FDA Issues Draft Guidance for Rx/Dx Co-Development – What’s Next?

FDA released long-awaited draft guidance on its regulation of co-developed drug diagnostic combination products.  PMC asked FDA to issue a draft guidance on this topic since innovators had many questions about how regulation of these two products together would work.  I can describe it in one word: short.

It outlines a collaborative process through FDA where the device and drug centers will coordinate review.  It reiterates the FDA practice of using ‘generic’ descriptors of diagnostic tests in drug labels and offers some flexibility and examples around products where a drug might be ready to go to market but the diagnostic is not yet.

While the community welcomes FDA’s current thinking on the topic, given the brevity of the document, I wonder if it left much unsaid.

For example: The focus (which will probably be welcomed by sponsors) is on collaboration between the drug and device centers but no details are given.  Also, while the personalized medicine community generally agrees that FDA regulation of companion diagnostics should be risk-based, there is a lot of debate in the community about what FDA means by risk in the case of a diagnostic.

In recent months, PMC has been working with over 100 members to develop legislative specifications for the next iteration of the Genomics and Personalized Medicine Act.  I will provide more detail on this in an upcoming blog entry, but one of the key concepts of these specifications is to “incentivize personalized medicine by creating a transparent and predictable regulatory environment for personalized medicine products.”  While the FDA’s draft guidance issued last week certainly leaves me wanting more, I do believe this represents an important step in that direction.

Nevertheless, our efforts toward this goal must continue. The FDA is seeking comment on the document for the next 60 days, and I encourage you to submit feedback.  We also welcome your participation in a discussion here at The Age of Personalized Medicine Blog by submitting a comment below.  We expect that FDA will continue to expand its guidance in the coming months, and receiving input from all stakeholders in the development, regulation, and use of co-developed drug diagnostic combination products is essential to their success.

PCORI: Together Moving Comparative Effectiveness Research and Personalized Medicine Forward

Today, I had the privilege of addressing the Patient-Centered Outcomes Research Institute (PCORI) Board of Governors on behalf of the Personalized Medicine Coalition (PMC).  As noted in my previous blog on its member selection, the PCORI Board of Governors was established by the Patient Protection and Affordable Care Act and is tasked with assisting patients, clinicians, purchasers, and policymakers in making informed, evidence-based, health decisions. 

As a science-driven organization, PMC supports health decisions made on sound science.  When working with legislative drafters and the community to develop the legislation creating PCORI, we recognized the need to ensure that the research supported by this group must be personalized and continually updated.  The resulting law creating PCORI aligned the principles of personalized medicine with those of comparative effectiveness research.  It underscores the importance of eliminating an “average effects” approach to CER, which obscures subgroup differences and skews treatment decision-making away from individualized care.

To make sure this opportunity is realized, we requested that the Board designate an ad hoc committee on personalized medicine to work with both the Board and the Methodology Committee.  We believe that such an ad hoc committee will help the Board and methodology committee comply with the Congressional intent that the research overseen and conducted by PCORI be personalized when appropriate.  High levels of communication and collaboration have been essential to the advancement of personalized medicine, and the same will hold true for advancing CER that is aligned with personalized medicine.

Achieving alignment between CER and personalized medicine holds substantial opportunities, but we realize it will be challenging. These challenges cut across a range of issues, from study designs, to operating procedures, to research priorities.  An ad hoc committee will provide a venue for bringing a diversity of expertise and perspectives together to focus on this common objective.  Such a group will help in coordinating research across a range of public and private sector activities and ensure that the most relevant evidence gaps and research questions are identified related to personalized medicine, and will help with matching study designs to questions.

The science of personalized medicine is fast moving and sometimes highly specialized. Legislation and regulation takes time to catch up. The personalized medicine community is committed to seeing that PCORI’s research keeps pace with scientific advancement and welcomes the opportunity to achieve this end.

We are at a crossroads of research, regulation and legislation. It is an exciting time!

Personalized Cancer Care Requires Effective Diagnostic Tools

On July 8th, the New York Times published a lead story “How Bright Promise in Cancer Testing Fell Apart” that questions the value of genetic testing to determine cancer treatment strategies. I wrote a letter to the editor, which the New York Times published on July 12^th and read in part:

The larger reality is that patients benefit from personalized medicine every day.

Safe and effective targeted therapeutics have reshaped treatment for breast cancer and leukemia, for example; and just last month, reports from the American Society of Clinical Oncology of successful clinical trials for targeted therapies in melanoma and lung cancer are expected to do the same.

In fact, complex genomic tests have been on the market for years. A survey by the Tufts Center for the Study of Drug Development found that 94 percent of biopharmaceutical companies are investing in personalized medicine and that 100 percent are using biomarkers to learn more about compounds during the discovery phase of their research.

