Posts Tagged ‘FDA’

FDA Outlines Personalized Medicine Policy with Publication of LDT Draft Guidance Document, Final Guidance on Companion Diagnostics

August 15, 2014

On July 31, FDA announced drastic changes to regulation for personalized medicine products and services when it coupled the release of the long-awaited final guidance document on the regulation of companion diagnostic devices with a proposed framework for regulating laboratory developed tests (LDTs), which was also long-awaited or long-feared, depending on your perspective.

The final guidance on In Vitro Companion Diagnostic Devices was welcomed by the personalized medicine community because in the document, FDA clarified the path for co-developed drug-diagnostic products, and finalized their assertion that new targeted therapeutics will not be kept from the market if the diagnostic kit is not ready at the same time. This enables promising new drugs to come to market while also allowing the laboratory community to fill testing needs in cases where an FDA-approved kit is not available for therapeutic selection, dosing and avoidance decisions.

However, many issues remain to be addressed.

To address concerns that FDA regulation will pose obstacles to an already challenged laboratory industry, there is a rather long transition phase — nine years — and an initial focus on high-risk. FDA defines high-risk LDTs as those with the same intended use as cleared or approved companion diagnostics, LDTs with the same intended use as an FDA-approved Class III medical device, and certain LDTs for determining the safety and efficacy of blood or blood products. This focus and transition period will allow clinical laboratories and FDA time to adjust. By focusing initial regulation on high-risk LDTs, FDA makes a strong argument for the framework, and slices off a rather small segment of the LDT market.

Many have argued that FDA does not have the bandwidth to regulate LDTs. FDA responded to this claim by reiterating enforcement discretion for the vast majority of LDTs and outlining a process for LDT regulation, which might be less onerous than traditional regulatory pathways for medical devices.

Although FDA made great efforts to address concerns about the Agency’s new regulatory enforcement, it did not address perceived conflicts between laboratory regulation under Clinical Laboratory Improvement Amendments (CLIA) and this new framework. Furthermore, FDA intends to use an expert advisory panel to provide recommendations to the Agency on LDTs risks and classification on certain categories of LDTs, as appropriate. I suspect that defining those categories will be contentious and, at times, difficult.

Investors have long argued that clarity is necessary in both regulation and reimbursement for continued advancement of personalized medicine. We now have clarity on FDA’s current thinking although many issues remain unresolved. The community has time to consider this framework and may soon have a chance to provide public comments. And finally, the pharmaceutical industry has the FDA’s assurance that targeted treatments will not be held up by co-development challenges.

BREAKING NEWS: FDA Notifies Congress of its Intent to Publish Framework for Regulatory Oversight of Laboratory Developed Tests (LDTs)

July 31, 2014

Summers in D.C. are notoriously slow. FDA, however, has added excitement to this summer by informing Congress of its intent to publish a long-awaited framework for LDT regulation.

In its notice to Congress, FDA included what appears to be a draft of the document. After the mandatory 60-day Congressional review, the draft guidance document will be formally issued for public comment.

Within the draft framework, FDA proposes a risk-based, phased system of oversight. They recognize community concerns around access and do not intend to interrupt the marketing and sale of currently available tests. Furthermore, FDA expresses the intent to continue using enforcement discretion for forensic and organ transplantation uses, traditional LDTs, and LDTs for unmet needs.

The document outlines the history of LDT regulation, FDA’s policy of enforcement discretion, and how personalized medicine has caused FDA to reconsider that policy.

We will continue to provide updates on the development of framework for regulatory oversight of LDTs, with additional in-depth commentary next week on this issue and the related news of FDA’s final guidance on companion diagnostics.

For additional information on the current regulation of LDTs, please read PMC’s report “Pathways for Oversight of Diagnostics.”

Balancing the Need for Guidance, Communications, and Education to Support Innovation in Personalized Medicine Diagnostics

May 13, 2013

Recently, I had the opportunity to moderate a thought-provoking panel at the PMC/BIO Solutions Summit. The summit brought together key stakeholders to discuss solutions to barriers in the development of innovative personalized medicine diagnostics. A big question for those developing potentially game-changing technologies in an increasingly cost conscious environment is the need for “Evidentiary Standards and Data Requirements for Payer Coverage.”

Determining the data requirements for coverage is becoming an increasingly frustrating issue for diagnostics manufacturers, which face rising demands for evidence but continued lack of clarity about payer standards for evidence-based decision-making, leading many to ask the question, “Why can’t payers just tell us what their standards are?” Complicating the picture is that diagnostics can come to market via different pathways with different levels of supporting evidence (e.g., companion diagnostics reviewed by FDA for clinical validity with the companion therapeutic and tests developed, validated, and introduced to the market by laboratories).

The panelists – who represented leaders from industry, non-profit advocacy, and government working to create solutions for some of these market access barriers – noted a couple of issues at play. One is that having a payer “pick list” or hard criteria for coverage removes the flexibility that is so often needed in these gray-area coverage decisions. The second is that given the volume of products they are evaluating, most payers don’t have the bandwidth to be experts in the nuances of the trial design for every technology. Third is that across all stakeholders, there is a wide range of knowledge on innovative products and personalized medicine and that basic education for the majority of stakeholders to better understand these products is lacking.

