National Bioeconomy Blueprint Showcases Personalized Medicine as Model for Strengthening U.S. Bioeconomy

Last week, the White House released its National Bioeconomy Blueprint.  It lays out some strategic objectives designed to help realize the full potential of the U.S. biotechnology sector to generate economic growth by creating jobs and addressing societal needs.

As an example of how the government’s efforts can facilitate the development of a more robust bioeconomy, the report discusses the impact of the Human Genome Project and the development of personalized medicine. The Blueprint cites the Case for Personalized Medicine, 3^rd Edition, noting “advances in recent technologies have increased the momentum of personalized medicine – customized healthcare based on specific genetic or other information of an individual patient.”

While we agree that policies are needed to help support research and development (R&D), improve translational and regulatory science, improve regulation in other areas, enhance workforce training, and develop new public-private partnerships and precompetitive collaborations, we are concerned they are not sufficient to allow us to realize Adriana Jenkins’ dying wish, that all patients have access to personalized treatments.

The White House is correct to shine a light on FDA and direct the agency to focus attention on application review times, coordinated parallel reviews of products, and continued improvement of regulatory science. In reality, we are already seeing the benefits of this increased coordination – with FDA’s accelerated review and approval of Kalydeco™ for cystic fibrosis and Xalkori^® for non-small cell lung cancer, each with a companion diagnostic approved together and in advance of FDA time lines.

Still, engaging on regulatory science and streamlining FDA processes will only go so far to improve the bioeconomy and bring personalized medicine to patients. For instance, current comparative effectiveness research (CER) and health technology assessment (HTA) models, which are not addressed in the Blueprint, are not well aligned with the science of personalized medicine and such misalignment causes additional barriers to market entry and patient access after FDA approval. The Blueprint highlights coverage with evidence development (CED) as a potential HTA model, but it should be noted that although CED can be a good tool, if done wrong, it can also chill innovation.

Likewise, unclear and unrealistic expectations for obtaining Medicare coverage and adequate payment for personalized medicine products and services are a substantial hurdle, preventing companies from seizing the full scientific potential and translating that into new treatments and the resulting high-paying jobs and economic contributions that follow.

We look forward to working with the Administration and with Congress to shape policies that will help support the ability of companies to continue to develop personalized medicines and bring them to patients, including research and development tax credits, delivery system reforms, and regulatory and reimbursement policies. Given the tremendous potential of personalized medicines, it’s key that we get the policies right to foster companies working on personalized medicine and thereby improve patients’ lives and our economy.

The Genomics and Personalized Medicine Act Needs a “Fresh Look”

“Now is the time for a national effort to move personalized medicine forward,” said Representative Anna Eshoo at the Center for the Study of the Presidency and Congress and Health Affairs’ Capitol Hill briefing held last week, on the same day that the Food and Drug Administration (FDA) released its plan Driving Biomedical Innovation:  Initiatives for Improving Products for Patients.

At the briefing, Congresswoman Eshoo affirmed the Personalized Medicine Coalition’s long-held contention that the 2010 version of the Genomics and Personalized Medicine Act needed an infusion of new ideas.  (See my blog post from August 23, 2011.)

Also speaking to the audience of congressional staff, reporters, patient advocates industry representatives, and others, were several government officials tasked with enforcing policies that affect personalized medicine innovation.  They presented their thoughts on the state of personalized medicine and their respective agencies’ roles, highlighting their progress. Both Janet Woodcock, M.D. and Elizabeth Mansfield, Ph.D. of FDA highlighted FDA’s efforts in bringing regulators and industry to the table to discuss the challenges of regulating personalized medicine products and services.  These discussions informed the agency’s new report—dubbed a blueprint for innovation—which includes major focus areas in building the infrastructure to drive and support personalized medicine and in creating a rapid drug development pathway for important targeted therapies.  They also acknowledged that changes are still needed at FDA to accommodate personalized medicine and that the agency will continue its leadership role by building a system to support the development of personalized medicines, including investments in regulatory science and by clarifying agency policies.