If it were not for the opportunities this field offers to improve outcomes by matching the right treatments to the right patients, we would not see industry or the federal government making these investments in an effort to advance the frontiers of medicine.

Clearly, as the story reports, some trials will fail and unfortunately, even a single case of fraud is too many.  Nevertheless, to leap to the conclusion (and print it on the front page of the New York Times) that cancer testing is in danger of “falling apart” or to suggest that we are locked into a one-size-fits-all paradigm overlooks current and future capacity to improve diagnosis and target treatment.  We should not lose sight of the value personalized medicine provides to patients while maintaining our commitment to scientifically sound research that supports molecular medicine.

No Perfect Solutions for Innovators Facing Reimbursement Challenges

PMC organized a panel discussion of experts to discuss the coverage, coding and reimbursement challenges facing innovators of personalized medicine diagnostic tests at the Biotechnology Industry Organization’s International Convention last week.

The panel built on a PMC-commissioned study released last December written by David Parker, Vice President, Boston Healthcare Associates.  He discussed the multi-faceted nature of diagnostics reimbursement and how the status quo reduces patient access to innovative tests.

The panel also included Kristin Pothier, Partner, Health Advances, and author of a study for BIO looking at the impact and feasibility of possible reforms to the reimbursement system and ultimately concluding that there is no perfect solution but that a combination approach to reform is needed to improve patient access.

Offering their perspectives on challenges facing diagnostics innovators were Steve Phillips, Director, Health Policy, Johnson & Johnson and Scott Allocco, President, Biomarker Strategies, co-chairs of PMC’s reimbursement task force.  Phillips agreed that a comprehensive approach to reforming reimbursement is needed for diagnostics and was comfortable working within the current environment.  Allocco suggested that a more radical change is needed to ensure that investors receive adequate returns on their investment. 

As the panelists discussed, pursuing legislative and regulatory solutions to coverage, coding and reimbursement problems is important to moving personalized medicine forward. But PMC is also seeking opportunities to solve problems by engaging with policymakers outside of government. For example, several panelists described how the American Medical Association’s Current Procedural Terminology (CPT®) coding system hampers reimbursement of molecular diagnostics and shared their concerns that the proposed additions of new molecular diagnostics codes are not specific enough to ensure that tests reimbursed at full value.  Although a new set of codes will be rolled out in 2012, I encourage those interested in this issue to join me at the AMA’s Molecular Pathology Workgroup meeting in Chicago on July 20.

One audience participant asked whether the U.S. can draw from best practices in coding, coverage, evidence and reimbursement from other countries.  PMC will be examining this topic in the coming months and will release a paper looking at challenges diagnostics companies face in the European Union this fall.  As long as personalized medicine is disadvantaged by the current system, PMC will advocate for changes to it.

Reinventing Business Models to Get Us Around the Bend in Personalized Medicine

In recent years the scientific community has taken great strides in reaching a new understanding of genomics and the molecular underpinnings of diseases.  As a result, we’ve reached a turning point in personalized medicine: for the first time, society is finally in a position to start bringing personalized medicine from the lab to the clinic on a large scale.

Johnson & Johnson is committed to helping “be the change” in bringing personalized medicine from the lab to the clinic, with the goal of helping guide delivery of our medicines to the right patients at the right doses at the right times.  Equipped with our new understanding of personalized medicine, we’re changing the way we approach all aspects of drug development programs.  Pharmacogenetic, genomic and imaging data are now guiding our actions related to nearly every therapeutic we develop, and we’ve modified our companion diagnostics programs to identify clinically relevant biomarkers and diagnostic tools earlier in drug development programs.

To bring personalized medicine forward, scientists, regulators, policymakers, physicians, the patient community, payors, and pharmaceutical and diagnostics companies must work in concert.  We must find ways to more successfully work together, adopt a new mindset, and pursue a more open, “networked” approach to innovation.  I’m thrilled to be leading a team focused on this effort at Johnson & Johnson.

But this effort—the striving for open, collaborative, multi-disciplinary innovation—by its very nature is bigger than just one company.  The challenge before us—before society, really—is to take the science, the efforts of industry and the complexity of our health delivery systems, and pave a clear path forward for fully realizing the promise of personalized medicine. Research and development of personalized medicines and companion diagnostics requires equal or sometimes larger investment than traditional drug development programs—even as target populations are, by definition, becoming narrower.  To ensure we’re able to fully tap into the promise and potential of personalized medicine, we must work together to reinvent current business models in ways that incentivize the further pursuit and use of personalized medicine.

Personalized medicine pushes us to look at disease from many different angles, to persevere through complexity, and to tailor healthcare solutions to the unique needs of individual patients or subsets of patients.  In doing so, we can improve health outcomes and enhance quality of life. Even with the challenges ahead of us, these are very exciting times.  Because along with the challenges – the horizon is full of possibilities.