“Getting Access to Your Cancer Treatment

“Getting Access to Your Cancer Treatment" (Chris Beardmore) [#73] 1Brad Power and Vanessa Hugo October 18, 2023 “I would generally argue that a lot of therapies that are out there today may have been developed in the old traditional approach where you've run a trial, and you've basically said this therapy is more effective in, let's say, greater than 50% of the population.

And as a result, it may not necessarily be as effective as pursuing something which is more targeted.” – Chris Beardmore “The biggest problem we face right now, as GBM patients, even if we can get xCures, Cancer Commons, Anova, or whatever other organization to recommend these combination therapies for us to try, whether they're still investigational or off label, is just the access.

It's unreal, the barriers we face in trying to get access.” – Vanessa Hugo “We're really good if a tumor board or a group of professionals has come back and said, ‘Hey, if you can find an MDM2 targeted drug, that would really help John Doe. Please get out here and help us do that.’ We can get those on clinical trials or in compassionate use in 60-70% of the cases we request.

” – Chris Beardmore “What we should be probably moving to is something we call ‘n of 1’ – get large volumes of patients moving through their journey, and then look for the signals of where they're finding success and double down on those areas.

” – Chris Beardmore Meeting Summary Advanced cancer patients and their caregivers who are highly engaged in finding ways to treat their disease can get special tests done (like “functional testing” where drugs are tried on live tumor tissue) or get drug combination suggestions (from services like Cancer Commons or CureMatch).

They can decide which treatment recommendations are the best for them and attempt to pursue them, but they are often confronted with the challenge of getting access to treatment if it is “off label” (FDA-approved drugs that have not been approved for that application). Chris Beardmore, CEO and Co-Founder, Anova Enterprises, Inc.

, is uniquely qualified to provide valuable insights and practical tips to help navigate the healthcare system. His background in the regulatory system and clinical trials has allowed him to run down all the paths that can get patients and caregivers the drugs they want. Anova is operationalizing treatment access, especially to treatments outside the standard of care.

Why might patients want to get access to drugs outside the standard of care (off label)? ●The standard of care guidelines are out of date.

“Getting Access to Your Cancer Treatment" (Chris Beardmore) [#73] 2●The standard therapy may have been developed in a traditional clinical trial approach which has shown that the therapy is effective in greater than than 50% of the population, but may not necessarily be as effective as pursuing something which is more targeted. ●Novel tests point to a therapy that is personalized, but it has never been run in a cl

eatment" (Chris Beardmore) [#73] 2●The standard therapy may have been developed in a traditional clinical trial approach which has shown that the therapy is effective in greater than than 50% of the population, but may not necessarily be as effective as pursuing something which is more targeted.

●Novel tests point to a therapy that is personalized, but it has never been run in a clinical trial to bring that therapy into the standard of care. For example, for patients with a brain cancer like glioblastoma (GBM), there are some patients who may not respond well to the FDA-approved first line standard of care chemotherapy temozolomide, based on their “MGMT methylation status ”.

(MGMT is an enzyme which causes resistance to chemotherapy by compromising a DNA repair mechanism.) For those who may not respond to temozolomide, targeted therapy may be worthwhile. ●The prospects with the standard of care treatment (e.g., a 20% success rate) are not good enough for the patient and caregivers.

What are typical barriers patients and caregivers face in accessing personalized treatments outside the standard of care (off label)? ●Regulators won’t approve them due to safety concerns. ●Physicians won’t prescribe them due to safety and liability concerns. ●Insurance companies won’t reimburse them due to cost concerns. ●Pharmaceutical companies won’t provide the drugs due to profit concerns.

How can patients and caregivers access non-standard treatments they think are best for them? ●Pursue patient assistance programs from manufacturers – pharmaceutical companies have protocols for providing access to a therapy, especially if a patient needs financial help.

●Find and enroll in clinical trials ●Take advantage of “compassionate use” or “expanded access” (a process provided by regulators for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available) ●Negotiate with insurance companies How does Anova help patients and caregivers (and physicians) access non-standard treatments that they believe are best?

●Patient Assistance Programs: An example Anova has seen is patients getting access to pembrolizumab (Keytruda) through Merck’s patient assistance program when their insurance company has refused to authorize it. A billing professional can reach out to the patient assistance program and ask for the drug.

●Clinical trials: Anova is running three clinical trials for patients with newly diagnosed glioblastoma (brain cancer): a cell-based therapy, an immunotherapy, and a small molecule that crosses the blood-brain barrier.

They are also helping non-small cell lung cancer patients with a unique biomarker (an EGFR exon 20 insertion mutation) access a targeted treatment made by a Chinese pharmaceutical company ( Dizal Pharma).

aditional clinical trial approach which has shown that the therapy is effective in greater than than 50% of the population, but may not necessarily be as effective as pursuing something which is more targeted. ●Novel tests point to a therapy that is personalized, but it has never been run in a clinical trial to bring that therapy into the standard of care.

For example, for patients with a brain cancer like glioblastoma (GBM), there are some patients who may not respond well to the FDA-approved first line standard of care chemotherapy temozolomide, based on their “MGMT methylation status ”. (MGMT is an enzyme which causes resistance to chemotherapy by compromising a DNA repair mechanism.

) For those who may not respond to temozolomide, targeted therapy may be worthwhile. ●The prospects with the standard of care treatment (e.g., a 20% success rate) are not good enough for the patient and caregivers. What are typical barriers patients and caregivers face in accessing personalized treatments outside the standard of care (off label)?

