Winning FDA Approval Not the Finish Line for Medical Device Companies

Marc Oczachowski, CEO of EDAP TMS, opens the kimono to talk about his company’s arduous journey with high intensity focused ultrasound for treating prostate cancer, from the FDA PMA and 510k clearance process to obtaining a CPT code for insurance reimbursement.  Oczachowski says many companies believe FDA approval is the Holy Grail and expect revenues to start rolling in and carry them to reimbursement.  But they often run out of money before they reach the finish line.  The article first appeared in MDDI on August 23, 2019, and was re-printed with permission.

By Marc Oczachowski

Medical device companies attempting to market a new treatment in the U.S. often make the erroneous assumption that winning approval from the US Food & Drug Administration (FDA) is the final destination, and success is theirs. In fact, going through the regulatory process is only the halfway point. The grueling path from FDA approval to reimbursement is like a treacherous walk through Death Valley and many companies don’t make it through.

In this case study, Marc Oczachowski, CEO of French medical device manufacturer EDAP TMS, discusses the trials and triumphs of bringing robotic high intensity focused ultrasound (HIFU), a non-invasive procedure for localized prostate cancer, to the U.S.  Oczachowski shares lessons learned as EDAP TMS began its 14-year journey, starting with the FDA pre-market approval (PMA) process, shifting to the more expedient de novo 510k and winning FDA clearance.  Then getting through the “Death Valley gauntlet” to finally win the American Medical Association’s (AMA) approval of a new Category 1 CPT code for treating prostate cancer –and reimbursement from insurers. 

Jumping through Hoops: the Rigors of FDA Regulatory Approval

We faced a protracted process when we first applied for Pre-Market Approval (PMA) for EDAP’s first HIFU device. HIFU is a non-invasive option that had been in use in Europe, Asia and South America since 2000.  It allows the surgeon to destroy only the diseased portion of the prostate, thereby preserving healthy surrounding tissue and nerves. As a result, HIFU minimizes the risks of side effects typically associated with the more aggressive treatments like radical surgery or radiation therapy, including erectile dysfunction and urinary incontinence.

But the FDA does not recognize clinical data outside the U.S. – so the long-term studies of patients conducted in France and Germany showing high overall rates of cancer survival over five and 10 years had no bearing on the agency’s assessment of our application. 

Based on FDA feedback, we commissioned a three-year, multi-centered clinical study of 135 US patients to evaluate the efficacy of HIFU as a treatment for localized prostate cancer. We partnered with a leading urologist at Duke University, as well as urologists from high level institutions around the U.S. to conduct this research. 

The study demonstrated the efficacy of cancerous tissue ablation using HIFU. Because the study mandated by the FDA was performed on low-risk patients and the two-year follow-up was too short to draw a conclusion about oncological control, panel members could not confirm the effectiveness of our device as a prostate cancer therapy. To win approval of HIFU as a treatment for localized prostate cancer would have required a long term, randomized multi-centered study with more than 1,000 patients.

At that point the FDA recommended that we apply for clearance of the HIFU device for prostate tissue ablation, based on a de novo 510(k), rather than seek approval as a device to treat a particular disease.  The FDA clears medical devices that are equivalent in safety and efficacy to existing devices. After approximately six months (October 2015), the FDA cleared HIFU for prostate tissue ablation based on the results of the same 135-prostate cancer patient study. But it took 10 years altogether to navigate the FDA path and receive clearance. We also had to convince urologists and hospitals to take a bet on our first generation device, since patients would have to pay out of pocket for the procedure because it was not yet covered by Medicare or private insurance.  

Fortunately, once a medical device is FDA cleared and new iterations of the same technology are released, the approval process is faster and clinical data outside the U.S. becomes acceptable. When we launched our second generation HIFU system [called Focal One®], we could submit clinical data from several European studies, and that expedited FDA clearance.

When we finally got market clearance for Ablatherm® in the U.S., Focal One, which combines MRI and biopsy data with ultrasound imaging, had been approved two years earlier and was already in use throughout Europe, Asia and South America. 

