biospecimens

Guest:

Anna Barker, Co-Director, Complex Adaptive Systems Center, ASU Bio and Contact Info

Listen (3:21) Andy's challenge

Listen (4:39) The year of immunotherapy

Listen (4:53) iSpy 2 and a new approach to clinical trials

Listen (6:42) The swan

Listen (6:35) A coalition of the willing

Listen (5:14) Six reasons biomarkers fail

In 2003, the then Director of the National Cancer Insitute, Andrew von Eschenbach, issued a challenge: “to eliminate the suffering and death from cancer, and to do so by 2015.”

Anna Barker is the former Deputy Director of the National Cancer Insitute, and she was at the meeting when Dr. von Eschenbach issued his challenge. Now at the end of 2014, we talk with Anna about the challenge and how far we have come. She offers her vote for the three most important developments in cancer this past year.

We perform therapeutic and diagnostic clinical trials to learn the safety and efficacy of drugs and tests in a clinical setting.  Typically, we think about the biospecimens in a clinical study in terms of operational requirements such as sample collection, processing, storage and analysis.  Our ability to obtain and test high quality patient samples is an important consideration during the design of clinical research programs and may significantly limit the questions that we can ask in trials. 

For example, our ability to develop drugs for precision medicine in diseases such as cancer depends on access to high quality samples.  The fundamental elements of a trial are the objectives--what questions we intend to address--and the endpoints--what will be measured to answer those questions.   Trials are also defined by the patient population or the inclusion and exclusion criteria determining who is enrolled.   Successful clinical studies require careful planning and design of the study protocol to ensure that the study is feasible and that we capture the lessons learned.  Often, endpoints rely on patient samples for laboratory testing. 

Patient enrollment may require rapid laboratory testing for a molecular or genetic alteration to meet the inclusion criteria.  Researchers also collect patient samples for long term storage for future analysis in the hope that advances in technologies and disease knowledge will open new opportunities.  These study endpoints require robust biospecimen planning during the early development of research programs all the way through to the medical writing of the study protocol.   Study planning is typically based upon experience and expertise.  However, the industry is more frequently utilizing best practices and data-driven analytics and modeling. These tools will improve our planning and preparations for biomarker-driven trials that require biospecimens.

Targeted therapeutics and drug candidates that will rely on patient laboratory test results are becoming common in oncology and other diseases.  Diseases are also being reclassified by molecular biomarkers and complex laboratory tests.  As a result, the biospecimen considerations that have been discussed in the ongoing Biospecimen Series at Mendelspod are becoming critical success factors for drug development. 

In the planning stages we often would like to target a small patient population who may greatly benefit from a new drug compared to the general population.  We then have to ask if we can identify, test and enroll those patients if the samples for testing are difficult to obtain or unstable for shipment and testing.  Similarly, we may have a novel drug with a new mechanism of action that promises to address a critical clinical need.  In this situation we are often limited by the test to certain sample types that may cause a great deal of patient inconvenience or duress.   Difficulties in obtaining  repeated tumor biopsies is one example that has resulted in the development of tests that rely on surrogate samples that may be obtained from the blood.  These considerations often restrict many of our options for study designs include choice of diseases, patient populations and regions or clinical sites. These restrictions also impact the patient journey in the trial. 

Trial protocols summarize the collection and use of samples in a table or schedule of events or assessments.  What may appear to be a simple table, ultimately impacts the patient experience and determines trial feasibility and success.  Researchers must balance the potential knowledge gained, time, cost and risk to maximize the benefit of the study to current and future patients.  Biospecimen planning is increasingly an important part of this equation.  The Mendelspod series Back to Basics: Improving Biospecimens supports a greater understanding of the challenges and opportunities for improving how we utilize critical patient samples.  These improvements will also enable more powerful clinical studies, addressing challenging clinical questions and advancing novel drugs and diagnostics.

Guest:

Carolyn Compton, Professor of Pathology, ASU Bio and Contact Info

Listen (4:54) A historic new consensus

Listen (6:33) CAP committed to enforcement

Listen (2:50) Five core variables

Listen (5:16) Non-governmental leadership

Listen (4:20) Problem unique to biomedicine

Listen (3:25) Ramifications for our audience

Listen (4:48) Does this mean more work and higher prices?

A unique event happened at ASU last week that has the potential to positively impact all of biomedical research and therefore patients.

Carolyn Compton has been a tireless crusader for higher standards in biospecimen collection, handling, and storage. (See our interview earlier this year with Carolyn here where she argues that uneven sample quality is a major reason that biomedical research has such poor reproducibility rates.) Beginning as a pathologist herself, Carolyn has served at various organizations, including a stint at the National Cancer Insitute, which have prepared her for a special leadership role on the topic of biosamples. Last week she saw her long time dream come true.

“I’m delighted to report that we did in fact come to consensus after four days of discussion on what standards would be needed to raise the quality of ALL biospecimens from ALL patients to a standard that would be acceptable—perhaps not optimized—transparent and of known quality level for all patient samples. This is huge!” Carolyn exclaims in today’s show.

What does Carolyn mean by consensus? As we’ve been uncovering in our recent series on the topic, there have been no uniform standards in the collection, handling, and storage of biospecimens. It’s all been done with “poetic license” Caroly says. Until now. Last week Carolyn and a very special organization at ASU known as the National Biomarker Development Alliance put on two “convergence conferences” back-to-back that brought the major stakeholders—pathologists, physicians, patients, tool makers, and regulators—together AND found broad agreement on five basic standards to improve biospecimen handling.

This is indeed huge. Because the President and President Elect of the College of American Pathologists attended the conferences and committed to enforce these five basic standards in all of the labs which they accredit.

In her ebullience, Carolyn is careful to say that this is just the beginning, that the standards must now be written out and optimized. But it’s a very strong beginning which will have ramifications throughout the industry.

Who will be most affected? Patients, Carolyn says. Because tests, such as the popular HER2 assay for breast cancer, will be more accurate.

This story is not only unique in how it will change the course of the industry, but a powerful example of how non-governmental leadership can effect much needed change.

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