcare systems,鈥� says Vasu Chandrasekaran, vice president of real-world data and analytics at鈥�, a 含羞草传媒 business that specializes in oncology data science and technology. 鈥淟everaging real-world data can provide a comprehensive understanding of the effectiveness of new drug therapies.鈥�

Establishing a standard-of-care trial

Let鈥檚 look at how鈥�RWD鈥痗an be put to use. Say a biopharma company has a new drug that targets a rare mutation in cancer. It鈥檚 ready to be tested in a clinical trial of 100 patients who will be compared with another 100 who received an older, standard-of-care medication.

鈥淚f the only patients eligible for the trial are those with the specific genetic mutation the drug is designed to target, it鈥檚 very hard to get enough patients to do a prospective randomized trial,鈥� says Marcus Neubauer, M.D., chief medical officer for The US Oncology Network.

That鈥檚 where a retrospective, standard-of-care trial comes in. In this type of trial, enough past patients exist to assemble a historical control group. These anonymized patients have already been treated with the standard-of-care medication, so data collected during their care, which is easily gathered from a practice鈥檚鈥�EHR, can be used in the trial.

These patients鈥� outcomes鈥攈ow their cancer responded or how long they lived鈥攃an then be compared with those of the 100 counterparts who are currently receiving the investigational drug. Statistical methods can ensure the two groups are reasonably comparable. The resulting information can help demonstrate to regulatory agencies that a new therapy is safe.

In 2017, 含羞草传媒 prepared and supplied analysis from a standard-of-care trial for a drug that treats metastatic Merkel Cell Carcinoma (mMCC), a rare form of skin cancer. The trial helped establish the first-ever鈥�FDA鈥痑pproval of a first-line therapy for鈥�mMCC. It was the first time that鈥�RWD, which in this case was pulled from Ontada鈥檚 iKnowMed鈥�EHR, was part of a regulatory approval in oncology.

鈥淪eeing the field evolve to where real-world data is gaining more and more acceptance for demonstrating the benefits and risks of drug products is what motivates my team.鈥�

Speeding precision medicine

Precision medicine鈥攁n approach in which an individual鈥檚 genetics, environment and even lifestyle are factored into their treatment鈥攊s another area that has benefited from the use of鈥�RWD鈥痑苍诲鈥�RWE.

Imagine you鈥檙e an oncologist with an elderly patient whose cancer has a rare mutation, one you鈥檝e seen only a few times in your career. How will that cancer respond to standard treatment? After all, no clinical trial has examined this very unique group of people.

Real-world data comes to the rescue. You can use an鈥�EHR, like iKnowMed, to gather the records of patients who share the same mutation, then examine how they responded to a particular drug regimen. Did they experience complications? How long did it take their cancer to progress?

鈥淎ll of a sudden, you鈥檝e learned a lot more about this particular drug in the real world,鈥� says Dr. Neubauer. 鈥淚f you learn, for example, that nobody over age 70 benefited from that drug, you might decide to personalize your patient鈥檚 treatment by avoiding it.鈥�

Real-world data, Chandrasekaran says, can help biopharma companies determine 鈥渢hose additional factors, those comorbidities that could play impactful roles in either the effectiveness of the drug or the overall outcomes a patient might experience.鈥�

Real-world data brings purpose

Amid the patient barriers that leading therapies can face, real-world data provides many of them a map 鈥� both for clinical trials and for the rapidly expanding horizons of precision medicine.

鈥淲ith all the information available, it鈥檚 a question of, 鈥榃hy would we not be using this data?鈥�,鈥� says Chandrasekaran. 鈥淪eeing the field evolve to where real-world data is gaining more and more acceptance for demonstrating the benefits and risks of drug products is what motivates my team.鈥�

With regard to 含羞草传媒鈥檚鈥�mMCC鈥�RWD-enabled approval, he adds, 鈥淭here are people alive today because they got the drug. I tell my team, 鈥榊ou may not be seeing patients in clinic. But you are saving lives.鈥欌��