Beyond Standard Care: Additional Options that could Enhance your Treatment Today
How Doctors Typically Treat Patients
Doctors usually treat patients following what is called "standard of care" (SOC), based on guidelines from national committees. For cancer care, these include the National Comprehensive Cancer Network (NCCN) and the American Society of Clinical Oncology (ASCO). This approach creates uniform care across the country. These guidelines may show how strong the evidence is for each recommendation, with randomized controlled trials (RCTs) considered the best evidence.
Limitations of Standard Care
However, this approach has shortcomings. What if standard treatments don't provide a good quality of life or survival rates? What if your disease has gotten worse despite standard treatments? You might be offered a clinical trial, but what if none are suitable for you, or they are too difficult to join? In rare cases, your doctor might know about a promising new drug and put in significant effort to request compassionate use from the drug company. But are there other options?
Our Philosophy on Treatment Options
We believe there are! Here's our approach:
Medical treatment should be evidence-based, but we think those words have been misinterpreted to only mean data in national guidelines. True evidence-based medicine (EBM) includes three parts:
- Best Research Evidence/National guidelines
- Clinical Expertise/Experience
- Patient Values and Preferences
The second part is growing rapidly thanks to AI, electronic medical records, and new blood and tissue tests that better capture cancer biology. Much information is now available from retrospective studies linking biomarkers, treatments and outcomes, and from case reports, early clinical trials, and patient-reported outcomes. We refer to such data as promising treatments.
Financial Orphan Treatments
These promising treatments fall into three main categories:
- Existing generic FDA-approved drugs
- Supplements
- Lifestyle changes
These haven't yet passed the gold standard of showing effectiveness in randomised studies, so they aren't in national guidelines. For example, a drug approved for a non-cancer disease might show promising evidence of prolonging survival in cancer patients. If there is supporting data from animal studies, it would suggest causation and not just correlation.
The irony is that pharmaceutical companies won't fund definitive studies to validate such ideas because there's little or no money to be made on these treatments. We call them "financial orphans."
How We Help
At the Morningside Center, we provide information about these immediately actionable financial orphan treatments. We fund clinical studies which will take years to mature, but meanwhile, we encourage you to discuss these promising ideas with your healthcare providers to see if they might help you now – carefully weighing the evidence and assessing risks and benefits in your specific case.
Don't be surprised if your oncologist hasn't heard of these treatments. They aren't supported by RCTs, generally considered the gold standard for medical practice. However, your doctor can legally prescribe these treatments (if prescriptions are needed) as "off-label use" if other options have been carefully considered and there's some supporting medical data.
Taking Action on Your Own is not a Good Idea
Some interventions don't require a doctor's prescription. You could try these yourself, but we urge you to check with your doctor first, since there could be unexpected interactions with your other treatments or you might have medical conditions that make them problematic.
Understanding Off-Label Use
The law allows healthcare providers to offer off-label treatments if there's some supporting medical data. However, doctors may not do this for various reasons: they don't know about these options, they worry about liability, they aren't paid for the extra time needed to explain these treatments and monitor side effects, they feel pressure to follow national guidelines strictly, or they're uncomfortable asking patients to pay if insurance doesn't cover the cost of the off-label use. We'll discuss ways to address these concerns in a future blog. In the meantime, to enhance your chances of receiving these interventions, you may wish to offer to sign an informed consent form and to pay for these treatments (since insurance typically only covers what's in national treatment guidelines). The good news is that many of these treatments are affordable, have minimal side effects, and are readily available.
Help Advance Medical Knowledge
Finally, we have an important request: please donate your medical data to our web-based registry/observational study. We'll analyse this data regularly and make it publicly available. Our goal is to find patterns - for example, which cancer types benefit most from which treatments, alone or in combination. This information could help you and others, now and in the future.
Joining the registry takes just a few minutes and doesn't require any effort from your healthcare providers. Please spread the word in your cancer community. Together, you'll speed up discoveries that could save thousands of lives every year.