Author Archives: Real World Health Care Editorial Staff

Advancing Melanoma Research & Supporting Patients

Our series on melanoma concludes as we shine the spotlight on the Melanoma Research Foundation (MRF). We spoke with the MRF’s Director of Education, Shelby Moneer, MS, CHES and MRF’s Scientific Advisory Committee Co-Chair, Michael B. Atkins, MD, PhD, Deputy Director of the Georgetown-Lombardi Comprehensive Cancer Center, Georgetown University.

The MRF is the largest independent organization devoted to melanoma. Its mission is three-fold. It supports medical research that leads to more effective treatments and eventually a cure for melanoma. It educates patients, physicians and the public about the prevention, diagnosis and treatment of melanoma. And it serves as an advocate for the melanoma community to raise awareness of melanoma and the need for a cure.

Research & Patient Support Priorities

Real World Health Care: What type of research programs does the MRF support?

Shelby Moneer, Melanoma Research Foundation

Shelby Moneer: The Melanoma Research Foundation is committed to advancing a broad scientific agenda across the disciplines of prevention, diagnosis and treatment. As part of the MRF’s mission to support medical research to find effective treatments and a cure for melanoma, we are proud to collaborate with internationally recognized research institutions, investigators, government entities and leading melanoma organizations. The MRF currently offers research awards for medical students, junior investigators and senior investigators in both clinical and translational science. We fund research related to cutaneous, mucosal and ocular melanoma.

RWHC: How does the MRF support the patient community?

SM: The MRF provides a broad range of patient support programs offering access to peer-to-peer educational information, as well as professionally moderated support networks, financial and treatment assistance and more. These are continually updated and expanded as new resources become available because we believe that educated and informed patients with a strong support network live longer, better lives.

The MRF advocates for the melanoma community on the local, state and federal level on issues that promote melanoma awareness, prevention and treatment. Current priorities include increased federal funding for melanoma research, restrictions on the use of indoor tanning devices and expanded access to sunscreen in schools. The MRF trains and supports a nationwide network of dedicated volunteers who serve as advocates everywhere from their local communities to the halls of Congress.

RWHC: What are some of the biggest challenges facing researchers studying melanoma?

Dr. Michael B. Atkins, Georgetown-Lombardi Comprehensive Cancer Center

Michael Atkins: The MRF feels that the biggest challenge is helping to launch the careers of young investigators who are interested in doing research into the biology and treatment of patients with melanoma. The MRF is addressing this challenge by offering an expanded number of Career Development grants in the coming year targeted toward young investigators.

Treating Melanoma Patients

RWHC: How are clinicians addressing the challenges of treating patients with melanoma?

MA: For clinicians, the big challenges are learning how to optimally use new treatment options for patients with melanoma. For example, identifying how to integrate targeted therapy with immune therapy, when to stop immune therapy, how to treat patients with variant melanoma presentations (acral, mucosal, uveal) and patients with CNS metastases, how to treat patients with resistance to immunotherapy, determining the best adjuvant therapy for patients with resected high and intermediate risk melanoma, identifying predictive biomarkers for particular treatment approaches, identifying safe and effective combination regimens, and learning how to manage the novel toxicities of immunotherapies.

These questions can only be answered through continued clinical research. The MRF is addressing these challenges through the Melanoma Research Foundation Breakthrough Consortium (MRFBC), which brings together clinical investigators from 20 leading academic melanoma centers to design and conduct clinical trials addressing these types of questions.

RWHC: How can patients work with clinicians to optimize their treatment?

MA: Melanoma patients face the challenge of getting the best treatments for their disease in a setting that is convenient for them and will get them back to their normal lives as quickly and as functionally intact as possible. Patients can work with clinicians by participating in clinical trials aimed at both providing tomorrow’s treatments today and addressing the important unanswered questions facing researchers. Patients can also go to organizations like the MRF to gather information about optimal management of their disease, unanswered questions and available clinical trials. Their support community can work with and contribute to MRF to foster the clinical and basic research that will lead to even further advances in the treatment of patients with melanoma.

