Accessing New Cancer Diagnostics: Personalized Treatment & Toxicity Tests

•Radiation toxicity test : If you have been diagnosed with prostate cancer and are considering radiation treatment, this test predicts whether you will have an 8- to 10-fold risk of severe toxicity, allowing you to choose safer radiation treatment options or other treatments. (MiraDx offers the P ROSTOX test .)

•Immune checkpoint inhibitor toxicity test : If you are considering getting an immune checkpoint inhibitor (a type of immunotherapy that enhances the immune system's ability to fight cancer by releasing "brakes" on immune cells, allowing them to recognize and attack cancer cells more effectively), this test predicts whether you will have a 10-fold risk of autoimmune-related adverse reactions or side effects, allowing you to choose safer treatment options. (MiraDx offers the PREVIOTOX test .) How do these novel toxicity tests work?

•Test process: You get a cheek swab, which is analyzed for genetic markers that are compared to known risk patterns to identify whether you are high or low risk for a toxic reaction.

•Scientific foundation : Your microRNA and noncoding DNA are analyzed for systemic stress response to detect potential triggers of adverse reactions. What are the challenges that patients face in accessing new tests?

•Doctor buy-in: Doctors are often required to order tests, and your doctor may not have heard of a new one or know how to interpret test results from it. They are often only interested in tests that will provide information to help with the decision immediately in front of them, while you may have bigger and longer-term objectives.

•Insurance reimbursement : The test may not have been incorporated into the standard care guidelines. Health insurance companies are slow to reimburse patients for new tests. What can you do to access new tests?

•Consider alternative access routes : Contact the vendor and look into research studies, early-access programs, nonprofit registry studies.

•Advocate for yourself : Ask your doctor about new diagnostic options that could provide personalized treatment insights; be persistent.

•Understand incentives : Recognize that doctors must see a benefit in ordering the test and that insurance ultimately determines reimbursement.

•Spread awareness : Share patient testimonials on the value of tests for predicting outcomes and encourage patient advocacy groups to highlight the potential cost savings and improved patient outcomes from these predictive tests.

•Donate: Support nonprofits that help patients access new tests. What are the challenges that diagnostic companies face in getting a new test to market?

•Funding: Venture capital firms are primarily focused on making money, which can conflict with the goal of helping patients. They want a business that has the potential to become huge, which is more possible in therapeutics — diagnostic services are typically priced much lower. Pharmaceutical companies offer the deepest pockets for funding new ventures, but testing startups must contort themselves to meet the research and development needs of pharmaceutical companies, which may be different than patients' care needs.

•Insurance reimbursement : Getting insurance to cover a new test is extremely difficult. Insurance companies are often reluctant to pay for preventive or risk-assessment tests.

•Multiple customers : A successful test must align the interests of three key stakeholders: patients, doctors (who must see a benefit in ordering the test), and insurance companies (which pay for the test). The incentives for many players in the medical industry are sometimes optimized for their own profits, not what is best for patients.

•Market adoption: Convincing doctors to use a new test and pharmaceutical companies to recognize its value can be challenging.

•Regulatory hurdles : Navigating FDA and Clinical Laboratory Improvement Amendments (CLIA) certification requirements is complex and time consuming.

•Commercialization costs : Developing and marketing a new diagnostic test requires significant financial investment. What are some clever ways to provide patients with better access to new tests?

•Set slow payoff expectations : Find investors, such as friends and family, who are patient and interested in a mission-focused approach rather than profit alone; apply for government grants, such as Small Business Innovation Research (SBIR) grants; avoid venture capital.

•Create a foundation : A nonprofit can enable access for patients who can't afford the full price of the test.

•Create a study: Offer the test through a clinical trial to give patients access; gather valuable real-world data; and provide clinical controls, e.g., an institutional review board, or IRB. (Clinical trials often pay patients or for referrals.)

•Create value propositions for all customers : Design tests that simultaneously benefit patients (through better outcomes), doctors (through better outcomes and less work), and insurers (through savings); win-win-win.

•Form research partnerships : Work with academic medical centers to offer and develop the test through collaborative studies, and leverage their credibility.

•Be transparent: Educate patients and doctors about test benefits and build trust through open dialogue.

•Engage patient advocacy groups : Speak to cancer support groups and work with them to help organize research that gathers patient insights.

•Promote awareness : Help spread information about the test's ability to predict outcomes and its potential to help patients choose the safest treatment option. How can you learn more?

