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This article will analyze the impact of Revlimid on a different outcome: multiple myeloma mortality rates in the US.
Progression-Free Survival (PFS) has become a standard improvement measurement in oncology clinical trials, as PFS usage in clinical trials as a medical product effectiveness outcome has increased from 0% from 1975 to 26% in 2009.3 Nevertheless, some argue that PFS is not a sufficient measurement for delivering improvements in patient quality of life (QOL).3
Celgene’s Lenalidomide (Revlimid) relied heavily on PFS outcomes in its clinical trials to evaluate Revlimid’s effectiveness in improving multiple myeloma outcomes.4 Additionally, rising concerns from biopharmaceutical sponsors regarding payer reimbursement are changing the way sponsors design their clinical trials in order to yield supportive data not only for FDA approval, but also for payer reimbursement approvals.
This article will analyze the impact of Revlimid on a different outcome: multiple myeloma mortality rates in the US. Moreover, this article will communicate the patient’s perspective as it relates to how they perceive improvements in health outcomes from oncology drugs, and discuss payer impact.
Revlimid: Celgene’s Biggest Revenue Driver
Revlimid has been the driving vehicle of Celgene’s revenue growth over the past few years. Revlimid sales have increased from $3.2, $3.7, and $4.2 billion from 2011-2013, respectively, and are expected to continue increasing in following years.1Figure 1 illustrates that Revlimid represented 67% of Celgene’s total revenue portfolio in 2013, with Vidaza and Abraxane following suit. Revlimid, which is approved for multiple myeloma indications, had demonstrated significant improvements in progression free survival (PFS) rates with patients.2
Interestingly, the multiple myeloma mortality rates decreased after the release of Revlimid and its competitive product, Velcade, as shown in Figure 2.
Revlimid Significantly Reduced Multiple Myeloma Mortality Rates
In order to confirm the whether Revlimid and Velcade impacted trends in Figure 2, we conducted a financial multiple regression to evaluate the effect of medical product sales on multiple myeloma mortality rates from 2006 – 2010. Figure 3 demonstrates that an increase in Revlimid sales was statistically associated with a reduction in multiple myeloma mortality rates; we did not find any statistically significant relationship between Velcade sales and multiple myeloma mortality rates. An adjusted R2 figure of 0.99 suggests that most of the impact on the multiple myeloma mortality rate reduction is associated with an increase in Revlimid sales, leaving 1% for other influencing factors. Based on this analysis, Celgene would have to sell nearly $40 billion of Revlimid in order to nearly eradicate multiple myeloma mortality, which is ten times more Revlimid sales than in 2013.
It is important to emphasize that this analysis does not factor in patients who do not respond to the drug, but, rather an epidemiological standpoint based on multiple myeloma mortality and Revlimid sales for the time period analyzed.
The Cost Impact
According to Surveillance, Epidemiology, and End Results Program (SEER) data, an annual average of 18,977 patients were diagnosed with multiple myeloma, 10,285 patients died, and 8,692 patients survived from 2006-2010,4 as illustrated in Table 1.
We leveraged averaging methods from Table 1, SEER, and regression coefficients from Figure 3 to generate a cost model that evaluates the financial impact of Revlimid sales on the multiple myeloma death rate (Figure 4). Based on this model, it costs roughly $3.3 million in Revlimid sales to save one patient from multiple myeloma death. This figure does not include associated medical costs (i.e., hospitalization, concomitant medications, etc.).
The Patient’s Perspective
Payers are scrutinizing biopharmaceutical enterprises by ensuring that the value of their medicines are validated, and payers are starting to consult with patients and patient advocacy groups, as well as requiring elaborate quality of life outcomes analyses in clinical trials in order to determine reimbursement criteria once a medical product is FDA approved.
While PFS is becoming a more common measurement for analyzing medical product outcomes, some patients disagree. “PFS is not as valuable as Overall Survival… the chance to extend life by just a little bit is very unappealing… At Stage IV cancer diagnosis, my aspirations are not to squeeze extra time out, but rather to find a cure or a significant and durable response… I am looking for exponential gains rather than incremental,” said T.J. Sharpe, a cancer survivor and clinical trial participant. Patients have also expressed concerns regarding the pricing of therapies, “If a drug buys me six months, and that price is $12,000/month, I then think about the impact on my family; do we want to double up on them (family) by also putting them at a financial strain?” added Sharpe.
Payer reimbursement seemed to improve the perception on drug pricing, “If insurance covers the drug, and I have a 5% co-pay, it is much easier to digest, but, there shouldn’t be economic factors and decisions involved when you’re sick,” said Sharpe. However, the tiered process of payer reimbursement hinders patients from accessing novel drugs that are proven to improve health outcomes. “Insurance companies force people to undergo other treatments first before they get approved to take better treatments… There are barriers to obtain the pharmaceuticals that may save their lives,” added Sharpe.
Revlimid is Impacting Patient Lives, Cost Remains a Matter of Debate
Clinical trial results have demonstrated that Revlimid improved patients’ lives via PFS measures. This analysis supports the notion that PFS not only predicted improvement in clinical trial settings, but the measurement has also impacted multiple myeloma mortality in post-marketing settings.
The pricing of medical products, nonetheless, remains a big issue in the biopharmaceutical industry and increasing pressures from payers will force biopharmaceutical enterprises to change their clinical trial design to better demonstrate the value of their medical products. “Very soon, unless we can give real metrics to payers and make patients, payers and physicians feel that what we’re doing is really worth something, we’re not going to get reimbursed for it,” said Sam Waksal, CEO of Kadmon in a NYBIO address.6
* Rates are per 100,000 and are age-adjusted to the 2000 US Std Population (19 age groups - Census P25-1130). Regression lines are calculated using the Joinpoint Regression Program Version 4.0.3, April 2013, National Cancer Institute.
** Mortality source: US Mortality Files, National Center for Health Statistics, CDC.
*** Cancer sites include invasive cases only unless otherwise noted.