RWHC: What does the MRF see as the most promising treatments on the horizon for melanoma?

MA: The MRF sees many promising new treatment opportunities including novel combination immunotherapies, triple combination of BRAF/MEK and anti-PD1 regimens and moving effective treatments for advanced disease into the adjuvant high risk setting.

Categories: General, Melanoma

Cutting-Edge Melanoma Treatment at Rutgers Cancer Institute of New Jersey

This week, Real World Health Care continues our series on melanoma by interviewing two colleagues from the Rutgers Cancer Institute of New Jersey, a National Cancer Institute-designated Comprehensive Cancer Center.

Sharad Goyal, MD, is Associate Professor and Director of Clinical and Translational Research in the department of Radiation Oncology. He treats patients with brain tumors, melanoma and skin cancers, providing comprehensive cancer evaluation and the latest treatment planning technology to “map” tumors. He designs radiation treatments with pinpoint accuracy, ensuring that tumors get the most effective dose while healthy tissues and organs are spared.

Ann W. Silk, MD, is a medical oncologist who cares for patients with melanoma, and other skin cancers. She also leads clinical trials focused on combination treatment with immunotherapy and viral therapy for melanoma and other cancers.

Drs. Goyal and Silk discuss their work as part of a multidisciplinary team that translates research of investigational treatments and directly applies them to patient therapies.

Managing Multiple Melanoma Brain Metastases

Real World Health Care: How is treatment advancing for patients with multiple brain metastases (MBM) from melanoma?

Sharad Goyal; Faculty and Staff, Rutgers – Robert Wood Johnson Medical School, New Brunswick, NJ. 05/01/2014 Photo by Steve Hockstein/HarvardStudio.com

Sharad Goyal: The treatment of brain metastases from melanoma is controversial and includes surgical resection, stereotactic radiosurgery (SRS) and whole brain radiation (WBRT). Several new classes of agents have revolutionized the treatment of metastatic melanoma, allowing for subsets of patients to have long-term survival. Given this, management of MBM from melanoma is continually evolving.

As patients are living longer due to more effective systemic therapy, surveillance and management of intracranial disease is increasingly important. At this time, the standard of management for patients with MBM from melanoma includes SRS, WBRT, or a combination of both.

In addition, emerging data supports the notion that SRS, in combination with targeted therapies or immune therapy, may reduce the need for whole brain radiation. Prospective studies are required to fully evaluate the efficacy of these novel regimens in combination with radiation therapy. Given the advances in systemic therapy of melanoma, it is critical that oncologists treating these patients be aware of new treatment paradigms to optimize the outcomes for all patients with metastatic melanoma.

RWHC: Are there ways to treat melanoma in order to avoid or lessen the chances of brain metastases? Does early initial detection of melanoma help?

SG: Early detection of melanoma will always help reduce the chance a patient develops brain metastases. There is usually a fairly lengthy period when the tumor expands beneath the top layer of skin but doesn’t go any deeper. This allows time for screening, early detection, treatment, and a full recovery if the tumor is discovered before it spreads.

After a patient is diagnosed with melanoma, the use of certain anticancer treatments that are given after a cancer is surgically removed, such as interferon alfa and ipilimumab, will allow for an improvement in overall survival in patients with stage III or IV melanoma. In addition, the use of anti-PD-1 checkpoint inhibitors prolongs overall survival in patients with stage IV melanoma. Currently, many studies are underway investigating the optimal anticancer treatment in patients with Stage III melanoma.

Promising Therapies

RWHC: What are the most promising therapies on the horizon for metastatic melanoma?

