TAPUR STUDY OVERVIEW
1. Why is ASCO conducting the TAPUR Study?
2. What are the TAPUR study objectives?
3. What is the endpoint of the TAPUR study?
4. What is a targeted therapy and how does it work?
5. Is the TAPUR study randomized or non-randomized? Is it comparing TAPUR drugs to other therapies?
PATIENT PARTICIPATION
6. Who will be eligible to particpate in the TAPUR study?
7. What if a patient does not have one of the drug-cancer-gene matches specified in the study?
8. How likely is it that a genomic target will be found in a patient's cancer?
9. What type of treatment will the patient receive?
10. Will patients have to pay for treatment?
11. What should patients who are thinking about participating in TAPUR consider?
12. How will the genomic testing be performed?
13. Doctors can already prescribe a commercially available drug off-label. Why would they or their patients want to take part in the TAPUR study?
14. How can a patient find a participating clinician?
15. Will a patient be allowed to continue a drug after the study ends? How would the cost of the drug be covered at that point?
STUDY CONDUCT
16. What safeguards will be put into place to ensure the TAPUR study follows best clinical trial practices?
17. If a genomic variation is found in a patients tumor, is the TAPUR study likely to offer a drug that targets the abnormality?
18. How will patients get access to targeted therapies?
19. Will ASCO make study results available to participating investigators, patients, drug companies, and the public?
20. How will ASCO determine whether a treatment is effective or ineffective?
ADDITIONAL QUESTIONS
21. The TAPUR study sounds similar to CancerLinQ? Isn't the TAPUR registry a duplication of efforts?
22. Will this study divert patients from other promising clinical trials?
23. I am a patient advocate and would like to find out more information and/or get involved with the TAPUR Study. How can I connect with the study team?
24. How do I obtain a genomic profiling test and how much does it cost?
25. What if I have additional questions about TAPUR?
TAPUR STUDY OVERVIEW
1. Why is ASCO conducting the TAPUR study?
In launching the Targeted Agent and Profiling Utilization Registry (TAPUR) study, ASCO aims to collect “real-world” data on clinical outcomes to help learn additional uses of molecularly-targeted cancer drugs outside of indications approved by the Food and Drug Administration (FDA). The prospective, non-randomized clinical trial will collect information on the anti-tumor activity and toxicity of commercially available, targeted cancer drugs in a range of cancer types, including any advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma with a genomic variation known to be a drug target or to predict sensitivity to a drug.
In oncology, it’s becoming more common to conduct tumor genomic profiling—particularly for patients who have advanced cancer or patients who have exhausted all standard treatment options—to determine whether or not the results might suggest a previously unrecognized therapy option. Although not always the case, it may be reasonable to expect that a targeted agent that is effective in patients selected according to a biomarker (i.e., genomic alteration) in one type of cancer may be effective for patients with another type of cancer that harbors the same genomic alteration. Thus, genomic profiling might reveal therapy options for patients such as treatment with an investigational agent in a clinical trial or use of a targeted therapy drug that has been approved by FDA for a different cancer.
However, it’s often difficult for patients to get access to and reimbursement for anti-cancer agents used off-label. And, even if an oncologist can get access to a drug and treat the patient, the larger medical community has no way to learn whether that particular drug worked in that patient with those particular characteristics. Current prescribing practices don’t capture the outcomes of patients who receive therapies off-label.
ASCO hopes to address both of these problems with the TAPUR study.
2. What are the TAPUR study objectives?
- To describe the anti-tumor activity and toxicity of commercially available, targeted anti-cancer drugs used for treatment of patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma with a genomic variant known to be a drug target or to predict sensitivity to a drug.
- To record the treatment-related adverse events experienced by patients.
- To create a prospective database of patient outcomes following treatment of patients with advanced solid tumors, multiple myeloma or B cell non-Hodgkin lymphoma that harbors a genomic variant known to be a target of an approved targeted anti-cancer drugs or to predict sensitivity to a drug with a commercially available targeted agent provided by the study.
- To catalogue the commercially available tumor genomic tests used by clinical oncologists in the usual care of patients with advanced cancer.
- To determine the concordance of the treatment plan proposed by the treating oncologist with that recommended by the Molecular Tumor Board in applicable situations.
3. What is the endpoint of the TAPUR study?
Objective tumor response or stable disease at 16 weeks after treatment initiation are the primary study endpoints. The study will collect information on progression-free survival, overall survival, duration of treatment on study and treatment-related high grade and serious adverse events.
4. What is a targeted therapy and how does it work?
A targeted therapy delivers a treatment that is aimed at a specific molecular feature of the cancer cell. Often this is a genomic element that enables the cancerous cell to grow uncontrollably or prevents the body from identifying and eliminating the cancerous cell. The targeted therapy inhibits the genomic element to attack cancer growth at the molecular level.