At the same time, Jeffrey Roche, M.D., M.P.H. from the Centers for Medicare and Medicaid services (CMS), underscored his agency’s time-consuming coverage decision-making process, which is based on published research used to determine what is “reasonable and necessary.”

Indeed more coordination is needed at the federal level.  Speakers representing academe, industry, and patients reinforced this sentiment, in addition to emphasizing the need for industry incentives to develop new products and services and a CMS reimbursement policy that can speed the adoption of personalized medicine by paying enough for personalized medicine diagnostic tests so that research and development costs are recouped.

Patient advocate and PMC-member Donna Cryer summed it up best, when sharing her thoughts on what can foster innovation and the adoption of personalized medicine: We need HHS level coordination, and a streamlined FDA process for personalized medicines and their diagnostic partners.  CMS needs to support the science of personalized medicine through a system of care that repays innovators for the research that goes into the development of these products.  And tax credits are necessary to help companies through what is still very difficult science. 

PMC is pleased by Rep. Eshoo’s leadership in advancing personalized medicine as evidenced by her participation in this event and her willingness to consider all of the policy proposals PMC and others have put forth.  We look forward to working with her to vet policy recommendations suggested by others and to see the Genomics and Personalized Medicine Act introduced in this Congress.

Looking Forward, Looking Back

At the Food and Drug Administration (FDA), I had a wonderful opportunity to spend nearly 20 years working in various capacities: in drug development science, regulatory oversight, and clinical pharmacology. Earlier this summer, I had the privilege to meet with the Personalized Medicine Coalition’s public policy committee to discuss the state of personalized medicine just after my departure from the FDA and before I began my new position leading the pharmacometrics and systems pharmacology initiative in the interdisciplinary Institute of Therapeutic Innovation at the University of Florida’s Research and Academic Center in Lake Nona (Orlando).  Personalized medicine will continue to be a passion for me in my new position.

The timing of my talk was serendipitous:  Dr. Steven Spielberg was just named as the new Deputy Commissioner for Medical Products and Tobacco at FDA. His perspective is strongly rooted in understanding disease at the molecular level, having served as director of the Center for Personalized Medicine and Therapeutic Innovation at Children’s Mercy Hospital in Kansas City, Missouri. And the FDA had just announced the draft guidance document for personalized medicine and companion diagnostics. This guidance should be seen as part of a mosaic of guidance documents from the FDA that will together guide the regulation of personalized medicine products. The Personalized Medicine Coalition has begun putting the mosaic together by listing the relevant guidance documents as a reference tool on its website.

I had a chance to reflect on the new direction FDA has taken over the past several years to embrace what we’re seeing in research and scientific discovery. Like the industrial revolution, the genomic revolution could take 60 years to fully unfold, but in the last ten years, the field and the FDA have made great progress. Many of these changes are leading researchers to re-evaluate how we focus research efforts, assess new therapeutics, and apply our shared learning through the R&D continuum.  As we’ve heard from the industry, researchers are stratifying medicines earlier in the R&D process and about 50% of new drugs have a biomarker that can potentially lead to a personalized approach. While many challenges remain, I am encouraged by the four-fold increase in Investigational New Drugs (INDs) and New Drug Applications (NDAs) that include genomic data since 2008.

To address this growth in genomic submissions, the FDA has new employees with expertise in genomics dedicated to review of these products and efforts to coordinate personalized medicine reviews will continue. I see a new era of personalized medicine not just in oncology but in cardiovascular disease, infectious disease, and neurodegenerative diseases, among others and the FDA is working to be ready.

I called on all members of the Personalized Medicine Coalition to find new ways to enable collaboration and to export the knowledge they are fostering at their companies and organizations to benefits for public health. By doing so, our combined efforts can lead to dramatic advances in improving therapeutics and healthcare. We are only just beginning to understand how new networks and powerful tools for sharing best practices can help us solve some of our most complex disease and drug response questions.