Ann W. Silk, MD, Rutgers Cancer Institute of New Jersey

Ann Silk: Melanoma is the “posterchild” for breakthrough immunotherapies. At Rutgers CINJ, we are testing novel combinations of new agents coupled with immunotherapies. Those novel agents help to “prime” tumors so immunotherapies work better. One example of this is combining an immune checkpoint inhibitor pembrolizumab with intra-tumoral injections of talimogene laherperepvec, which is a live herpesvirus that infects cancer cells, replicates within them, and lyses the cancer cell, thus killing the cell. We’re also seeing promising early results in trials that combine intra-tumoral injections of coxsackievirus with injections of pembrolizumab, with response rate of 60%, which we reported at this year’s American Association of Cancer Research Annual Meeting. We have just recently opened a study in which we will combine IL-2 cytokine therapy with pembrolizumab, a combination of two drugs that have already demonstrated good activity in metastatic melanoma.  They are each FDA-approved as single-agents, but we are studying them as a combination therapy. The goal of these studies is to build on the success of immunotherapies to increase response rates, particularly complete response rates.

Access to Clinical Trials

RWHC: What are some of the biggest challenges facing researchers studying metastatic melanoma?

AS: Access to clinical trials is a challenge, from a patient or participant status. Running clinical trials requires vast resources and numerous support personnel, so they tend to be concentrated at large medical centers. As a result, only about five percent of the patient population has access to these trials. I think that number should be closer to 80 percent. Trials aren’t only important for the patients receiving the treatment; the knowledge we gain also helps future patients.

Treating Melanoma

RWHC: What are the biggest challenges facing clinicians treating patients with metastatic melanoma?

SG: One of the most challenging types of cancer to treat is melanoma and the most challenging area it can spread to is the brain. With the advancements being made in cancer treatment, the odds of survival for many of these patients are changing dramatically.

In recent years, our understanding of cancer biology has progressed substantially and this has led to the development of targeted therapies and immunotherapies. These novel therapies have prolonged survival in a disease which previously had a dismal outcome. As patients are living longer due to more effective systemic therapy, surveillance and management of intracranial disease is of increasing importance.

AS: There are many standard therapies approved to treat melanoma, and as indicated earlier, there’s lots of research activity in this area as well. Patients benefit from the wealth of approved therapies on the market. But while about 30-40 percent of patients will respond very well to these therapies, the majority still struggle and succumb to their disease. Patients who don’t respond to the first line of treatment must move on to a second line of treatment. Once those treatment options are exhausted, the only course of action is to try and get the patient enrolled in an investigational therapy trial. In many cases, the patient would benefit from earlier involvement in clinical trials.

Early and Current Inspirations

RWHC: What initially interested you in researching melanoma and treating people with the disease? What continues to inspire you?

AS: I became interested in the interplay between cancer and the immune system in my first job as a data manager at the Dana-Farber Cancer Institute, where I managed one of the first clinical trials of ipilumumab, which was later FDA approved with the distinction of being the first drug that prolonged survival in melanoma. I was inspired by researchers who were on the forefront of personalizing medicine by using the immune system to attack cancer cells. Ever since then, in both my research and clinical practice, I haven’t found any better ally in the fight against cancer than retraining the immune system to attack cancer cells.

SG: Throughout my career, I have been inspired by the cancer patients I treat.  The relationships that I have developed with patients and their families are unlike those in almost any other medical specialty. Once a patient has a diagnosis of cancer, I am able to see the patient and his or her family members on a regular basis and develop a long-lasting relationship with them.

New Series to Launch: Alzheimer’s Disease and the Central Nervous System

According to a report from the Alzheimer’s Foundation of America, as many as 5.1 million Americans may have Alzheimer’s Disease (AD), a figure that is expected to triple to nearly 13.8 million by 2050. Alzheimer’s is the sixth leading cause of death in America, and individuals with AD use a disproportionate amount of health care resources compared to people with other diseases.

While there currently is no cure for AD, researchers are optimistic that effective treatments are on the horizon. We look forward to highlighting some of this research with you as we launch our series on Alzheimer’s disease and the Central Nervous System. We’ll also be sharing perspectives and programs from leading Alzheimer’s patient advocacy groups.

We encourage you to check back here over the coming months, and don’t forget to join the discussion on Twitter via @RWHCblog.

 

Big Data in Healthcare: Speaking with Dr. Philip Bourne, National Institutes of Health

Our series on Big Data in Health Care continues this week with a conversation with Dr. Philip Bourne, Associate Director for Data Science, National Institutes of Health. Dr. Bourne discusses the goals of the NIH Big Data to Knowledge (BD2K) program and the challenges faced in leveraging big data to improve health outcomes.

