In the US, the FDA (Food and Drug Administration) approves new medical devices. The CMS (Centers for Medicare & Medicaid Services) decides whether to cover the cost. The UK approximation of this is the MHRA (Medicines and Healthcare products Regulatory Agency) for approval and NICE (National Institute for Health and Care Excellence) for whether to cover. In Europe, the EMA (European Medicines Agency) handles approval via CE (Conformité Européenne) marking and health technology assessment bodies support EU payers making their own reimbursement decisions.

These organizations are under relentless pressure from patients, the public, clinicians and industry to rapidly approve new devices. And the same parties are ready to come down hard on regulators if a device turns out to be ineffective and/or unsafe with accompanying loss of public trust.

We all want patients to have access to the best treatments. We don’t like the idea that patients might die before the treatment that could have saved them was approved for use. This is an emotive driver for fast-track approvals. But it is important for regulators to keep the brakes on until there are assurances about safety (and effectiveness!). In principle, early approval with the safety net of post-marketing studies is a good approach – PMA (Pre-Market Approval). However, in practice, post-marketing studies have to be enforced and withdrawing a therapy after approval can be more problematic than never having approved it at all.

The Trump administration had recently allowed the CMS to reimburse devices with breakthrough designation and PMA approval while post-marketing studies are being completed. This was scheduled to be effective from 15th March 2021. This is controversial as it lowered the bar for new devices with a promise of post-marketing studies. More specifically in cardiovascular disease, such as heart failure, some are concerned that the bar for approval is lower for  devices benefiting from breakthrough designation,  than for drugs targeting the same indication and unmet need.

Very recently, the Biden administration has placed a freeze on implementing this rule whilst it is reviewed meaning it could be delayed or even not implemented at all. Naturally, device companies are disappointed as it affects their timelines, and return on investment. On the one hand, raising the bar discourages innovation because it becomes non-viable to bother innovating and on the other hand, patients and payers could get better value. I’m on the side of wanting better value, and I’m cynical about “breakthrough” devices (and especially cynical about the ability of anyone to identify such a device before it gets properly assessed in a double-blind placebo-controlled setting).

Most new devices are, at best, moderately effective with risks that just about don’t outweigh the benefits and, at worst, downright harmful. A “breakthrough” should be the rare exception to this rule i.e. significantly effective and acceptably safe. Then the argument for expedited approval (PMA) and CMS reimbursement would be a no brainer. Unfortunately, we are unable to tell a device apart from a “breakthrough” device.

Faiez Zannad says “the CMS is concerned about new FDA-reported data that more than 400 devices have been designated as breakthroughs to-date, which is obviously an excessive use of the breakthrough definition. So, rather an FDA issue. It is likely that all breakthroughs are not real breakthroughs. Too much leniency would kill the notion of breakthrough. An example of a reasonable use of the breakthrough designation is the Barostim device, after the BeAT-HF trial. This pivotal trial was actually the very first trial to be designed and conducted under the Expedited Access Pathway / Breakthrough Device Designation. The device delivers baroreflex stimulation to heart failure patients with reduced ejection fraction and persistent symptoms and was approved on the basis of improved quality of life and exercise capacity and decrease in NT-pro-BNP, all surrogate endpoints accepted by the FDA as the basis of a “conditional approval”, when  all 3 are influenced favourably and concomitantly. The planned scheme mandated that BeAT-HF continues enrolment and follow up, post approval, as an event driven trial with heart failure hospitalization and cardiovascular death as the primary endpoint.”

The definition of a breakthrough device for the FDA is that the device has to provide treatment or diagnosis of a life-threatening or irreversibly debilitating disease and has to meet one of the following criteria:

  1. Represents breakthrough technology
  2. No approved or cleared alternatives exist
  3. Offers significant advantages over existing approved or cleared alternatives
  4. Device availability is in the best interests of patients


So this is a bit of a pickle because while speeding up Medicare coverage of FDA-approved devices is a laudable initiative for patients accessing new treatments, if the FDA “breakthrough” pathway is too lenient, then coverage will be applied prematurely to many unproven devices. Obviously alignment between regulators and payers is highly desirable in order not to disappoint patients with approved devices which they may not be able to afford, and innovative fragile small companies, who most frequently develop such devices, with approved devices having no market access plan.


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