Personalized Medicine Education and Advocacy

Thought leadership in personalized medicine

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Can We Assess the Value of Personalized Medicine in Treating Cancer?

Guest Blog
from Dan Leonard, M.A., President, National Pharmaceutical Council


Dan Leonard, M.A.

Although there is a bright spotlight on the field of personalized medicine thanks to the Obama administration’s Cancer Moonshot and Precision Medicine initiatives, there are real concerns about how targeted medicines will be considered in value assessment frameworks, which are geared toward evaluating treatments for a population rather than individual patients.

This week, National Pharmaceutical Council (NPC) President Dan Leonard sat down with Amy M. Miller, Ph.D., Executive Vice President, PMC, to discuss these issues.

Dan Leonard (DL): Amy, thank you for joining me to talk about personalized medicine and value frameworks. Tell us a little about personalized medicine and its impact on patient care, especially in light of ongoing debates about costs, coverage and value.

Amy Miller (AM): Thank you for this opportunity. Personalized medicine is an evolving field in which physicians use diagnostic tests to determine which medical treatments will work best for each patient. By combining the data from those tests with an individual’s medical history, circumstances and values, health care providers can develop targeted treatment and prevention plans. This concept challenges how health care products and services are discovered, developed, regulated, covered, paid for and delivered in the clinic. Therefore, it is no surprise that personalized medicine challenges how value assessments are conducted.

DL: The basis for personalized medicine is that every patient is unique and will respond to treatments differently, something NPC also has demonstrated in our research. How can a value assessment framework take that important concept into consideration?  

AM: Fortunately for value assessment framework designers, many organizations have published suggestions for them. It is important for the value assessment questions to accurately reflect available data. Furthermore, when looking at disease areas with targeted therapeutics, value assessment frameworks must look at the full complement of clinical tools, including the diagnostic test or tests and the clinical outcome differences between a targeted and non-targeted treatment approach.

DL: The Institute for Clinical and Economic Review (ICER) is currently evaluating treatments for non-small-cell lung cancer (NSCLC), a disease that is unique to each patient and requires targeted treatment. They’ll be hosting a public meeting about that evaluation on October 20. What kinds of factors should ICER be considering as part of its review of NSCLC treatments?

AM: NSCLC is a disease where personalized medicine has transformed care over the last decade, and patients have seen tremendous improvements in morbidity, mortality and quality of life as a result. We’ve seen evolution in treatments targeting many driver mutations in the tumor. We’ve seen evolution in the types of diagnostics used to select those treatments. I think ICER needs to carefully consider all that we’ve learned over the last decade using EGFR-mutation therapeutics, value those therapeutics from a patient’s perspective, and consider how we incentivize investments in PD-1 therapeutics so we can capitalize on their tremendous potential in a similar way. PD-1 inhibitors work quite well for a subset of patients, but we do not know how best to use them yet. We will figure it out, and when we do, it will likely have tremendous implications for patients, giving them longer, better lives than comparators like chemotherapy.

DL:  What have you heard from your member companies about ICER’s review of NSCLC treatments? Are there ways that ICER could better integrate those comments, as well as patient input?

AM: ICER concurrently opened a public comment period where stakeholders could suggest process changes.  One example of a process change that ICER could make now is to include a more representative group of stakeholders on its advisory council. Furthermore, historically, ICER has discussed the implications of its decision after a vote on the value assessment. Simply listening to the public before voting would reassure stakeholders that ICER values their input.

DL: You had asked ICER to provide a longer comment period for NSCLC, which they granted. Do you think that was enough time? How could they improve the comment process in the future?

AM: PMC is a coalition representing pharmaceutical manufacturers, diagnostic companies, patients, providers, payers and other stakeholders, and the perspectives that these groups have together are often valuable to those who seek input on their work. However, for a coalition to engage a group like ICER requires that our members consider the issues from their perspective before joining a conversation about how to support a concept. ICER’s comment period (30 days and, in this case, including holidays) did not allow for us to engage.  For a document of this magnitude and import, we suggested allowing for a 60 – 90 day comment period, which conforms to other organizations’ timelines. We hope ICER will consider that.

DL: These are exciting times for personalized medicine, with rapid developments in understanding this science and finding new cures. With these developments, how could — or should — ICER update its reports to remain current?