Real World Health Care: Why did the NIH establish BD2K?

Dr. Philip Bourne, National Institutes of Health

Dr. Philip Bourne, National Institutes of Health

Philip Bourne: Several years ago, NIH Director Francis Collins set up a data and informatics working group in response to the increasing amounts of digital data being generated in biomedical research. That working group led to the development of BD2K.

RWHC: What are the goals of BD2K?

PB: As set out by the working group’s report, the goals of BD2K are to promote the “fair” finding, access, sharing, incorporation and re-use of digital content and analytical tools within the entire spectrum of health care. Our goals also include promoting enhanced and diversified training around the process of analyzing large amounts of data and achieving sustainability of the complete digital biomedical ecosystem.

RWHC: “Big data” is a big buzzword these days, and it’s being leveraged among a wide range of industries with varying degrees of success. Where does the health care industry currently stand in terms of its overall ability to generate, gather, analyze and share big data toward the goal of improving positive health outcomes?

PB: I think there’s a good analogy here between revolutionary changes in other industries and the potential revolutionary change that big data may bring to health care. Take the photography business as an example. When photography went digital, it disrupted the industry and created a completely different business proposition. Today, the photography industry has less to do with pictures and more to do with visual communications platforms like Instagram.

The same kind of disruptor has the potential to happen in health care with the digitization of information. The disruption is happening slowly, even though the growth of digital content has been exponential. Next, we need to get to the “infection point” where big data takes off and becomes disruptive.

Because health care is not a true free market economy like photography, there are more restrictions. Today, the patient really does not have control over his or her health information, but if they had such control, it could be transformative.

RWHC: What are some of the biggest challenges facing the health care industry in terms of its ability to use big data to improve health outcomes?

PB: One of the biggest challenges is the lack of qualified professionals and training for those professionals in conducting analytics. Big data also represents a cultural shift in the industry as we move away from traditional ways of doing research to newer analytical methods. We need more education on the implications of that shift.

RWHC: Where is the health care industry seeing the most success in using big data to improve health outcomes, especially as it relates to health care delivery, treatment optimization and cost containment?

PB: The industry is just beginning to define and use data in different ways, and early stage successes haven’t been widely publicized. With that said, our new ability to mine health data records and analyze them to identify changes is statistically significant and provides important predictive tools. For example, researchers at Stanford are studying body mass indexes (BMIs) in specific regions to see if there is any correlation between BMI and the amount of fast food restaurants in those regions.

RWHC: Are there any individual BD2K programs or projects about which you’re particularly excited? What sort of initiatives can we expect to see from BD2K during 2016?

PB: We’ve recently been focusing on privacy, which is a clear issue when it comes to human subjects: How does the industry protect patient privacy? We’re also bringing in different types of professionals who have experience in analytics, but in other industries such as digital media and entertainment. We recently had a funding call in this area and are looking forward to seeing how these non-health care professionals apply their skills to biomedical problems — how the entertainment industry can help us visualize large amounts of data in a meaningful way.

We also continue to focus on developing the Commons, which is a shared virtual space where scientists can work with the digital objects of biomedical research. We have a mandate from the federal government and the NIH to promote the sharing and accessibility of research output: outcomes of clinical trials, papers, software and other data. The Commons lets us do that. It’s kind of like putting a bunch of Lego blocks in a public square and seeing what people can do with them.

Categories: Big Data, General

New Series: Big Data in Health Care

Welcome to 2016! The staff and sponsors of Real World Health Care wish you all a Happy & Healthy New Year. We look forward to another year of sharing ideas and insights from researchers on the forefront of real-world medical breakthroughs.

Next week, we’ll be kicking off a new blog series on implications of the use of big data in health care. From predicting disease and identifying targeted therapies and cures to improving our overall quality of life, big data is transforming the way health care decisions are made and care is delivered.

