On September 19, 2019, the FDA issued a draft guidance on the Safer Technologies Program for Medical Devices (STeP), which was created as a complement to the Breakthrough Devices Program. Similar to the Breakthrough Devices Program, the purpose of STeP is to help patients have more timely access to innovative devices and device-led combination products, expedite device development and review, and foster collaboration between FDA and sponsor. STeP shares many of the attractive features of the Breakthrough Devices Program such as early and more frequent engagement with FDA through sprint discussions and regular status updates, as well as coordination with FDA on a Data Development Plan (DDP) which could utilize least burdensome data collection approaches over the entire total product lifecycle. A complete dataset of clinical evidence is not needed to be considered for inclusion in STeP—only a demonstration of a reasonable expectation for technical and clinical success.
However, the similarities end there. STeP targets indications that are associated with morbidities and mortalities that are less serious than those eligible for the Breakthrough Devices Program. In other words, these diseases and conditions are not considered to be life-threatening or irreversibly debilitating. Moreover, STeP focuses on improving device safety, while the Breakthrough Devices Program focuses on improving device effectiveness.
For inclusion into the program, the device or device-led combination product should meet a general eligibility factor as well as two specific eligibility factors. It is important to note that these eligibility factors, unlike the Breakthrough Device designation criteria, are presented as recommendations and not requirements (i.e., use of “should” versus “must”). This is because the Breakthrough Device designation criteria are statutorily mandated through 21st Century Cures, while the STeP eligibility factors are not.
The general eligibility factor is that the device or device-led combination product should be subject to marketing authorization via PMA, De Novo request, or 510(k) pathways. This factor basically excludes any device that is class I and/or exempt from 510(k). For device-led combination products, the proposed safety improvement should be made to the device constituent part.
The device or device-led combination should also meet the following two specific eligibility factors:
- Devices or device-led combination products “should not be eligible for the Breakthrough. Devices Program due to the less serious nature of the disease or condition treated, diagnosed, or prevented by the device;” and
- Devices or device-led combination products “should be reasonably expected to significantly improve the benefit-risk profile of a treatment or diagnostic through substantial safety innovations that provide for one or more of the following:
- A reduction in the occurrence of a known serious adverse event,
- A reduction in the occurrence of a known device failure mode,
- A reduction in the occurrence of a known use-related hazard or use error, or
- An improvement in the safety of another device or intervention.”
For the first specific eligibility factor, the draft guidance clarifies that the target disease or condition should be considered non-life-threatening and reasonably reversible. Such diseases or conditions could affect patient quality of life or be debilitating for short timeframes, their health consequences might not significantly impact daily function, and/or they might not progress to a more serious disease or condition. STeP arguably casts a wider net in this regard in contrast with the Breakthrough Devices Program.
The second specific eligibility factor seems trickier to address and can be considered the criterion that makes or breaks the decision for a device to be included in STeP. Although this factor explicitly looks for an improvement in the benefit-risk profile, it appears that parts (a) through (d) of the factor is more focused on decreasing risk and not increasing benefit. The draft guidance clarifies that the known safety concerns should be officially identified in an FDA Safety Communication or recall, or “otherwise identified as a significant safety issue of public health importance.” The “otherwise” phrase in this explanation can be quite subjective. Device risk can be categorized on the basis of severity, types, number, rates, probability, and duration of harmful events. What are the thresholds for each of these descriptors and how are these thresholds established? Hopefully, FDA will demystify these aspects in finalizing the guidance.
This is complicated with FDA’s recent initiatives in the patient preference realm. Risk tolerance varies among patients and affects patients’ decisions as to whether they are willing to accept higher risks for a higher probable benefit. Will FDA consider patient preference information (PPI) in meeting the second factor for inclusion into STeP? The draft guidance is silent on the role of PPI in FDA’s decision-making.
Moreover, the draft guidance states that FDA will consider devices and device-led combination products that have the potential for safety improvements over the current standard of care (SOC), which may only include FDA-approved drugs or biologics, or other technologies. This suggests that FDA will consider safety concerns that are not directly device-related. If the SOC does not incorporate routine use of a device, it may be difficult to determine if the device is actually providing for safety improvements in the SOC. At best, the device may not disrupt the SOC at all, and at worst, the device may introduce new safety concerns or even compromise the effectiveness of the SOC, which may only be realized in the long term.
STeP also raises some interesting questions from a policy perspective. The draft guidance clarifies that the acceptance of a modified device into STeP will not impact the obligations or responsibilities of a manufacturer with respect to any recall or correction. However, the draft guidance does not discuss how STeP may affect proposed or recent reclassifications. When a device is associated with significant safety signals, FDA may consider increasing its regulatory control over the device through upclassification. If affected sponsors are able to enter STeP with the goal of mitigating these safety signals, how much would this good-faith effort help stay the upclassification? If a class I device that would normally not be eligible for STeP was recently upclassified to class II, could STeP provide a basis for reversing the upclassification?
STeP promises a safety-based regulatory pathway consistent with FDA’s mission in protecting and promoting public health. However, the draft guidance seems to raise more questions than solutions. Ultimately, the success of STeP would largely depend on the details of its implementation. FDA is requesting public comments be submitted under docket number FDA-2019-D-4048 by November 18, 2019. Parexel will stay abreast of any updates regarding STeP and will keep our clients informed.
Parexel’s subject matter experts are comprised of former regulators, industry professionals, physicians, and commercialization specialists with a proven track record of providing end-to-end solutions for our clients in global markets. Our experts have broad experience in traditional therapeutic and diagnostic medical devices as well as novel and innovative technologies such as combination products, digital health, and personalized medicine. From obtaining STeP or Breakthrough Device designation to marketing authorization and beyond, Parexel can help you every step of the way throughout your total product lifecycle.