Summary
In this episode of Real World Talk, host Kevin Keogh talks to Dr. Rafael Fonseca, a hematologist who specializes in multiple myeloma, and Interim Director of the Mayo Clinic’s Cancer Center. They discuss the gulf between the idealized world of clinical trials and the reality experienced by patients with an incurable cancer like multiple myeloma — and how real world data can help bridge the gap.
Highlights
- [00:22] Introduction — Kevin Keogh introduces Rafael Fonseca, MD, a hematologist who specializes in multiple myeloma, and Interim Director of the Mayo Clinic’s Cancer Center.
- [01:04] Multiple myeloma — Dr. Fonseca explains the unique characteristics of multiple myeloma, a cancer that starts in plasma cells and can progress until it eventually erodes bone marrow.
- [03:06] New treatments — Recently there has been a lot of interest in multiple myeloma, leading to many treatment options that have made it possible to control: but unfortunately, it’s not considered curable.
- [07:24] Running ahead, falling behind — There are so many clinical trials studying multiple myeloma and moving the field forward that by the time they’re finished, they’re semi-obsolete — which is why real world data is useful.
- [08:43] Back to reality — The ideal world of textbooks and clinical trials is a fantasy. In real life, there can be more than one or two variations in how a drug is used, or the sequence drugs are used in, and that’s important data to have.
- [11:00] Supporting information — For example, if you want to send a patient into a clinical trial but you aren’t sure how a drug they’ve previously taken will interact with the treatment on trial — because no Phase III clinical trial has been done on the subject — real world data is really useful.
- [13:23] Give the best drug first — In the case of multiple myeloma, real world data has shown that many patients don’t make it to subsequent clinical trials. So doctors should give out the most effective drugs first.
- [19:22] Beyond the price tag — Evaluating the true dollar cost of treating a disease is about more than drug price. You also have to consider other factors, such as rates of hospitalization.
- [20:24] Giving medicine the real-life treatment — Dr. Fonseca says that when you work out living expenses, you don’t just look at how much your mortgage costs: you look at how long you’ll be paying it for, and other bills. That holistic approach should apply in medicine too.
- [29:56] Real world value — When considering value-based care models, real world data can help ensure that the results you see in a lab are actually scalable.
- [32:53] Money gets results — Dr. Fonseca believes that interfering with the cost of expensive drugs will hurt patients later, because it stifles the incentive to fund research which leads to innovations.
- [39:37] Thinking ahead — Judging the value of a drug requires taking into account quantifiable and unquantifiable measures, including toxicity and its potential to keep a patient alive until an even better treatment comes along.
Key Points
- Real world data proved multiple myeloma patients need the best treatment upfront. Ideally, researchers have patients come to follow-up clinical trials so they can test whether giving a certain drug after other drugs affects outcomes. But real world data showed that many multiple myeloma patients don’t make it to the next trial, for various reasons. Doctors who want to understand potential interactions between drugs therefore turn to real world data. This insight also encourages doctors to give their most promising treatment as soon as possible.
- Calculating the cost of treatment is about more than the price of drugs. Calculating the dollar cost of disease progression versus treatment that controls disease requires a holistic approach. Treatment means spending more on drugs: but someone whose disease is not being controlled may have more hospitalizations, which are a major expense. And that’s before you consider the impact on a patient’s emotional welfare.
- Interfering in the price of expensive drugs dampens innovation. Dr. Fonseca believes that the major reason multiple myeloma has received so much attention recently is because there’s money to be made from novel treatments. He argues that lowering drug prices or funding them through the government removes the incentive for investors, which means less research and less innovation.