•See other conversations we have had on new diagnostic tests and bringing them to market, such as:

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•To learn more about toxicity tests, visit the website MiraDx to learn about their research and upcoming presentations at conferences like ASCO, and join their patient registry (through MiraKind) to potentially access tests and participate in their research.

•Contact Joanne Weidhaas at jweidhaas@mednet.ucla.edu. The information and opinions expressed on this website or platform, or during discussions and presentations (both verbal and written) are not intended as health care recommendations or medical advice by Cancer Patient Lab, its principals, presenters, participants, or representatives for any medical treatment, product, or course of action. You should always consult a doctor about your specific situation before pursuing any health care program, treatment, product or other course of action that might affect your health. Meeting Notes KEYWORDS Cancer diagnostics, MiraDx, microRNAs, radiation therapy, immune checkpoint inhibitors, toxicity prediction, patient response, UCLA, clinical trials, nonprofit, patient access, biomarkers, DNA analysis, prostate cancer, autoimmune diseases. SPEAKERS Joanne Weidhaas (71%), Rick Davis (7%), Brad Power (5%), Richard Anders (5%), Cindy Ness (3%), Roger Royse (3%), Russ Hollyer (3%), Alane Watkins (2%) CHAT CONTRIBUTORS Russ Hollyer, Rick Davis, Michael Nagle, Chris Apfel, Helen, Alane Watkins SUMMARY Joanne Weidhaas, a radiation oncologist and co-founder of MiraDx, discussed her journey in developing molecular diagnostics for cancer. She highlighted the company's focus on microRNAs, which constitute 80% of DNA and play crucial roles in cancer biomarkers. MiraDx's first test predicts ovarian cancer risk, and their prostate cancer test identifies risk of radiation toxicity, helping patients choose safer treatment paths. Despite challenges in securing venture capital and insurance reimbursement, MiraDx has successfully validated its prostate cancer test and is expanding into immunotherapy toxicity prediction. The nonprofit MiraKind suppor supports patient education and access. OUTLINE Overview and Founding of MiraDx

•The discussion focused on Joanne Weidhaas's diagnostic company and its novel market approach.

•She is a radiation oncologist who founded MiraDx and the nonprofit MiraKind.

•She collaborates with Frank Slack, a PhD developmental biologist who discovered the first human microRNA, leading to their work on cancer and microRNAs.

•MicroRNAs control messenger RNAs and play a role in cancer biomarkers.

•There were challenges bringing their findings to market, including the complexities of the medical system and the difficulty of securing venture capital funding. Challenges and Strategic Decisions

•Dr. Weidhaas faced initial challenges in starting MiraDx, including the decision to avoid venture capital funding and focus on patient education and access.

•It was important to align with the medical ecosystem incentives, including those related to insurance and the benefits of using the test that doctors must see. .

•She went to Stanford Business School to learn about the complexities of the medical system and the importance of aligning tests with patient and doctor needs. Development and Expansion of MiraDx

•The lab was moved from New Haven to UCLA to focus on translational research, including studies on prostate cancer and radiation therapy.

•Their first product was successful. It identifies patients at risk of different toxicities from radiation treatment.

•They learned the importance of aligning with patient, doctor, and insurer interests.

•COVID-19 impacted their work, including a pivot to COVID testing, and the subsequent validation of their prostate cancer test. Early Access Program and Nonprofit Initiatives

•Their prostate cancer test has an early-access program, which involves top radiation oncology clinics and gathers patient and doctor feedback.

•The nonprofit MiraKind is enabling patient access and education through such measures as a study that allows patients to join a registry and access the test.

•Holding patient education important, MiraDX takes a two-pronged approach of advancing research and enabling patient access. Prostate Cancer and Immunotherapy

•The tests are for toxic reactions to radiation for prostate cancer patients and to immune checkpoint inhibitors.

•Patients at risk of toxicity from radiation treatment should be offered viable alternatives.

•Working with pharmaceutical companies on the need for a better understanding of patient responses to immunotherapy is challenging.

•Getting access to patient samples from pharmaceutical companies is challenging.

•Independ

•Independent validation of tests is important.

•There are no direct competitors in radiation toxicity prediction, but the field is complex.

•Working with insurance is challenging, and a collaborative approach is needed to ensure patient access to meaningful information. Final Thoughts

•Patient and doctor involvement is important in moving the field forward.