●Regulators won’t approve them due to safety concerns. ●Physicians won’t prescribe them due to safety and liability concerns. ●Insurance companies won’t reimburse them due to cost concerns. ●Pharmaceutical companies won’t provide the drugs due to profit concerns. How can patients and caregivers access non-standard treatments they think are best for them?

●Pursue patient assistance programs from manufacturers – pharmaceutical companies have protocols for providing access to a therapy, especially if a patient needs financial help.

●Find and enroll in clinical trials ●Take advantage of “compassionate use” or “expanded access” (a process provided by regulators for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available) ●Negotiate with insurance companies How does Anova help patients and caregivers (and physicians) access non-standard treatments that they believe are best?

●Patient Assistance Programs: An example Anova has seen is patients getting access to pembrolizumab (Keytruda) through Merck’s patient assistance program when their insurance company has refused to authorize it. A billing professional can reach out to the patient assistance program and ask for the drug.

●Clinical trials: Anova is running three clinical trials for patients with newly diagnosed glioblastoma (brain cancer): a cell-based therapy, an immunotherapy, and a small molecule that crosses the blood-brain barrier.

They are also helping non-small cell lung cancer patients with a unique biomarker (an EGFR exon 20 insertion mutation) access a targeted treatment made by a Chinese pharmaceutical company ( Dizal Pharma). Anova works with contract research organizations and Dizal to open up study sites within 5-10 working days.

ll cell lung cancer patients with a unique biomarker (an EGFR exon 20 insertion mutation) access a targeted treatment made by a Chinese pharmaceutical company ( Dizal Pharma). Anova works with contract research organizations and Dizal to open up study sites within 5-10 working days. Dizal provides robust funding because they see value; while a trial may

“Getting Access to Your Cancer Treatment" (Chris Beardmore) [#73] 3cost a half million dollars to run, they're losing a million dollars per day that the drugs are not on the market.

●Compassionate use : Anova has operationalized compassionate use opportunities with companies like Kintara Therapeutics , which is developing novel solid tumor cancer therapies; Epitopoietic Research Corporation , a Belgian pharmaceutical company; and NeOnc Technologies , developing intranasal inhalation dosing therapies for the treatment of brain and lung cancers.

A patient newly diagnosed with a Grade 3 astrocytoma (a growth of cells that starts in the brain or spinal cord; there's no cure for grade 3 and grade 4 astrocytomas, as they grow and spread quickly.) underwent standard of care treatment but wanted to also pursue a therapy targeted at a genetic mutation (IDH1).

Anova got her compassionate use access to a targeted treatment made by NeOnc Technologies - an inhalable version of perillyl alcohol. She has been receiving that therapy now for 10 months with no evidence of progression. ●Negotiation with insurance companies : The physician of a cancer patient with glioblastoma recommended bevacizumab as a second line therapy.

Bevacizumab costs about $14,000 per month and doesn’t confer a survival benefit. Anova requested that the insurance instead cover a different targeted drug that matched the patient’s particular tumor mutation. The targeted drug costs $2,000 per month (vs. $14,000), so the insurance company agreed. Anova occasionally works with individual patients through agreements with navigation groups (e.g.

, Private Health) or pro bono, ad hoc. Call to Action: How can patients and caregivers make access to personalized treatments easier in the future? 1.Lobby politicians and regulators to ease access to personalized treatment options. 2.

Create and join “basket trials” (designs in which a targeted therapy is evaluated on multiple diseases that have common molecular alterations), “virtual trials”, or “learning systems” to track real world experience of what is working and not working for each patient.

For example, the Musella Foundation has been running a virtual trial learning system, a registry of brain tumor patients where the treatments they do and the outcomes are tracked. 3.Take advantage of existing programs, like the FDA’s Project Facilitate, set up to expand access to investigational cancer products. Anova’s compassionate use requests can be approved in as little as two hours.

ake advantage of existing programs, like the FDA’s Project Facilitate, set up to expand access to investigational cancer products. Anova’s compassionate use requests can be approved in as little as two hours. Rather than investing millions more into finding new therapeutic targets, patient assistance groups (e.g.

, Anova, xCures, Cancer Commons) should join together in funding more compassionate use programs for the targeted therapies that already exist. 4.Develop a guide to physicians willing to treat patients off label. For example, Glenn Sabin and colleagues are developing a list of “pioneering prescribers” who are open minded about providing access to compassionate use drugs and combination therapies.

5.Don’t participate in a clinical trial from a pharmaceutical company that doesn't have a compassionate use program. As long as they are getting enough patients to fill their trial, they should be able to make extra doses available to those who ask.

“Getting Access to Your Cancer Treatment" (Chris Beardmore) [#73] 46.Grade academic medical centers on how many clinical trials they offer for all patients for all lines of therapy. They should offer a minimum of 40 phase one clinical trials and 200 trial offerings.

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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.

“Getting Access to Your Cancer Treatment" (Chris Beardmore) [#73] 5Meeting Notes Discussion Outline 1.Access to cancer treatments off-label. (0:03) 2.Improving access to clinical trials and managing complex regulatory approvals. (3:23) 3.Access to therapies for aggressive diseases. (7:36) 4.Personalized cancer treatment options. (13:28) 5.Personalized medicine and patient empowerment. (18:21) 6.

Personalized medicine and patient advocacy. (22:21) 7.Personalized medicine and patient advocacy. (28:17) 8.Personalized cancer treatment and collaboration opportunities. (32:53) 9.Improving access to cancer treatments through education and collaboration. (37:43) 10.Improving access to experimental treatments for life-threatening illnesses. (42:52) 11.

Improving access to clinical trials for patients. (48:24) 12.Cancer treatment decision-making and progression.