Debra Madden is a 2-time cancer survivor who was diagnosed with Hodgkin's lymphoma as a young adult and breast cancer nearly 20 years later, which was thought to be secondary to the radiation she had received for her original cancer treatment. Debra became an active Cancer Research Advocate following her second cancer diagnosis at the age of 42 years. She is currently a member of the ECOG/ACRIN Cancer Research Group and the Patient-Centered Outcomes Research Institute's Advisory Panel on the Assessment of Prevention, Diagnosis, and Treatment Options. She also serves on multiple grant review panels, including the Congressionally Directed Medical Research Program Breast Cancer Research Program. Debra blogs at "Musings of a Cancer Research Advocate", (https://draemadden.wordpress.com/) and you can follow her on Twitter at @AdvocateDebM.
Why this patient advocate is concerned about the newly proposed Medicare Part B model regarding its level of transparency and the evidence.
Pricing Based on Evidence of Value
And now, to my second major concern regarding the recent CMS announcement of a Part B Drug Payment Model. As noted in Part I of this article, CMS is proposing using value- or “indications-based” pricing according to a medication’s different levels of clinical effectiveness for different disease indications. The example they use is a case in which a new medication is indicated for 2 different types of cancer—where the drug was significantly more effective than existing treatment for one cancer type, but not so for the other cancer type. CMS notes that their use of indications-based pricing may lead to higher payments when the medication is used to treat the first cancer type and lower payments when it is used to treat the second cancer type.
CMS notes in the Federal Register that “The Institute for Clinical and Economic Review (ICER) is currently producing reports on high-impact drugs that analyze comparative effectiveness and cost-effectiveness before calculating a benchmark price for each drug…ICER's reports reflect the dependence of the value of medications on evidence available for certain target populations.” They then explain their proposal to use indications-based pricing when appropriately supported by high-quality evidence, including “published studies and reviews or evidence-based clinical practice guidelines, such as the ICER reports,” to align medication pricing with patient outcomes for specific clinical indications. They stress reliance on robust scientific evidence from “neutral and/or independent sources,…randomized trial designs where possible, and measures outcomes,” while understanding that the quality of the evidence that is available can greatly vary for any specific drug agent or disease indication.
In my roles as a cancer research advocate, patient, and cancer survivor, I’m always deeply concerned about anything that interferes with evidence-based, patient-centered clinician and patient decision making—including governmental mandates that are not rooted in the science—and misleading, inaccurate, or overly simplistic discussion about complex medical and health topics in the popular media that may cause individuals to make dangerous decisions about their health due to such misinformation. I’m similarly concerned when the facts surrounding such complex information become buried or are lost either inadvertently due to passionately held opinions and personal experiences, or perhaps purposefully due to the inconvenience of facts that may fly in the face of a mission or agenda.
And this leads to my distress concerning the volley of arrows that has recently been aimed at ICER due to its mention in this CMS proposal—where it was named as one potential source of evidence that could underpin proposed approaches to value-based pricing for injectable drugs in CMS’ Part B program. As I’ve described above, much of the focus and debate on the overall CMS proposal has been on the ASP pricing changes—again, concerns I strongly share—yet my worry is that ICER’s and others’ proposed value-based approaches are being cast in the same wide net that is directed toward the CMS proposal as a whole. Unfortunately, several inaccurate and misguided characterizations of ICER’s mission, goals, and practices have recently emerged as a result.
“It’s all about the evidence.” Those who have read my blog, Musings of a Cancer Research Advocate,” will recognize this as the blog’s tag line. As an independent patient advocate serving on multiple scientific advisory panels and committees, a critical quality for my involvement is that the organizations and entities in question stand for evidence-based positions. Accordingly, it is this independent, robust focus on the evidence that caught my attention years ago when I first learned of ICER’s work. It is what led me to accept an invitation to speak at one of their policy round tables, and it later resulted in my being asked to serve as an Advisory Board member of ICER’s New England Comparative Effectiveness Public Advisory Council (CEPAC).
