Accessing Cancer Treatments: Insurance, Clinical Trials & Compassionate Use
Featuring: Chris Beardmore, CEO and Co-Founder, Anova Enterprises, Inc., Vanessa Hugo
In short
When standard cancer treatments aren't working well enough, patients can pursue off-label drugs, clinical trials, compassionate use, and insurance negotiations — but real barriers from regulators, physicians, insurers, and drug companies make that hard. Chris Beardmore, CEO of Anova Enterprises, walks through each access pathway with concrete examples, including how Anova has helped patients get approved for compassionate use in as little as two hours and negotiated insurance coverage for targeted therapies that cost less than the standard alternative.
- •If your insurer denies a drug, ask a billing professional to contact the manufacturer's patient assistance program directly — Anova has seen patients get pembrolizumab (Keytruda) this way after insurance refusals.
- •Compassionate use (also called expanded access) is a real FDA pathway for life-threatening conditions — if a tumor board has identified a specific targeted drug for you, organizations like Anova report success getting access in 60–70% of cases.
- •When negotiating with insurance, a cheaper targeted drug matched to your tumor's mutation can sometimes replace a more expensive standard drug — bring cost comparisons and biomarker data to the conversation.
- •Before joining a pharmaceutical company's clinical trial, ask whether they have a compassionate use program — if they have enough trial participants, they may be able to provide access to extra doses for others who need it.
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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.
” – 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.
” – 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.
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). , 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. 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). Anova works with contract research organizations and Dizal to open up study sites within 5-10 working days. Dizal provi provides robust funding because they see value; while a trial may 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. 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. 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. 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. 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. (53:14) SUMMARY KEYWORDS patients, GBM, work, drug, therapy, groups, compassionate, physicians, trial, biopharmaceutical companies, access, mutation, clinical trials, fda, disease, chris, approved, ucla, treatments, patient assistance programs SPEAKERS Chris Beardmore (77%), Brad Power (10%), Vanessa Hugo (4%), Brian McCloskey (3%), Glenn Sabin (3%), Rick Stanton (2%) MEETING
Full transcript
Brad Power We're honored to have Chris Beardmore with us. Chris is the Co-founder and CEO of Anova Enterprises, which, among other things, helps patients get access to treatments. He works in clinical trials. He'll describe his background in greater detail, but it is a very important part of the process for us as advanced cancer patients.
I was introduced to Chris Beardmore by Chris Schuler, who has been very helpful with introductions for us and is working in brain cancer and fundraising. I was very taken by Anova’s services because early on in our journey, we worked closely with CureMatch, which recommends drug combinations that match patients’ biomarker profiles. Drug combinations offer, perhaps, a more durable response to patients.
But most of the clinical trials that come up that get drugs approved are monotherapies where they are treated by themselves. When you're using the scientific method, you want to control everything else and just test for that one thing. The result is that most of the randomized clinical trials have approved the drug as a single therapy at maximum tolerated dose.
Drug combinations actually are a good option, but there's limited randomized clinical trial evidence for many combinations. Increasingly, they're running 6trials with immunotherapies plus something else, but many aren't. ” There's no evidence that a clinician can use to prescribe them. So they will hesitate to prescribe that even though the patient may have decided that's really what they would like to use.
The issue of access once you've decided what your best treatment option is, is a recurring one for our patients. I saw that Chris had some ideas on how to cut that Gordian knot and help people get access. His company does a lot of other things. He'll tell us about that. ” Chris Beardmore 3:23 I'm a regulatory person by training.
I started my career running IRBs (institutional review boards) Animal Care and Use (looking out for humane treatment of animals), radiation, and safety committees at the University of Maryland, at UCLA, and for the Department of Defense. A lot of that work early on was about access.
At the University of Maryland, not only were we dealing with an AIDS epidemic, and what are you doing with combination therapies to try and address HIV, and what was creating individuals who were so immune-suppressed, but also emergency room research, tirilazad mesyl ate (a drug for the acute treatment of brain and spinal injuries and strokes), and a lot of work in the “golden hour” (the period of time immediately after a traumatic injury during which there is the highest likelihood that prompt medical and surgical treatment will prevent death) came out of that institution.
At UCLA, a lot of the work was around new drugs like targeted therapies like Herceptin, Patrick Soon-Shiong working on islet cell transplants, artificial hearts, things like that. And compassionate use became something that we did quite a 7bit of.
For the Department of Defense we were working on biowarfare protective products – what you do when you have national emergencies and may need something like an anthrax vaccine or an emergency use authorization of a COVID vaccine. When I left academic medical centers, I started building cancer centers and was focused on how to improve access to clinical trials both with an idea that participation in studies probably improves outcomes.
Some of that is probably related to improvements in technology and the precision medicine transformation and how products are probably hitting targets more effectively theoretically and resulting in better outcomes. But it also probably comes from the rigor that's applied to clinical research and compassionate use, that if you have to report adverse events, you have to deal and manage those adverse events.
8 9 I think everybody understands that, ultimately, precision medicine has created a world where we are looking at therapies that have increasingly refined FDA approvals. I'd like to point to this one – the approval of Keytruda. Right now, the package insert, which is the document that's inserted into each vial of Keytruda, has all of the approved indications for the therapy.
What you used to see would be an approval of a drug for a wide target population. You might see something like approval of a drug like sunitinib (Sutent) for, say, second line colorectal cancer. What you're seeing in these targeted therapies are package inserts that cover a large number of diseases.
Here, you've got Keytruda already approved for melanoma, non-small cell lung cancer, head and neck, squamous cell cancers, liver cancers, and cervical cancers. Each of those individual treatment patient populations have very specific lines of therapy and other requirements.
In non- small cell lung cancer, one of I think, three approvals is in combination with Pemetrexed, a platinum-based chemotherapy, as first line treatment, so long as you don't have an EGFR or an ALK mutation. So what a patient needs to do in order to become informed, or a physician to ultimately keep all of this straight, is becoming more complex.
I would argue that at some point, we're going to be past the ability of the human brain to really manage this. Chris Beardmore 7:59 If you look at the different ways that people can gain access to therapies, I largely think of them in these four categories. 10 Certainly there are FDA-authorized or approved products that are out there.
Patients should be discussing treatment options with their physicians all the time to figure out what's approved or what's about to be approved. However, having said that, 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 than 50% of the population.
As a result, it may not necessarily be as effective as pursuing something which is more targeted. For example, I've been talking quite a bit with people in the glioblastoma space.
If you look at the Stupp protocol, which is the regimen they use for all patients with newly diagnosed high grade brain tumors, gliomas, they will resect the tumor if they can, and they will administer radiation and temozolomide (a chemotherapy) based upon a study that was issued nearly 20 years ago. In the end, more patients will do better with that regimen than not.
What is clear now that we're in this era of precision medicine is that a lot of things impact whether or not a patient is going to respond to temozolomide. Methylation status of the MGMT promoter is a big condition there, or whether or not the tumor can be impacted through radio sensitization, that sort of thing.
You're beginning to see other mutations leading folks to believe that maybe it would be worthwhile to pursue targeted therapy in a world where effective first line therapy is pretty limited. 11 Just because the therapy is out there doesn't necessarily mean an insurance company or a physician is going to be eager to go find it and prescribe it.
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