AdvaMed is calling for legislative and regulatory reforms to the rare-disease-focused humanitarian device exemption FDA approval pathway.

Better incentives are needed for manufacturers to pursue HDEs, and FDA should be more flexible with its data requirements, Susan Alpert, chief regulatory officer for Medtronic, said in explaining the trade group's position during a June 30 FDA public hearing on rare diseases in Silver Spring, Md.

"Many of these technologies are going to come from small companies, and they need incentives in order to be able to spend their time and their energy on developing these products," Alpert said.

To enter the market with an HDE, a sponsor must prove safety and "probable" effectiveness for a product intended to treat a maximum of 4,000 patients per year.

A company is often restricted from pricing an HDE device for more than the per-item cost of R&D, fabrication and distribution, though 2007 congressional reforms let firms profit on HDEs for pediatric indications ¹ Also see "Guidance: How To Profit From Humanitarian-Use Devices In Pediatric Patients" - Medtech Insight, 11 August, 2008.).

Orphan Drug Program Raised As Model

To make the HDE program more attractive, Alpert suggested taking cues from the orphan drug program.

For instance, drug companies are awarded a seven-year period of market exclusivity for therapies approved as an orphan drug to treat rare diseases. A similar approached should be applied to devices, she said. AdvaMed took efforts to get exclusivity provisions for products studied in pediatric populations written into 2007 user fee reauthorization legislation, but it did not gain much traction (² Also see "Industry Dissatisfied With Senate-Approved Pediatric Device Provisions" - Medtech Insight, 14 May, 2007.).

Alpert also pointed to the orphan drug tax credit as a model. Further, she pressed for more leeway in the data requirements for HDE applications.

FDA has authority to request a variety of evidence types beyond randomized trials, Alpert stressed.

She suggested that the agency permit firms to conduct more bench and animal testing, rather than clinical trials, and to extrapolate data when appropriate.

Similarly, "more pathways are needed" to transition an HDE to a PMA, she asserted. "If you still have that very small population and you don't have capacity to develop the kind of data that will support the PMA, maintaining a product in the marketplace once you've recouped the individual development cost is a challenge," she said.

FDA should also enhance its communication with external parties to facilitate rare disease device approvals, Alpert said.

She recommended that the agency hire more rare disease experts to speed device approvals, appoint an ombudsman specializing in pediatrics and rare diseases, and work with manufacturers to generate predictable regulatory requirements to meet the "safety and probable benefit" standards for HDEs.

AdvaMed made similar suggestions (see sidebar: " ³ AdvaMed Recommends Steps To Address Orphan/Pediatric Devices ") for spurring the development of devices to treat rare diseases, as well as pediatric patients, at a June 24 FDA workshop on identifying unmet public health needs.

HDE Cap Of 4,000 Called Arbitrary

Alpert and other presenters also questioned the appropriateness of the 4,000-per-year cap for HDEs.

Robert Campbell, a pediatric orthopedic surgeon with Children's Hospital of Philadelphia, called the threshold "arbitrary," and suggested that 10,000 patients per year "might be a better benchmark."

Art Kessler, president of the Dystonia Medical Research Foundation, which funds studies and other efforts to assist people suffering from the rare neurological disorder, said that FDA's classification of the HDE devices as "investigational" creates problems in access to devices for treating the disorder.

For example, dystonia can be treated with deep brain stimulation, he said. Deep brain stimulation is performed via Medtronic's DBS system, which was approved as an HDE for chronic intractable dystonia in 2003. But because payers know the HDE designation as "investigational," they deny coverage. This is "a real barrier to patient access to these life-changing treatments," Kessler said.

"We encourage FDA to work to remove the investigational designation," he said. "Funding does not exist to conduct the clinical trials necessary to determine efficacy" for the product.

- Sue Darcey ( ⁴ s.darcey@elsevier.com ) and Jessica Bylander ( ⁵ j.bylander@elsevier.com )

Industry Seeks Reforms To FDA Humanitarian Device Exemption Program

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