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ROS1 Real-World Data (RWD) Project

The ROS1ders are working to collect real-world data to improve outcomes for patients who have ROS1+ cancer.

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The Need for Real-World Data (RWD) in ROS1+ Cancer

ROS1+ cancer is relatively rare, occurring in only 1%-2% or non-small cell lung cancer cases--this means fewer than 2000 cases are diagnosed in the USA each year. About half of oncologists do not even test for the ROS1 biomarker in patients who have lung cancer.  While ROS1+ cancer has been found in several cancers other than lung, ROS1 biomarker testing is not yet recommended in guidelines for those cancers.


Many oncologists will never meet a patient who has ROS1+ lung cancer, and few clinics will see enough ROS1+ patients to be able to gather data on a sizeable cohort. It's unlikely a Phase 3 trial comparing ROS1 treatments would accrue enough participants to be statistically valid.  How then do we gather information for evidence-based treatment of ROS1+ cancer? 


Enter Real-World Data (RWD) RWD is “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.”  Collecting diagnostic and treatment data and outcome information from real patients in the clinic may be able to help us identify best practices for the care and treatment of patients who have ROS1+ cancer no matter where they are treated.


The chart below shows data sources that can be used to generate real-world data.






Types of RWD.png

RWD collected informally, such as polls in The ROS1ders private Facebook group, can be useful in generating research hypotheses.  However, to influence regulatory policy or support evidence-based medical choices, more rigorous and formal data collection is required.  Real-World Evidence (RWE) is “clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD.” All real-world evidence comes from RWD, but not all RWD is of sufficient quality to be used in RWE. RWE might be used to support regulatory decision making, and so must be held to higher data integrity standards.

Image credit:  "Types of real-world data" by ASCPT  is licensed under CC-BY-NC-ND

ROS1 Real-World Data (RWD) Project

Ethically collecting and sharing patient data to maximize benefit to
ROS1+ cancer patients, clinicians, and researchers

Ideally, The ROS1ders would like our Real-World Data Project to achieve the following goals:

  • Involve patients in all aspects of the project, from the outset

  • Gather data on patient characteristics, environmental exposures, diagnosis, treatment, and outcomes while avoiding participant “survey burnout”

  • Link  surveys, electronic health records, specimens, and genomic test results across sources and data silos

  • Ensure researchers and collection platforms are able to release data rapidly in compatible formats

  • Share data and results with all participants, clinicians, and researchers interested in ROS1+ cancer

  • Obtain ethics approval from an Independent Review Board

  • Allow The ROS1ders to access identified data to facilitate patient navigation among members of The ROS1ders

  • Maintain patient trust throughout the process

We continue to learn about the challenges these goals present and are taking steps towards achieving them.  One step is collaborating on LUNGevity Foundation's Project PEER (a patient registry) to learn more about the types of RWD we can realistically collect, and how they might improve ROS1+ cancer outcomes. Another step is networking with Count Me In and RARE Alliance to learn more about RWD best practices. We are also learning about ways to use Patient-Reported Outcomes (PROs) to collect RWD; one of our officers coauthored ESMO's upcoming Clinical Practices Guidelines on "The role of patient-reported outcome measures in the continuum of cancer clinical care."


The ROS1ders occasionally conduct SurveyMonkey polls among patients and caregivers to gather information that might help improve outcomes for patients. For instance,  we may run a poll to determine what percentage of The ROS1ders would qualify under a trial's proposed eligibility criteria, or determine how many members are treated with which TKIs.  We sometimes share the (de-identified) results of these polls on our website or with researchers.

On May 3, 2022, the ROS1ders hosted a virtual Research Roundtable on the topic real-world data, or RWD (view the recording here). This webinar brought together international ROS1 expert clinician and researchers with members of The ROS1ders to discuss which research questions we want to answer, and how we might go about collecting and analyzing RWD to answer those questions. We published the findings in a white paper titled “Real-World Data for ROS1+ Cancer
and Other Rare Cancers.
” Translations of the white paper in several languages will be posted here soon.

Real-World Data for ROS1+ Cancer and Other Rare Cancers © 2022 by The ROS1ders is
licensed under CC BY-SA 4.0

Need for RWD
ROS1 RWD Project
ROS1ders-Icons-Maroon-Outreach (1).png

How Can I Participate?