AM: In the case of NSCLC, new clinical evidence was published during ICER’s comment period, and for PD-1 inhibitors, I think we’ll see new data coming in more than once a year going forward. Because ICER is not alone in the value assessment trade, I’d suggest that the field of value assessment coordinate and come up with best practices for updating their findings. That way, innovators, patients and providers will have a timetable to engage with the update.

DL: Thanks so much for speaking with me.

For more about value framework assessments, check out NPC’s Guiding Practices for Patient-Centered Value Assessment and Current Landscape: Value Assessment Frameworks and watch the video from the organization’s conference, Assessing Value: Promise and Pitfalls.

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Beyond the Barriers: Deconstructing the Regulatory and Reimbursement Hurdles for Companion Diagnostics

Guest Blog
by Alessandra Cesano, M.D., Ph.D., Chief Medical Officer, NanoString Technologies


Alessandra Cesano, M.D., Ph.D.

On November 16, a group of diagnostic industry representatives will convene to discuss the regulatory and reimbursement hurdles for personalized medicine diagnostics during the 12th Annual Personalized Medicine Conference at Harvard Medical School. These kinds of discussions have never been more important.

The push for personalized medicine came to the national forefront in January when President Obama announced the National Cancer Moonshot. This bold initiative aims to accelerate the discovery of personalized treatments tailored to an individual’s genetic profile and/or the tumor’s biology. Companion diagnostics (CDx) play an important role in precision medicine, as they are designed to enrich care for patients who will benefit from a “companion” drug, by helping to characterize the disease’s biology and matching it with the mechanism of action of a specific drug.

Because many of the new drugs in the pipeline work on a specific genetic or biological target that is present in some, but not all, patients with a certain kind of cancer, there is a need for an accompanying test to determine if the drug will or will not have a benefit for a specific patient. These tests may also point to which patients are at immediate risk for harmful side effects.

The promise of companion diagnostics is not under debate, but there are regulatory and reimbursement hurdles that need to be overcome before these tests achieve widespread acceptance and deliver on the promise. First is the cost. The development of a companion diagnostic requires a significant investment, along the lines of tens of millions of dollars. However, presently, the value that companion diagnostics bring to the health care system, specifically in terms of improving patient outcomes and effectiveness of the care delivered, is not appropriately recognized by the reimbursement system.

Supporting the development of CDx tests will require significant investment up front, but once adopted they will help the health care system realize considerable cost and time savings. Currently, the health care system favors the “one-size-fits-all” approach to drug delivery, despite the fact that the subgroup of patients benefiting from treatment is on average only 20 – 30 percent. By using CDx, we can enrich the patient population for which a specific drug is effective, resulting in better outcomes and significantly reduced costs for the health care system. Designing and executing appropriate clinical trials to demonstrate the “clinical utility” and cost-effectiveness of selection biomarkers in each particular clinical setting will be an important part of the evidence needed to obtain test reimbursement.

Another obstacle involves how CDx and personalized medicine have impacted the regulatory landscape. While the regulatory path to a CDx is relatively well defined by regulatory guidelines, a gray area remains on when/how a  “generic” version of those tests (aka laboratory-developed tests or LDTs, which are analytically developed by a single laboratory without a clinical validation requirement) will be regulated by the FDA. This uncertainty has negatively affected the investor community’s appetite for diagnostic companies.

In light of these hurdles, a promising solution to driving CDx adoption is partnership among diagnostic manufacturers and the pharmaceutical industry. Biopharmaceutical companies are developing many therapies and as a result need to enroll patients in many hundreds of clinical trials. The clinical development of their drugs is going to demand enrichment strategies based on biomarkers. In fact, I can see a future in which it is the exception, rather than the rule, that drugs don’t have biomarkers when they come to market.

The biopharmaceutical industry is going to work with in vitro diagnostic companies that have the technology and the capabilities to both analyze the tumor’s biology and build an in vitro diagnostic product that can win clearance from the FDA. Ideally, these tests will be able to cut across whole drug classes (targets). Because of a limited supply of biotissue, the tests will need to be as holistic as possible. Thus, multi-plexed assays that allow for investigation of multiple aspects of biology in a single sample would be preferred.

Because of their unique ability to “match-make” a tumor’s biology with the right therapeutic choice, companion diagnostics are important for the efficient and effective treatment of patients. If the National Cancer Moonshot and other initiatives are going to be successful, there needs to be an alignment among all the stakeholders — including regulators, payers, pharmaceutical companies, physicians, patients and advocacy groups — recognizing the value of companion diagnostics in making “precision medicine” not just a promise but finally a reality.