5. Is the TAPUR study randomized or non-randomized? Is it comparing TAPUR drugs to other therapies?
The TAPUR study is a non-randomized clinical trial. All enrolled participants will receive a targeted therapy as treatment. While there are many drugs being offered in TAPUR, each drug is being used individually.
PATIENT PARTICPATION
6. Who will be eligible to participate in the TAPUR study?
TAPUR is designed to include a broader patient population than is typically enrolled in clinical trials. The study will include patients who have any advanced solid tumor, multiple myeloma, or B cell non-Hodgkin lymphoma and are no longer responding to standard anti-cancer treatment or for whom no acceptable standard treatment is available. If a patient has tumor testing results that reveal a genomic variation, a clinician will review drugs available on TAPUR that potentially target that variation. The clinician can choose from one of the drug-cancer-gene matches specified in the study or request a review by the study’s Molecular Tumor Board. Patients will be screened to determine if they are healthy enough to participate based on broad inclusion/exclusion criteria.
7. What if a patient does not have one of the drug-cancer-gene matches specified in the study?
The TAPUR Molecular Tumor Board (a group of experts convened by ASCO) will provide review and discussion of cases that do not fit the criteria specified in the protocol, as well as cases where the treating physician requests guidance. The ultimate treatment will be the decision of the patient and the treating physician. Some possible outcomes other than enrolling on the TAPUR study include:
- Treatment on another clinical trial. The Molecular Tumor Board will be able to provide information on alternate ongoing clinical trials for consideration.
- Treatment with off-label therapy outside of TAPUR protocol. The treating physician may recommend administering an off-label commercially available therapy that is not included in TAPUR.
8. How likely is it that a genomic target will be found in a patient’s cancer?
The short answer is – we are not sure. Scientists and clinicians are still working to understand which of the genomic variations play a critical role in cancer development. TAPUR will help clinicians and scientists understand which of the genomic variations can be successfully targeted with FDA-approved cancer drugs – so-called “actionable” genomic variants. Recently reported genomic profiling studies performed in patients with advanced cancer suggest that actionable genomic variants are found in 20-40% of patients’ tumors.
9. What type of treatment will the patient receive?
Treatment with a commercially available, targeted anti-cancer drug matched to a genomic variant identified in a tumor biopsy specimen. ASCO has invited a number of pharmaceutical companies to provide marketed, targeted drugs and additional resources to support the development of the TAPUR study’s infrastructure. The following companies have agreed to participate in the TAPUR study:
- AstraZeneca
- Bayer
- Boehringer-Ingelheim
- Bristol-Myers Squibb
- Eli Lilly and Company
- Genentech
- Merck
- Pfizer
10. Will patients have to pay for treatment?
Research participants in TAPUR will receive the anti-cancer drugs at no charge. Administration of the drugs is according to the FDA-approved dosing and determined by the treating physician. As a result, ASCO expects that routine clinical care costs will be covered by the patient’s insurance plan. Patients who receive treatment with a drug available in the protocol will be followed for standard toxicity and efficacy outcomes including tumor response, progression-free and overall survival as well as duration of treatment and serious side effects.
11. What should patients who are thinking about participating in TAPUR consider?
As with any treatment decision including participation in a clinical trial, patients should discuss all options with their physician. Patient-oriented information about clinical trials participation is also available on ASCO’s Cancer.Net website. Patients eligible for TAPUR must have a cancer with a genomic variation that is targeted by a TAPUR study drug. The specific type of genomic test performed on a tumor specimen is at the discretion of the physician and patient.
12. How will the genomic testing be performed?
The patient and his/her physician will decide if performing a genomic test on a tumor specimen is medically appropriate. The choice of test is up to the physician and patient, including whether to use a test performed at any point during the patient’s care. Because TAPUR does not require new testing, the genomic testing is not provided as part of the TAPUR study. To be eligible to participate in TAPUR, a patient must have results from a genomic test performed on a tumor specimen or on cell-free DNA in plasma (“liquid biopsy”) or an immunohistochemistry (IHC) test for protein expression performed on a tumor specimen, that demonstrates that a genomic variant known to be a drug target or to predict sensitivity to a drug is present in the patient’s cancer.
The test must be performed in a lab that has obtained CLIA certification, CAP accreditation, and for labs offering services to residents of New York, accreditation from the state of New York. Contact TAPUR@asco.org for more information.
13. Doctors can already prescribe a commercially available drug off-label. Why would they or their patients want to take part in the TAPUR study?
Commercially available drugs can involve significant costs to the patient because insurance companies often only reimburse for off-label uses that have demonstrated efficacy and are recommended in professional society clinical guidelines or included in standard drug compendia. In the TAPUR study the drugs will be provided at no cost to the trial participants. In addition, TAPUR will provide the opportunity to capture the patient’s experience with the drug so that other patients and oncologists can learn how these targeted drugs are working to treat other cancer types.