Over the next couple months, our guest bloggers will address issues including:

  • The use of analytics to identify and manage high-risk and high-cost patients.
  • How to extract clinical value from biomedical big data.
  • The use of big data to understand the human condition.
  • The role of large prospective randomized trials in the era of big data.
  • Distinctions between research and operational data within a “continuously learning” health system.
  • Implications of big data science for nurse leaders.

We look forward to sharing these expert views with all of you and hope that Real World Health Care continues to be a source of useful information throughout 2016.

Personalized Medicine & Companion Diagnostics: Speaking with Dr. Razelle Kurzrock

Real World Health Care recently sat down with Dr. Razelle Kurzrock, Director, Center for Personalized Cancer Therapy & Clinical Trials Office, University of California San Diego Moores Cancer Center, to discuss the present state and future direction of genomics and immunotherapy.

Real World Health Care:  Why was the Center for Personalized Cancer Therapy formed at Moores Cancer Center? What was its initial goal and has that goal changed?

Dr. Razelle Kurzrock, U.C. San Diego Moores Cancer Center

Dr. Razelle Kurzrock, U.C. San Diego Moores Cancer Center

Razelle Kurzrok:  The strategy for the Cancer Center really began to evolve with the arrival of Director Scott M. Lippman. We worked together at MD Anderson Cancer Center, and we both had an interest in moving into the precision medicine and personalized cancer medicine field. Based on our experience, we thought the Center could be transformative for patients with cancer. So, we started with a business plan that focused on the Center’s essential components: vigorous clinical trials, researching hereditary predispositions in cancer, genomics and immunotherapy. That plan and those components became the cornerstone for what we wanted to build and who we wanted to recruit.

The only change since then is that our scope has continued to grow. The field of genomics and immunotherapy has become more exciting. We also realized we needed a strong educational component, primarily because physicians were not overly familiar with genomics and immunotherapy, so we added a fellowship in personalized cancer therapy. We also opened a rare diseases clinic. Because some rare cancers don’t have an FDA-approved therapy, we look at these patients from a genomic or immunotherapy standpoint right from the start.

RWHC: How are patients selected to participate in your Experimental Therapeutics program?

RK:  We have clinical trials and therapies for patients in all disease groups. But in addition, we have early clinical trials and genomically targeted or immunotherapy trials within Experimental Therapeutics.  These trials are not disease based.  Included in the Experimental Therapeutics trials are phase I studies that do not concentrate on a particular cancer.  In the past, Phase I trials were considered as dose-finding trials. Today, these Phase I trials are more exciting and have therapeutic impact.  Even the FDA is now occasionally approving drugs after Phase I testing. Included in Experimental Therapeutics are basket or umbrella trials, which look at cancer from immune or genomic points of view, instead of as “breast” or “colon” cancer. Both early-stage and umbrella trials don’t always fit well into the disease site program, so we developed our Experimental Therapeutics program.

We also wanted to develop a new way of looking at cancer. We choose patients for Experimental Therapeutics based on what we think is best for the particular patient at hand. Our tumor board reviews patients, and if they feel a trial in a disease-based program is warranted, that’s what is recommended. However, if biological characteristics indicate that patients would do best with a genomic or immune approach that is not disease based, the patients are funneled into our Experimental Therapeutics program.

RWHC: Can you give an example of a type of experimental immunotherapy that is being tested?

RK:  While there are a number of novel molecules to talk about, I think it is our overall approach that is of interest. Immunotherapy is exciting…but it’s so exciting that the tendency is to give these therapies to everyone. Instead, we investigate the biomarkers that identify the patients who will respond well to a particular therapy. We know there are certain patients who will have a wonderful response to a particular immunotherapy drug, but other patients won’t. So we try to apply the personalized medicine approach to immunotherapy. We are now starting to identify biomarkers that will tell us which patient is best for immunotherapy and which may not benefit.

RWHC: Since creating the Center for Personalized Cancer Therapy, has Moores Cancer Center seen an increase in positive patient outcomes? If yes, can you please explain to our readers?