Legislative Specifications Would Create a Path Forward for Personalized Medicine

The Personalized Medicine Coalition has a long history of working with Congress on the Genomics and Personalized Medicine Act (GPMA); a bipartisan bill first introduced by Senators Obama and Burr in 2007.  The original bill was designed to take full advantage of our federal investment in the Human Genome Project by putting policies in place to enable personalized medicine.  Subsequent versions of the bill have not gone far enough to realign the health care system with personalized medicine.  In an effort to see a more effective version of GPMA introduced in this Congress – one that would move personalized medicine forward and would enjoy community support — PMC members developed a set of legislative specifications for Congressional consideration that are complementary to previous versions of GPMA.  We’ve asked Congresswoman Anna Eshoo to consider including them in her new version of the bill.

These recommendations include a range of important regulatory and policy concepts that can have a meaningful impact on advancing the discovery of and access to personalized medicine products.  They also echo important themes that emerged at the PMC’s Capitol Hill briefing in May.

Specifically, we propose codifying the Department of Health and Human Services’ Personalized Healthcare Initiative and providing the Secretary with an advisory committee populated by experts inside and outside of the government. We also reiterate the importance of ensuring adequate personalized medicine expertise and input on various federal advisory committees and within policy proposals.

Sponsors of personalized medicine products have had challenges taking drug-diagnostic combination products through the Food and Drug Administration (FDA). These experiences suggest that innovators need a predictable regulatory path at FDA so we propose an office for personalized medicine to coordinate therapeutic and diagnostic review, and enforce review timelines for both.

While the path from regulatory approval at FDA to adequate coverage and payment is clear (and usually predictable) for therapeutics, adequate coverage and payment for diagnostics can be long, complicated and frustrating.  To ensure that patients have access to the latest personalized medicines and diagnostics, we suggest policies at the Centers for Medicare and Medicaid Services that will ensure timely coverage and reimbursement for them.

Finally, to ease business model barriers, and incentivize the types of relationships that are the hallmark of personalized medicine, we suggest extending the qualifying therapeutic discovery tax credit from health care reform and expanding it to include personalized medicine companies.  We also suggest a new research and development tax credit to entities developing personalized medicine products.

We look forward to working with members of Congress in both chambers and in both parties to see that the Genomic and Personalized Medicine Act is introduced and signed into law.

The details of the legislative specifications can be found here.

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.

Fall Forecast: Personalized Medicine Policy and Events

Labor Day has come and gone, Congress is preparing to go back in session, and shortening days remind us that Daylight savings will be here before we know it. Even as the long days of summer draw to a close, the fall promises to be a busy and productive time in advancing policies to support personalized medicine, as well as convening industry stakeholders to connect, share ideas, and collaborate toward advancing the science, business, and policy of personalized medicine.

This fall we expect to see a maturing of activities started over the summer.  FDA hosted a 2-day meeting in July on the regulation of laboratory-developed tests (LDTs).  The meeting docket (FDA-2010-N-0274) provides a good overview to any who missed the event.  After that meeting, the House of Representatives held a hearing on consumer genomics.  This hearing made it clear that Congress is not satisfied with how gene scans are regulated, and sometimes that dissatisfaction was aimed at LDT regulation in general. The Senate is also concerned; GenomeWeb reported that Senator Hatch (R-UT) is considering legislation on the subject. 

In other legislative news, Rep. Patrick Kennedy (D-1^st RI) partnered with Rep. Anna Eshoo (D-14^th CA) to introduce an updated version of the Genomics and Personalized Medicine Act (HR 5440). PMC will continue our work to strengthen this bill through inclusions of research  incentives for personalized medicine and other improvements in the Senate version as we look for it to be re-introduced  in the next Congress. 

Next week, I will be joining Carolyn Clancy, Francis Collins, and  many other leading experts at the 2nd National Comparative Effectiveness Summit which will feature presentations and discussion on the legislative history and future of comparative effectiveness research (CER), how federal stimulus dollars for CER are being put to use, as well as the intersection between CER and personalized medicine. The focus of this meeting is particularly timely as the expected announcement of the federally-appointed Board of Governors for the Patient-Centered Outcomes Research Institute (PCORI) is expected later this month. The discussion on balancing CER and personalized medicine objectives will continue in October at the Comparative Effectiveness and Personalized Medicine:  An Essential Interface meeting hosted by the ECRI Institute, Agency for Healthcare Research and Quality, and the National Institutes of Health.