Focal One is designed specifically for focal treatment of the prostate. It fuses MRI and 3D biopsy data with real-time ultrasound imaging, which allows urologists to view integrated, detailed 3D images of the prostate on a large monitor and direct high intensity ultrasound waves to ablate the targeted area. 

With Focal One, urology surgeons can establish precise contours around the diseased tissue and ablate an even smaller portion of the prostate.  This lessens the damage to healthy tissue, and minimizes side effects of incontinence and impotence for patients.  Using Focal One, surgeons can customize the HIFU procedure for each patient and each clinical condition.

Brian Miles, MD, a renowned Houston urologist who performed the HIFU procedure at Houston Methodist Hospital using our first generation device observed Focal One in Germany and wanted to bring the new device to the hospital even before it was FDA cleared.

Dr. Miles observed, “Focal One gives Houston Methodist Hospital urologists the ability to plumb the depths of something until recently considered heresy.  The possibility of focal therapy to ablate only the diseased portion of the prostate is like performing a lumpectomy to remove only the diseased tissue of the breast in women with breast cancer.  We can now take only the infected tissue and leave the healthy tissue intact.  Focal therapy still leaves doctors with the options of radical surgery or radiation, should the cancer return. They don’t burn any bridges.”

Focal One received FDA clearance in June 2018.

Obtaining Medical Codes and Getting Payers to Reimburse for HIFU 

While winning FDA clearance for our first and second generation HIFU devices was a 13-year, complex process and a huge accomplishment, our journey was far from over.  The next hurdle was obtaining medical codes to facilitate reimbursement from public and private payers which can take two to four years. The pathway to reimbursement is where many companies fail because they don’t produce enough revenue following clearance to take them the rest of the way.

So as soon as the FDA cleared our first HIFU device we proceeded with our reimbursement strategy.  First, we worked with the Centers for Medicare & Medicaid Services (CMS) to establish atemporary Healthcare Common Procedure Coding System (HCPCS) C-code. In that process we had to educate Medicare Administrative Contractors (MACs) on the value of HIFU. The C-code, which took effect in July 2017, allows hospitals offering HIFU to submit a claim for Medicare patients although it does not include the urologist’s fee for the procedure, nor does it guarantee that the hospital or patient will be reimbursed. 

And the C-code was only part of our reimbursement strategy.  In 2018, we also began working with the American Urological Association (AUA) and the American Association of Clinical Urologists (AACU) to submit an application to the AMA for a new Category 1 CPT code, specifically for ablation of malignant prostate tissue.  So while we pursued clearance of Focal One, we also began a concurrent path toward securing the CPT code and reimbursement.  

We brought in urologists from top medical universities that were using our device, as well as the AUA and the AACU’s reimbursement committee representatives. We also worked with our competitor, SonaCare Medical, knowing that the AMA prefers to see competitors work together to develop the CPT code language.

Finally, in May 2019 we presented our case at the AMA CPT Panel meeting and the following month the AMA accepted our application to establish the new Category 1 CPT code that will facilitate reimbursement for the HIFU procedure. The CPT code description selected by the Panel is “Ablation of malignant prostate tissue, transrectal, with high intensity focused ultrasound (HIFU) including ultrasound guidance.”  

The code will be included in the next CPT Codebook, effective January 1, 2021. The CPT code will make it possible for men with localized prostate cancer who have the HIFU procedure to be reimbursed by their health insurer. By issuing a CPT code, the AMA is validating the clinical benefits of HIFU so it can take its rightful place as a standard procedure, alongside prostatectomy, radiation, cryotherapy, etc.

Three Key Take-Aways for Companies Marketing Medical Devices in the U.S. 

There is a lot to learn from our journey. Other companies trying to bring medical device technology into the US will have a long-and-winding road. There is success in great technology, hard work and perseverance. Here are my top three recommendations: 

• Make informed decisions about what you can afford and be prepared to engage for the long haul to reach the finish line. 
• Ideally we all want to have the right clinical study to get FDA approval but more often than not, you have to strike a balance between establishing enough clinical data to present to the FDA, and compromising where necessary to sell your technology.
• Be prepared to close the gap between regulatory clearance and private insurers covering the cost of a procedure.