14. How can a patient find a participating clinician?
The TAPUR study is open and enrolling at a number of sites within larger existing research networks that run research trials for the National Cancer Institute and industry. ASCO plans to continue to open enrollment at new sites across the country throughout the study. Information on open sites is available on our Participating Centers page. Providers and practices interested in participating in TAPUR should visit the Researchers page or email TAPUR@asco.org.
15. Will a patient be allowed to continue a drug after the study ends? How would the cost of the drug be covered at that point?
It is ASCO’s intent that patients who respond to a therapy would be able to continue to receive that drug at no cost for as long as they continue to benefit from the treatment.
STUDY CONDUCT
16. What safeguards will be put into place to ensure the TAPUR study follows best clinical trial practices?
The TAPUR protocol and consent form have been reviewed and approved by an Institutional Review Board.
In addition, ASCO has established three oversight groups—each of which will include patient representatives, investigators, and genomics specialists:
- Steering Group to oversee study operations, establish data sharing and publication policies, review plans to add or remove drugs from the study and approve participation of additional clinical study sites;
- Molecular Tumor Board to review the proposed drug-target match and report to the clinician on potential treatment options on or off the study;
- Data and Safety Monitoring Board to independently review study results to analyze and monitor for unexpected adverse events, to determine when enrollment of study cohorts should expand or cease, and to determine when to release data.
17. If a genomic variation is found in a patient’s tumor, is the TAPUR study likely to offer a drug that targets that abnormality?
There are currently 19 drugs available through the TAPUR Study, for a total of 16 therapies. These therapies will be able to treat many of the common genomic targets for which drugs have been developed.
18. How will patients get access to targeted therapies?
Once it is determined that the patient meets the defined trial criteria, his or her treating physician will select a drug from among those available in the TAPUR study protocol that targets the identified genomic variation in the patient’s tumor. If a relevant drug-target match is not described in the protocol, the physician may consult the Molecular Tumor Board which will review the clinical and genomic features of the case and identify potential treatment options on and off the study. All patients who receive treatment through TAPUR will be monitored for standard toxicity and efficacy outcomes including tumor response, progression-free and overall survival, as well as duration of treatment.
19. Will ASCO make study results available to participating investigators, patients, drug companies, and the public?
ASCO plans to make results available to all participants involved in the study. In addition, ASCO plans to report study results through existing mechanisms, i.e., scientific meetings and peer-reviewed publications.
20. How will ASCO determine whether a treatment is effective or ineffective?
The TAPUR Data and Safety Monitoring Board will review results and make a determination (according to standards in the protocol) whether a treatment is promising or not for a particular cancer and genomic variant. If a drug is determined to be ineffective for a particular cancer and genomic variant, the drug will no longer be offered in the study for that match, and ASCO will make this information public. If a drug is determined to be promising, ASCO will communicate the results to the drug manufacturer, which may decide to confirm the efficacy in another study. ASCO will also inform study stakeholders and release results from the study through peer-reviewed mechanisms.
ADDITIONAL QUESTIONS
21. The TAPUR study sounds similar to CancerLinQ? Isn’t the TAPUR registry a duplication of efforts?
No. The primary purpose of CancerLinQ is quality improvement, and the initial focus of CancerLinQ will not involve research uses. CancerLinQ is a learning healthcare system that collects data from electronic medical records from participating oncology practices to improve the quality of cancer care. The TAPUR study provides access to a study therapy and collects pre-specified data. Ultimately, CancerLinQ could facilitate the type of data collection required by the TAPUR study, but these projects are currently quite different.
22. Will this study divert patients from other promising clinical trials?
No, the protocol includes a process by which information on other available trials is provided to physicians so that physicians and patients may be informed about all clinical trial options. Patients and clinicians will ultimately make the determination about which treatment approach best fits their situation.
23. I am a patient advocate and would like to find out more information and/or get involved with the TAPUR Study. How can I connect with the study team?
We welcome patient advocates to complete our brief online survey and sign up to receive communications and updates about TAPUR and for those interested, information on how to get involved. The TAPUR team recognizes that patient advocates are an important part of clinical trials and can contribute to the planning and conduct of the TAPUR study, as well as assist in increasing public and patient awareness.
24. How do I obtain a genomic profiling test and how much does it cost?
Your oncologist can help you find out if obtaining a genomic profiling test could be beneficial for your cancer care. Insurance coverage for a genomic profiling test varies, depending on your insurance plan. Your cancer care team and representatives of your insurance plan can help you understand the details of your plan and the coverage for the costs of the test. ASCO’s Cancer.Net website also includes a detailed guide for patients and families on Managing the Cost of Cancer Care. In addition, companies that perform genomic testing may offer assistance programs for paying for the tests. This information can typically be found on the test provider’s website.
Please contact TAPUR@asco.org if you have any additional questions about TAPUR.
--- --- ---
Updated January 11, 2018