There are a number of other meetings taking place throughout the fall focused on a variety of topics and issues relevant to personalized medicine, beginning with the 6th Annual Burrill Personalized Medicine Meeting on September 27 & 28. Hosted by Burrill & Co and the Duke Institute for Genome Sciences & Policy, the meeting will focus on the impact of healthcare reform on personalized medicine. On October 14 & 15, the 3rd Annual Personalized Health Care National Conference is taking place at Ohio State University Medical Center’s Center for Personalized Health Care. This conference will include discussion of the challenges of making predictive, preventive, personalized, and participatory medicine a reality.

The conference season will culminate on November 17 & 18 with the 6th Annual Personalized Medicine Conference. Each year, the Partners HealthCare Center for Personalized Genetic Medicine, Harvard Medical School, and Harvard Business School bring together representatives from academia, diagnostic and pharmaceutical companies, healthcare providers, policymakers, and other industry experts making contributions to advancing personalized medicine. This year we’ll explore the ways personalized medicine impacts clinical care, and how lessons from these experiences can inform policies and plans to implement personalized medicine to improve patient care.

For more information on these and other conferences of importance to personalized medicine, please visit the Events page at The Age of Personalized Medicine website. And throughout the fall, stay tuned to The Age of Personalized Medicine Blog as an opportunity to engage in discussions stemming from these events and new policy developments.

The Genomics and Personalized Medicine Act

This summer marked the 10^th anniversary of the sequencing of the human genome.  In the course of these ten years, advances in genomic technologies have begun to bring forth the promise of personalized medicine to the clinic.  And as this clinical utility continues to expand, our national legislative priorities will also need to reflect this change in medical practice and create regulatory policies that support creation and adoption of targeted therapeutics and personalized medical programs.

The coming of August starts to signal the close of summer, and with it, the August recess for Congress when our representatives head back to their districts to spend time with their constituencies. However, with the mid-term elections only months away, this August recess also signals a slowdown of legislative efforts and a focus on campaigns. Among the unfinished business that will remain after the 111^th Congress closes and the 112^th is elected is HR 5440, the Genomics and Personalized Medicine Act of 2010.

The Genomics and Personalized Medicine Act of 2010 is an effort to accelerate research, coordinate federal activities across agencies, and ensure delivery of personalized medicine to the American public.  The bill would help guide this new age of discovery and medicine in the second decade beyond the sequencing of the genome.

Built upon then Senator Obama’s Genomics and Personalized Medicine Act of 2006, HR 5440 attempts to expand the provisions of the 2006 version by mandating the creation of the Office of Personalized Healthcare under the Secretary of Health and Human Services.  This office will be the point for policy coordination regarding personalized medicine strategic planning, education, coverage and reimbursement, and product regulation.  The bill will provide $150 million in funding for personalized medicine research grants linking genetic profiles with individuals’ biospecimens.  The bill would also strengthen the FDA’s regulatory authority in this area, clarifying requirements for diagnostic tests and potentially allowing FDA to require companion diagnostic tests for new drugs entering the market.

Currently the bill has received little notice in Congress, having only six cosponsors, despite it being a high priority of then Senator Obama in 2006.  In addition, this bill will face another hurdle in moving forward, as it was introduced by Rep. Patrick Kennedy who will not seek reelection this year.  In the next Congress a legislative champion will need to be identified to introduce the bill, much less generate momentum to move the bill forward.

Legislation moves slowly – the science of personalized medicine, however, does not.  If this past decade has shown us anything, it is that genomic technologies are rapidly moving towards clinical applications.  Establishing the appropriate legislative and regulatory frameworks to fully capitalize on the potential of our scientific advances can contribute greatly to the overall healthcare goals of our nation.  Researchers, clinicians, and advocates will all need to play a role in educating our legislators and policymakers of the importance of good policy that supports personalized medicine and disruptive innovations. Strong encouragement to reintroduce legislation supporting personalized medicine policy will need to be emphasized by all parties involved in improving health.