ICER’s mission is to help provide an independent source of analysis of evidence on effectiveness and value to improve the quality of care that patients receive. Further, ICER’s development of its value framework was explicitly structured to be deeply patient-centered, capturing what is most important to patients in their care—and with the intent of bringing more transparency to negotiations between insurers and the life science industry on coverage and pricing on new interventions and enabling patients, clinicians, and multiple additional stakeholders to have full impact on these critical discussions. In fact, ICER proactively seeks and works directly with patients, advocates, patient advocacy groups, clinical experts, and clinical specialty societies to obtain their input on what truly matters most to patients and those clinicians who care for them, orienting their report process around these clinical outcomes. What has become lost in this contentious discussion of the CMS proposal is that the first and most important step ICER takes with every review is a robust evidence-based analysis of clinical effectiveness.
The reality is that, of course, reasonable people may have very different opinions concerning the optimal approach to evaluating the value of medications and other health interventions. It is critical to respect such differences and to engage with them openly. ICER consistently demonstrates this through the structure used in the development of its reports and its public meetings, which ensures ongoing opportunity for these differing voices to be heard. It’s also understandable that any efforts to evaluate the evidence and provide guidance can lead to strong reactions. But it’s concerning when different perspectives, again whether due to passionately held opinions or interests or facts that do not fall into place with one’s mission, result in loud reactions that are colored by false narratives, lack of understanding, and/or misrepresentations. For example, in recent days, ICER has been said to “clearly be inspired” by the UK’s National Institute for Health and Care Excellence (NICE), to base their analyses solely focused on drug costs, to use “opaque methods” to review and assess the value of specific medications, and to represent the interests of the insurance industry. And who could miss former Arkansas’ Governor, Mike Huckabee’s, deft revisiting of the term “death panels” as he loudly misinterpreted ICER’s mission and its work?
That is why it’s so important to break through the noise and clearly stress the following: ICER’s work is focused on improving the use of evidence as a foundation for open, transparent, public dialogue—something that has been lacking for far too long—concerning clinical effectiveness, innovation, cost, and sustainability in today’s healthcare system. Rather than “representing the interests of the insurance industry” or following popular opinion, political ideologies, or biases, ICER and its reports follow the evidence. Each ICER report includes full analyses of the drugs’ clinical comparative effectiveness, including efficacy, toxicity, potential benefits, and potential harms and the value these treatments represent to patients and the healthcare system.
Yes, some ICER reports have found that the comparative effectiveness of a new medication is extremely limited (eg, ICER’s report on insulin degludec (Tresidba), a new long-acting insulin analog to meet the basal insulin needs of patients with type 1 or 2 diabetes mellitus) while other reports have found that the evidence is persuasive (such as its review of mepolizumab (Nucala), a humanized monoclonal antibody against interleukin-5, found to demonstrate a net health benefit when added to standard of care for the treatment of patients with severe eosinophilic asthma). And in some cases, ICER reports have found that the exceptional value of a new medication improves patient outcomes while also saving overall healthcare costs (eg, its review of Entresto, a combination of the angiotensin II receptor blocker valsartan and the neprilysin inhibitor sacubitril), which found evidence of clinical effectiveness and high value as compared with usual care with ACE inhibitors, due to improvements in clinical outcomes and quality of life and reduction in costly hospitalizations and other complications). These evidence-based reports support ICER’s overall goal of getting excellent, high-value drugs to market quickly, at a cost that is affordable to patients, without hindering the development of new and effective drugs.
So, importantly, ICER’s primary focus is its assessment of clinical efficacy—but its secondary focus on value and cost should not be thrown out with the bathwater.
Be sure to tune in to Part III tomorrow, where Ms Madden discusses the increasing financial struggles many patients with cancer or chronic conditions are experiencing, and the approaches several groups are now taking to bring greater awareness and a higher level of science to considerations of cost and overall value of care.