inVerse - Multiple Sclerosis as a case study
Multiple sclerosis (MS) is a progressive, immune-mediated disorder.
That means the system designed to keep your body healthy mistakenly attacks parts of the body that are vital to everyday functioning. The protective coverings of nerve cells are damaged, which leads to diminished function in the brain and spinal cord.
MS is a disease with unpredictable symptoms that can vary in intensity: while some people experience fatigue and numbness, severe cases of MS can cause paralysis, vision loss, and diminished brain function.
MS is a challenging disorder, but researchers have discovered many treatments that can slow its progression.
The way the disease changes and gets worse is different for each of the three types of MS:
Relapsing-remitting MS: People with this type have attacks when their symptoms get worse, called relapses, followed by full, partial, or no recovery. These flares seem to change over several days to weeks. Recovery from an attack takes weeks, sometimes months, but symptoms don’t get worse during this time. Most people have this type when they’re first diagnosed with MS.
Secondary-progressive MS: People who get this type usually start with relapsing-remitting MS. Over time, symptoms stop coming and going and begin getting steadily worse. The change may happen shortly after MS symptoms appear, or it may take years or decades.
Primary-progressive MS: In this type, symptoms gradually get worse without any obvious relapses or remissions. About 15% of all people with MS have this form, but it’s most common type for people diagnosed after age 40.
If we can understand disease cost burden for MS and determine what importantly disease progression costs, we can derive a clear and unambiguous case for personalised treatment to prevent disease progression.
Drug costs are on the increase
In a study published by JAMA Neurology, researchers found that multiple sclerosis patients who have Medicare Part D—government-funded health insurance that covers prescription drug costs—have seen their medication prices quadruple from 2006 to 2016.
The study looked at an average of 2.8 million Medicare beneficiaries every year from 2006 - 2016. The annual cost of treatment with self-administered disease modifying therapies (DMTs) for MS patients increased from $18,660 in 2006 to $75,847 in 2016, averaging a 12.8% increase annually, based on the list price of the treatments. Medicare's actual spending increased by 10 times over that decade, from $7,794 to $79,411 on average. And average out-of-pocket spending increased by seven times from $372 to $2673 over the 10 years.
Understanding US cost burden:
Worldwide, more than 2.3 million people have a diagnosis of MS. In the United States a recently completed prevalence study, funded by the National MS Society, has estimated that nearly 1 million people over the age of 18 live with a diagnosis of MS.
Understanding disease progression
With an estimated lifetime cost per MS patient of over $4 million, MS is the second-ranked chronic condition (behind congestive heart failure) in direct all-cause medical costs.3 For the U.S., the total estimated cost of MS is $28 billion per year.
The cost of a Phase III clinical trial ranges widely, though a new study pegs the median cost of 138 clinical trials in 2015 and 2016 at $19 million.
“This study identified more than 100-fold differences in the costs of pivotal trials that the FDA required to provide substantial evidence of benefit, with a central cluster of trials with estimated costs of $12.2 million to $33.1 million,” Thomas Moore of the Institute for Safe Medication Practices and others from Johns Hopkins wrote in JAMA Internal Medicine on Monday. “Costs were increased when larger patient enrolments were required to detect a difference from placebo or active drug comparator, and costs were highest when a new drug had to be proved to have clinical benefit that was noninferior to another drug already available.”
Analysis of key cost drivers of pharmaceutical clinical trials in the United States by trial phase, here we highlight spend on CNS clinical trials:
Source: https://www.researchgate.net/figure/Costs-by-therapeutic-area-in-US-million_fig3_293640487, article From Clinical Trials February 2016
Thus we can calculate approximately the cost of trials used to determine new personalised outcomes, if indeed such insight is possible at the patient level from the aggregate data.
inVerse - MS Case study Results
The question to be answered for personalised treatment discovery was formulated as follows: Is it possible to analyze clinical trials conducted for multiple sclerosis (MS) and find potential signals which could help to identify candidate personalised therapies?
inVerse - MS Case Study - the case for change
Using inVerse learning from aggregates capabilities we show here that by enabling granular insights and learnings from aggregate data such as successful and failed clinical trials, we can derive massive value for Payers and Healthcare systems.