Patients, caregivers, researchers, clinicians dealing with ROS1+ cancer, as well as supporters, can all play a role in our Real-World Data Project.

Clinicians and Researchers Focused on ROS1+ Cancer

Contact us and let of know of your interest.

Patients and Caregivers Living with ROS1+ Cancer

Connect with our community and begin sharing your experiences in our private Facebook group.  We'll make sure you're aware of upcoming opportunities to share your real-world data.

Supporters of ROS1+ Cancer Research

Please donate to The ROS1ders to support our Research Program.


RWD Terminology


Real-world data (RWD) is “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources” such as:

  • electronic health records

  • medical claims

  • disease registries

  • patient-generated data (e.g., home medication dosing records)

  • data from devices (e.g., wearable fitness trackers, mobile phones)

  • informal surveys and anecdotes

Real-world evidence (RWE) is “clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD.” All real-world evidence comes from RWD, but not all RWD is of sufficient quality to be used in RWE.  RWE might be used to support regulatory decision making, and so must be held to higher research standards.

Types of RWD that might contribute to RWE are:

  • Randomized clinical trials

  • Large simple trials

  • Observational studies (prospective and/or retrospective)

  • Pragmatic trials

  • Validated Patient Reported Outcomes (PRO) instruments

More info is available in the Framework for FDA's Real-World Evidence Program.​

A randomized clinical trial (RCT) is a trial in which the participants are divided by chance into separate groups that compare different treatments or other interventions. Using chance to divide people into groups means that the groups will be similar and that the effects of the treatments they receive can be compared more fairly. At the time of the trial, it is not known which treatment is best.

A large simple trial (LST) is a randomized controlled clinical trial that typically answers only one or two questions in a broader patient population. It is generally more efficient and less expensive than other large randomized controlled trials.

An observational study is type of study in which individuals are observed or certain outcomes are measured. No attempt is made to affect the outcome and is not considered a clinical trial.

  • A retrospective observational study identifies the population and determines the exposure/treatment from historical data (i.e., data generated prior to the initiation of the study). The variables and outcomes of interest are determined at the time the study is designed.  An example might be a study of electronic health records of people diagnosed with ROS1+ cancer since 2011.

  • A prospective observational study identifies the population of interest at the start of the study, and then collects treatment and outcome data from that point forward.  For example, an academic cancer center may choose in January 2020 to track data of all their patients who start taking entrectinib after that date. However, the study does not influence which treatment a patient receives.

A pragmatic study measures the benefits a treatment produces in routine clinical practice.  It might enable enrolling a more diverse population than in a clinical trial, examining how products work when given as part of clinical practice, and providing evidence for a regulatory labeling claim.

A Patient registry is an organized system that uses observational study methods to collect uniform data and evaluate specified outcomes for a population defined by a particular disease, condition or exposure.  “Registries” can refer to both programs that collect and store data and the records that are so created.  For instance, Australia has a ROS1 Registry to collect data on all patients in their country who have ROS1+ positive cancer. Here's a user's guide to patient registries prepared by the US Agency for Healthcare Research and Quality.

 A Patient Reported Outcome (PRO) is information about a patient’s health that comes directly from the patient. Examples of patient-reported outcomes include a patient’s description of their symptoms, their satisfaction with care, and how a disease or treatment affects their physical, mental, emotional, spiritual, and social well-being. In clinical trials, patient-reported outcomes may provide information about the side effects of the new treatment being studied. The use of patient-reported outcomes may help plan the best treatment and improve quality of care. A PRO instrument is a tool for gathering information that's been validated by research.  One example is the Patient Health Questionnaire-9 (PHQ-9), a form which captures patient responses in order to assess depression over the past 2 weeks.

IRB (also known as Institutional Review Board)

An IRB is a group of scientists, doctors, clergy, and patient advocates that review and approve the detailed plan for a clinical trial. IRBs are meant to protect the people who take part in a clinical trial. They check to see that the trial is well designed, legal, ethical, does not involve unneeded risks, and includes a safety plan for patients. There is an IRB at every health care facility that does clinical research, and every trial is reviewed by an IRB before the trial begins. 

This process is called "Ethics Review" in some countries. Every country has their own regulations that guide how ethics reviews are conducted. A study that will have sites in multiple countries must pass ethics review in each country where data will be collected from participants. There is no single ethics review board that can approve a study in multiple countries.

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