RK:  It certainly generates a lot of buzz when you have patients who were expected to die soon, but thanks to personalized cancer therapy, they are alive and doing well a year or two later. While these individual cases are important, we cannot rely on anecdotes.  We therefore also analyze data on patient outcomes in a systemic way, with a protocol that lets us evaluate patient outcome data on more than a case-by-case basis. This is our PREDICT program. In fact, we just submitted a paper on the first 450 patients to go through our program. The data shows improved outcomes in almost every parameter, so that good buzz we’re sensing is corroborated by solid data.

RWHC: What types of companion diagnostics is the Center for Personalized Cancer Therapy conducting to identify the best course of treatment for each individual patient?

RK:  We certainly take advantage of FDA-approved companion diagnostics, based on label indications. However, we’re also very involved in working with different molecular diagnostic assays and looking at patients to see what those assays tell us about the patient. We want to know if those assays help us predict the best drugs for the patient.

RWHC: What type of enhancements might we see in the future of personalized medicine if researchers can use genomics on blood samples to predict patient outcomes or response to treatment?

RK:  This is a really exciting area that we are working on within the Center’s liquid biopsy program. These are “biopsies” that look at DNA in the blood stream.  In effect, one can do the analysis on a small tube of blood.  And, it goes beyond the blood stream. We are also looking at genomics of DNA shed by tumors into the urine. These assays are still in a young phase and under development. But it’s amazing that we can take a blood or urine sample and use the genomic information in those samples to better understand response even before the patient gets a CAT scan. It can take months before a CAT scan shows response, but our research is beginning to suggest that urine or blood tests can give early information about both responses and resistance to treatment. We still have a lot work to do to prove the accuracy of the correlations, but when that happens, it will be revolutionary for the field.

Personalized Medicine & Companion Diagnostics: Speaking with Keith Stewart, Director of the Mayo Clinic Center for Individualized Medicine

Editor’s Note: This week, we sit down with Keith Stewart, director of the Mayo Clinic Center for Individualized Medicine.

Real World Health Care:  Why was the Center for Individualized Medicine formed at the Mayo Clinic? What were its initial goals and how have those goals changed?

Keith Stewart, Mayo Clinic Center for Individualized Medicine

Keith Stewart, Mayo Clinic Center for Individualized Medicine

Keith Stewart:   The Center was formed in 2012 with the idea that it would harness the power of the human genome to improve health care for our patients. It was considered to be one of three transformative initiatives for the future of the Mayo Clinic and a discipline in which Mayo Clinic should be a leader.

RWHC: How has personalized medicine and the work you’re doing at the Center for Individualized Medicine helped the Mayo Clinic to improve health outcomes?

KS:   By using the genome as a lifetime resource and not just a “one and done” test, we believe we will lower the costs of health care. For example genomic knowledge will improve the precision of diagnosis, reduce unnecessary testing, allow the right drug at the right time and ultimately improve health outcomes.

RWHC: What types of companion diagnostics are being conducted at the Center for Individualized Medicine to identify the best therapy for individual patients?

KS:          Many. One good example is pharmacogenomics where we have created 18 drug alerts in the electronic health record already. But we have many other examples: cancer gene panels in prostate cancer, glioblastoma, myeloma, sarcoma, and colorectal cancer. Panels in cardiac disease and neurology, for example in peripheral neuropathy, epilepsy, and movement disorders. And, of course, whole genome sequencing for families with rare diseases.

RWHC: In your opinion, what is the most exciting translational research being conducted at the Center for Individualized Medicine?

KS:   I am very excited about our work in the microbiome and how that will impact human health and how we might use genomic sequencing in infectious disease to identify pathogens that are hard to culture. But there are many such areas. We will be sequencing the pharmacogenomes of 10,000 of our patients and launching clinical trials in the areas of organ transplant and immune-oncology next year.

RWHC: Where do you see the future of blood cancer-related personalized medicine and companion diagnostics heading?

KS:          As an example we have built and are launching a gene panel in myeloma which identifies mutations but will also call common translocations. If successful this should replace the era of conventional cytogenetics and FISH testing. The same will be true in acute leukemias and lymphomas. A major area of interest is in immune-oncology and we will be launching trials in this area next year to understand how genomics can select for patients most likely to respond.

