The power of the right diagnosis...

Similarly, Bob Werner*, a 56 year-old patient with a long history of chronic pancreatitis, learned from an endoscopic ultrasound examination that there was a mass in the head of his pancreas, raising concern from his doctor that a malignancy was present. But pancreatitis often mimics pancreatic cancer and interpretation of a fine needle aspiration of the mass favored a benign, reactive process.

In both cases, treatment options included monitoring the tumor with the risk of its getting worse, or removing it surgically. The operation, called a pancreatectomy or “Whipple” procedure is a serious one with a high rate of complications, including diabetes.

In both cases, neither the pathologists attempting to read the microscopic slides, nor the GI specialists evaluating their respective patient’s history (including imaging studies and laboratory testing) could confidently conclude that it was, or was not, cancer.

Three years ago, there was no definitive or unequivocal diagnosis for cases like these. Although cancer is a complex, molecular-based disease, traditional diagnostic methods are based upon microscopic review of cell size and shape, sometimes leaving doctors with too little information to arrive at a firm diagnosis. And when front line pathology fails, patients find themselves cycling through processes of second opinions, testing and retesting, delayed treatment, and sometimes unnecessary or inappropriate treatment.

Fortunately, a new genomics-based technology exists that provides a definitive and unequivocal diagnosis for those situations when traditional methods are limited. RedPath Integrated Pathology’s PathFinderTG®, is a powerful new tool that uses a panel of 15-20 gene markers to construct a DNA fingerprint of a biopsy sample. It arms physicians and patients with an accurate, objective and clear picture of what’s going on, offering strong support for the right treatment plan.

Thankfully, for Susan and Bob, diagnoses came swiftly and definitively when their biopsies were sent to our laboratories in Pittsburgh. Susan learned that her lesion was non-cancerous and was able to avoid unnecessary surgery. Her tumor has shrunk considerably in the two years that have passed since the test.

Bob was diagnosed with pancreatic adenocarcinoma, one of the most aggressive of all cancers. Under traditional diagnostic conditions, a patient has virtually no chance of survival once the disease has spread beyond the pancreatic area. Thanks to PathFinderTG, there was no need for further and time-consuming confirmatory tests or follow-up surveillance; Bob was immediately scheduled for a Whipple procedure and his chances for survival increased dramatically.

A New Company in an Emerging Industry...

Launched with the commercialization of PCR technology and accelerated by the Human Genome Project, the $1.56 billion molecular diagnostics market is estimated to reach $3.7 billion by 2010. With an early emphasis on infectious diseases and microbiology, the field is now seeing explosive growth in oncology with a range of applications that spans screening, diagnosis, predictive testing, and chemo-sensitivity.

All of this is leading toward a more targeted and personalized approach to medicine for aging generations of baby boomers who have seen the growth of cancer in our lifetimes evolve from what was once a rare and secretive disease to a shared experience of afflicted loved ones. The juxtaposition of technology advances and the demands of informed and inquisitive “boomers” will drive the diagnosis and treatment of cancer to new levels of reduced morbidity and mortality.

RedPath is among a growing group of genomics-based companies that are advancing the molecular diagnostics industry in new directions – both in oncology (as evidenced by the examples above), as well as in the very nature of the questions our test can answer. Unlike others along the range of oncology-related diagnostics, RedPath’s PathFinderTG stands at the center of diagnosis itself. While it is not front-line pathology per se, it is pushing pathological science to an entirely new level, resolving previously indeterminate and equivocal diagnoses with precision and accuracy.

PathFinderTG provides a personalized and integrated approach to analyzing tissue specimens of all types, and answers key questions that beleaguer pathologists and clinicians. Is this benign or malignant cell proliferation? Is this tumor a recurrence or a metastasis of a past cancer, or is it a new primary cancer? Is this low grade or high grade dysplasia? PathFinderTG can be applied to a wide range of specialties, e.g., cancers of the GI tract, pancreas, brain, breast and genitourinary.

With advances in imaging and molecular-based screening tools, the numbers of diagnostic dilemmas facing pathologists today is actually on the rise. Detection alone is not diagnosis. An integrated approach to pathology that marries a required review of specific areas of concern with molecular analysis arms pathologists with a powerful tool and advances the science of pathology.

New Technology…New Challenges

Inventing a new technology is only the first of several critical steps in fulfilling the promise of a new approach. To become available to the patients it is intended to serve, the technology must be clinically and rigorously validated, and then successfully commercialized - which means it needs to be sold…and it also has to be paid for. Further, it must comply with all appropriate current regulatory requirements while also anticipating regulatory changes that can arise seemingly overnight. Each of these critical components is a challenge for any company in the life sciences sector, but they are particularly daunting for a young company in an emerging industry.

In RedPath’s case, clinical validation began more than 15 years ago, in the laboratories of our scientific founder, Dr. Sydney Finkelstein, who dedicated his career as a pathologist to the pursuit of marrying standard pathology with new molecular methods. This pursuit included countless case studies and hundreds of peer-reviewed articles, all to prove that the test was possible, accurate, safe, and effective. But while the test was available to support physicians in several major cancer centers, its use was restricted to a limited population. In order for true clinical adoption to occur, it needed a broader market.

The PathFinderTG technology was commercialized in 2004. A small but committed team set up the lab, secured CLIA and CAP approvals, convinced first customers, and began the cycle of financing to fuel the company.

For RedPath, risk became a fulltime partner. Selling a new test to skeptical physicians who rightfully demand ongoing proof and validation of clinical efficacy is not an easy task. Selling the business case to prospective investors is equally hard.

But we found believers – clinical and financial. Traction was gained and test requisitions increased. Multi-institutional studies were convened. Best of all, patients’ lives were impacted. We know because they told us. And, we needed them to tell us…we needed their success stories in order to get paid.

As a new technology, PathFinderTG did not readily fit into the existing payment system. There were no standard CPT codes to use in our billing process. In a bifurcated system that already separates customer from payor, the introduction of a new technology with a miscellaneous payment code raises the risk quotient exponentially. As a businesswoman I can remember thinking that there was something inherently wrong about performing a complex, sophisticated test that could potentially result in life-altering circumstances for the patient—and still not knowing whether or not our bill would be paid. Yet, this is exactly the road we were on for three years as we fought (and continue to fight) for payor adoption. Recent Medicare approval has eased that burden tremendously, but we are still fighting for payment one claim at a time, until we secure enough critical mass with a given payor to negotiate a coverage policy and payment agreement.

And just when we think we understand the rules, they change. PathFinderTG, like many genomics-based technologies, is a laboratory-developed test (LDT). It is offered as a service out of our laboratory. We follow good laboratory practices and we meet all of our licensing and regulatory requirements. We knew we were in a regulated environment, but the degree to which reimbursement and regulatory changes would impact our ability to deliver services and to be paid for them cannot be overstated

In the past three years, we have seen attempts to place additional regulatory requirements on an already heavily regulated industry by such entities as the Centers for Medicare and Medicaid Services, the FDA and Congress. Moves to regulate invitro-diagnostic lab tests as devices and/or to establish competitive bidding for clinical laboratory services could have tremendous impact on the ability of companies like ours to grow and innovate. Likewise, new Medicare billing requirements that place unfair financial burden on our hospital clients can potentially restrict access for Medicare beneficiaries.

The good news for all of the stakeholders in our Company – especially patients like Susan and Bob- is that RedPath is succeeding. We can see pathology diagnosis and cancer management changing in a way that will benefit many patients. We have experienced tremendous growth, met significant milestones for a new company in a new industry, and see a place for ourselves in the exciting new world of molecular-based personalized medicine.