Recently, Senator Al Franken (D-MN) called on Congress to create extra incentives for medical device makers to tackle rare diseases, according to an article in The Hill.
In making his opening remarks at a Senate Health Education Labor and Pensions (HELP) Committee hearing in late July, Franken noted his “concern that we still don’t have equivalent incentives for devices as we have for drugs.” His comments came as the hearing, entitled “Treating Rare and Neglected Pediatric Diseases: Promoting the Development of New Treatments and Cures,” focused “on ways to promote new treatments and cures for rare and neglected pediatric diseases.”
Consequently, he told the Committee that he thought more could be done, and looked forward to working on the issue, especially since Minnesota is home to several medical device giants, such as Medtronic.
Accordingly, because developing devices and investing in the necessary research and trials is so significant, and the risks are tremendous, medical device companies “say they need government help to make their investments in rare diseases worthwhile, because the affected populations are too tiny for them to turn a profit.”
One set of proposals that was offered at the hearing to help address funding of medical devices to combat rare disease came from the Advanced Medical Technology Association (AdvaMed). President and CEO Stephen Ubl said in a statement that we need to “create a regulatory environment where innovation to address these unmet needs can thrive.”
In his proposal, Ubl “suggested the Food and Drug Administration (FDA) start by developing general guidance for its humanitarian device exemption (HDE) program outlining appropriate types and levels of data necessary for approval.” Through the HDE program, devices are exempt from scientifically proving that they are effective for their intended purpose if they treat or diagnose diseases and conditions affecting fewer than 4,000 people in the United States from having.
This exemption would allow device companies to continue developing and testing their devices without having to overcome significant administrative and regulatory burdens that can costs billions of dollars and years waiting in the pipeline.
Ubl further called on Congress to “create a strong pediatric device R&D tax credit program as well as a tax credit for pediatric HDEs similar to the tax credit that currently exists for orphan drugs.” He asserted the need for this kind of tax credit because “medical technology companies also face potentially enormous (research and development) costs in developing new pediatric devices with little hope of recouping their investment due to the small market for some products.”
Bringing devices, which can treat rare and pediatric diseases, through the regulatory system in a more efficient way also benefits patients and physicians because it gives companies and inventors the ability to train, teach and work with doctors sooner. This exchange gives companies and physicians tremendously important data, hands-on experience and feedback. Moreover, by using a device sooner, companies and doctors can collaborate and learn about strengths and weaknesses in devices that occur in clinical care simultaneously so that the final product can be constantly updated and perfected. If a device is left in the pipeline waiting for approval and to pass regulatory muster, further defects and delays down the road could mean a much longer waiting process for patients, and no one should lose their childhood waiting for a device.
With 31 million people being added to the U.S. healthcare system over the next few years, including large amounts of children, we will need more devices and newer technology and innovation to help treat children and adults with complex diseases and conditions. Since the research and development of pharmaceuticals already have similar government benefit programs in place, it seems logical for Congress to extend such benefits to device makers.