Moving Forward Together: MDMA and FDA

Though he admits to feeling like a “grim reaper” at times, Mark Leahey, CEO of the Medical Device Manufacturers Association (MDMA), is “actually an optimist” about the future of the industry. One of the reasons for his optimism is the recent FDA effort of enhanced transparency and accountability led by Christy Foreman, Director of the Office of Device Evaluation (ODE) at the FDA. Last month, Biodesign Alumni sat down with both Leahey and Foreman to discuss their work, challenges, and opportunities for help.

What is the MDMA?

Leahey, a long-time DC resident and recent collaborator with Josh Makower, MD on FDA reform, sees MDMA’s role as more than an advocate for startups – MDMA is a liaison between the needs of the Hill and those of small companies that often lack the access and resources to efficiently navigate the regulatory process. In large part, the MDMA is a grassroots effort to maintain relationships with key Members of Congress, senior staff at FDA, and CMS.  MDMA also engages in a series of educational programs for its members.  For example, MDMA held its two-day FDA Forum in Palo Alto this March. MDMA represents not only small and mid-sized med tech companies, but also the interests of other members of the ecosystem including VC’s, private equity and law firms.

Where are the MDMA and FDA headed?

According to Leahey, “one of the biggest mistakes industry has made in the past was focusing primarily on speed rather than predictability.”  This approach led to the policy of FDA to issue more rejections in order to meet the timing requirements. To address this, Foreman has been utilizing the Medical Device User Fee Amendments (MDUFA III) as an opportunity to hire more staff and change the Agency’s approach to review. The new approach moves from a linear model to more of a “funnel,” according to Leahey.  Foreman describes these changes as “starting with broad, comprehensive questions and progressing to detailed ones over time.” This approach provides a number of advantages: first, it allows improved timing predictability. Through the hierarchical approach, the Agency replies within 15 days for a 510(k) to give a company a sense of where it stands on major issues and assurance that new issues will not surface later in the review, a phenomenon that often led to delays. Second, it fosters a collaborative dialogue. Companies are encouraged to communicate early and often to explain experimental plans, findings, and mitigations. While the Agency does not give direction to company, it will provide feedback given anticipated outcomes of the work. Third, Foreman has set up performance metrics that focus on total response time (Agency plus company response time) rather than Agency time only. This motivates the Agency to consider a company’s ability to respond to questions and further motivates collaboration and efficiency.

These changes notwithstanding, companies must think carefully about when to elevate a disagreement with FDA profile. Leahey suggests that if FDA makes a request for data that a company thinks is unwarranted, but fairly easy to complete, you may want to consider just conducting the bacteria testing to move the process along. However, if there is a disagreement over a major issue, you are better served elevating the issue sooner rather than later. Regardless, companies must recognize that FDA is operating in a much more risk-averse environment than 5 years ago so strategies that worked then may not work now. In part, this may reflect the economic times that give buyers leverage on small companies.  In other words, the potential for substantial value creation may justify a small company’s choice to spend the time and money to perform required studies. This may be particularly useful advice as evidence from the Agency gives hope that a US first-in-man may be  close at hand.

Opportunities Ahead

Leahey describes one of the biggest medical technology challenges to be the need to demonstrate long-term economic value, a goal that conflicts with typical insurer’s time horizon of about 3 years. An important counter-example is the Kaiser Permanente (KP) healthcare system, where patients remain, according to Leahey, for 15 years. Its model allows KP to take a long view on managing patient outcomes.

One of the latest avenues of medical technology is m-health. These are technologies that provide diagnostics, clinical decision support, telehealth, and electronic health record (EHR) compatibility with the aim of reducing healthcare costs, while simultaneously improving outcomes. Though the FDA position is still evolving, indicators from Christy Foreman and MDMA point towards a relatively open policy in the foreseeable future, particularly with respect to applications that populate EHR. MDMA and FDA policy on apps that affect therapy, however, will be regulated as other medical devices and are subject to software validation similar to other medical technologies.

Advice for the Small Medtech Company

As new medical technologies begin to identify needs, Foreman and Leahey agree on a few important goals for the small med device company looking to interact with the FDA:

• Patient focus. Focusing on the patient story provides more powerful advocacy than company marketing or the business case.  Often, the personal patient story is a straightforward and potent way to make a vivid case for medical technology.
• Take FDA satisfaction survey. With new regulations and leadership in place at the FDA, a collaborative approach ultimately optimizes chances of success and improves Agency predictability. These surveys are taken seriously at the Agency and have the ability to affect change.
• Provide data on economic value. Granular cost savings may make a compelling case for policy-makers and insurance companies alike. Though these cost-savings numbers may be difficult to obtain, both Leahey and Foreman agree that showing quantitative evidence of diligence across markets, patient segments, and healthcare providers will play an increasingly important role.
• Secure advocacy with diverse Congressional staff. While the FDA’s responsibility is to ensure safety and effectiveness, demonstrating potential for job creation and patient benefit across multiple geographies can be an important driver to garner support for a therapy.  This approach may be a compelling, yet often overlooked method of demonstrating economic value to ultimately drive product acceptance.

—

Pedram Afshar, MD PhD is a Senior Scientist with Medtronic’s Neuromodulation group, and was the 2009-2010 Biodesign Innovation Littlefield Fellow. He currently serves as CFO for the Stanford Biodesign Alumni Association.