RWHC: When full-genome sequencing becomes routine, what sort of information do you envision healthy people obtaining and applying as a result of having their genome sequenced?

KS:          I think the answer may not be what most people expect. Yes, we will find medically actionable things such as carrier status and pharmacogenomics, but to me, the most important thing might end up being what is negative. As an example, when I had my genome sequenced, it struck me that I would never again have to have any other genetic testing done for the rest of my life. So, if I have a blood clot, cancer, or develop Parkinson’s or dementia, I already know I am negative for the currently understood genetic risk factors.

Personalized Medicine & Companion Diagnostics: Speaking with Dr. Joshua Cohen, Tufts Center for the Study of Drug Development

Editor’s Note: In August, the Tufts Center for the Study of Drug Development (CSDD) hosted a roundtable of R&D leaders focused on development of companion diagnostics that can show their use in conjunction with personalized therapeutics that will lead to positive health outcomes. We spoke with Joshua P. Cohen, Ph.D., Research Associate Professor, Tufts CSDD about the promises and challenges in the field of personalized medicine.

Dr. Joshua Cohen

Dr. Joshua Cohen

Real World Healthcare: According to Tufts CSDD, 20 percent of new drugs winning approval in the U.S. last year were considered personalized medicines. What do you think is driving the growth you expect to see?

Joshua Cohen: More investment in the science of biomarker identification and validation, and more investment in the commercialization of personalized medicines and diagnostics.

RWHC: What reimbursement problems, if any, do you see for companion diagnostics?

JC: There are two challenges concerning companion diagnostic pricing and reimbursement. The first is coding. Traditionally, diagnostics have been code-stacked — coded for each individual activity involved in the preparation and use of a diagnostic. Each code is then assigned a price and, when taken together, the prices of individual codes make up the price that diagnostic manufacturers get reimbursed. Code-stacking does not, however, reflect the value of a diagnostic. It only reflects the price of individual components.

The value of a diagnostic is reflected by the second pricing and reimbursement challenge: clinical utility — the linkage between a companion diagnostic and positive health outcomes. The more clinical utility a diagnostic has the greater the chance it will be reimbursed and the higher price it can command. If a diagnostic differentiates between likely responders and non-responders, the value of that differentiation should be reflected in the diagnostic’s price.

RWHC: What can drug and diagnostic companies do to accelerate the development of biomarker efficacy and remove this key hurdle to the development of personalized medicine?

JC: Identification of biomarkers early in development. Coordination and communication with regulators early in development, as the regulatory processes for diagnostics and therapeutics are different. Also, use of next-generation sequencing to develop diagnostics, in which biomarkers with predictive claims undergo rigorous clinical (cross) validation.

RWHC: When it comes to personalized medicine, even high R&D success rates may not mean much if physicians won’t prescribe it and payers won’t reimburse it. Are you aware of any hesitancy to entering the space by the industry?

JC: There may be some hesitancy on the part of the biopharmaceutical industry because personalized medicine alters the blockbuster model. This said, many newly approved personalized medicines have high price tags. In some cases, these high price tags have made them blockbuster drugs (e.g., Herceptin, Gleevec). Physicians will prescribe personalized therapeutics as long as evidence suggests it does a good job at differentiating between likely responders and non-responders to a particular therapeutic, or indicates which sub-group is at risk for certain adverse effects. Similarly, payers will reimburse personalized therapeutics and companion diagnostics if evidence supports their effectiveness and safety. An issue has come up with respect to awareness on the part of the physicians about personalized medicine, and specifically the role that diagnostics play. In cases in which there is less awareness of the need to employ a certain diagnostic, less clinical adoption will occur.

RWHC: What fields of medicine are furthest along in development of personalized medicines?

JC: Oncology dominates. There is a better understanding of the science behind targeted therapies and the role that biomarkers play.

RWHC: Why do you like this field?

JC: It represents the promise of individualizing treatments, rather than relying on an iterative, trial-and-error method.