Several lessons and next steps came out of this discussion. First, panelists agreed that there must be more education for all stakeholders so that each stakeholder can actually evaluate novel products appropriately, a key finding echoed throughout the day. Second, the emphasis on outcomes must shift from only clinical outcomes to clinical outcomes and quality of life for patients. Finally, all panelists agreed the ideal situation is open, trusting lines of communication and split of the responsibility according to expertise.

At the end of the day, it may be incumbent on the molecular diagnostic community to shape the paradigm for evidence requirements so that payers can act as enablers, rather than watchdogs.

Approaching One-Year Anniversary at FDA, Stephen Spielberg Highlights Agency’s Progress in Personalized Medicine

August 20, 2012

In his address to the Personalized Medicine Coalition (PMC) Policy Committee at our most recent meeting, Stephen Spielberg, M.D., Ph.D., Deputy Commissioner for Medical Products and Tobacco at the U.S. Food and Drug Administration (FDA), announced that the Agency will develop a catalog of personalized medicine-related activities. The catalog, as Dr. Spielberg described, will provide a full accounting of the activities at the agency, including all regulatory divisions and regulatory science.

While reflecting on his 11-month tenure at the agency, Dr. Spielberg also noted that the largest area for advancing personalized medicine was through communication among stakeholders and FDA Centers.  He noted that “PMC is so important because we need dialogue; no one has a lock on complete information.”

The attendees of the policy meeting were pleased to hear that already, less than a year into Dr. Spielberg’s appointment at the agency, he was working to encourage collaboration and communication across divisions.  Dr. Spielberg was previously Director of the Center for Personalized Medicine and Therapeutic Innovation at Children’s Mercy Hospital, one of the founding members of the PMC and so has experience in bringing groups together to advance personalized medicine.

Dr. Spielberg outlined his optimism for personalized medicine at the FDA, noting that one third of the new drug approvals currently in review are for targeted or orphan indications.  He said that by year’s end, we could expect guidance documents on companion diagnostics and co-development, as well as the previously mentioned catalog.

These guidance documents and a FDA catalog may prove to be additional stepping stones to a broader understanding of the impact of personalized medicine on scientific research and clinical medicine.  At PMC, we will continue to work with FDA to promote broader engagement across the ecosystem of stakeholders and greater transparency around research and drug development.  We look forward to the issuance of these materials later this year and will engage a public discourse about them.

Hamburg Highlights FDA Commitment to Personalized Medicine at PMC Annual Luncheon

March 3, 2010

When Dr. Margaret (Peggy) Hamburg delivered the Sixth Annual State of Personalized Medicine Address at the National Press Club on February 25, she impressed and energized the audience by announcing the Agency’s intent to release draft guidance on companion diagnostic regulation by the end of 2010.

As the PMC contends and she noted to the audience, for innovations to occur in personalized medicine, FDA needs to outline clear expectations and standards for approval.

According to Hamburg, FDA intends “to clarify our expectations for the kinds of clinical trials and levels of confidence needed to satisfy us that a test is accurate and that it can be used to help shape clinical judgments.…We also are working internally to make sure we have a common understanding across all centers and throughout the agency about the kind of evidence needed when a test result is being used to shape a drug trial, or drug approval or relabeling.  We also intend to make sure the communication line between sponsors and CDER and CDRH is clear, and that sponsors get consistent advice about how to take the next step in development.

We expect to have this guidance finished by the end of the year.”

She also promised that FDA would be flexible, collaborative, open, and clear throughout this process.   Hamburg concluded her remarks by sharing her enthusiasm and commitment for reducing regulatory barriers at FDA for personalized medicine.

Dr. Hamburg has clearly outlined sound goals – to articulate clear procedures and requirements within and among FDA centers for the approval of personalized medicine products– and a sound way to achieve them –through an open and collaborative process.  PMC members are excited by this commitment and are equally committed to working with her to this end since achieving these goals represents a necessary step in assuring that our regulatory processes are appropriately designed for the science of personalized medicine.

The full text of Dr. Hamburg’s speech is available here: https://ageofpersonalizedmedicine.files.wordpress.com/2010/03/pmc-luncheon_hamburg-speech_02-25-10.pdf.

Latest Developments at FDA Very Promising for Personalized Medicine

February 24, 2010

Today HHS Secretary Kathleen Sebelius, NIH Director Francis Collins, and FDA Commissioner Margaret Hamburg hosted a joint press conference to announce an unprecedented collaboration between FDA and NIH aimed at accelerating the research to regulatory approval timeline for promising new therapies.  To assist in this effort, the initiative has established a joint leadership council that will seek to align biomedical research planning with the most pressing public health issues.  To learn more about the joint initiative, take a look at the press release from FDA or view the videocast of the joint press conference.

The collaboration between these two government agencies has great potential to advance personalized medicine, and as an article published today in MIT Technology Review indicates, advancing personalized medicine through regulatory streamlining is on the agenda for FDA.  According to the article, FDA is seeking to adjust current drug approval processes to better align with personalized medicine.  FDA expects to begin this process by developing guidelines that will enable the side-by-side approval of a drug and the companion diagnostic that will identify which population of patients is most likely to benefit from it.

We are very pleased that Dr. Hamburg, FDA Commissioner, is joining us as the keynote speaker tomorrow at the Sixth Annual State of Personalized Medicine Luncheon.  We look forward to hearing more from her on these exciting developments, and the opportunity they present to introduce a logical approval process for personalized medicine products.


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