In Conversation with Edward Abrahams, President, the Personalized Medicine Coalition

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Edward Abrahams, Ph.D., has been president of the Personalized Medicine Coalition (PMC) since its founding in late 2004. After serving as an anchor partner for the Annual Personalized Medicine Conference in collaboration with Harvard Medical School from 2005 to 2015, PMC took over the programming and management of the conference in 2016. This year, the Coalition will celebrate the 15thAnnual Personalized Medicine Conference programmed under the theme “The Paradigm Evolves.” Abrahams recently took time out of his busy schedule to talk with Clinical OMICs’ Editor in Chief Chris Anderson about how personalized medicine has evolved since the early days of the conference.

Clinical OMICs: Talk about PMC as an organization, and about how the first conference came about.

Edward Abrahams, Ph.D.: Following the mapping of the human genome, which we can date to 2003, a number of institutions came together and recognized that personalized medicine wasn’t going to happen just because the science pointed us in that direction. Rather, they understood the importance of the intervening variables between the science and the patient. Things like regulations, reimbursement, genetic privacy, education, clinical adoption, etc., all have to be considered if progress is going to be accelerated. When PMC was launched back in 2004, that was the essential goal — to lead us toward a time, as we said back then, when the right patient would get the right drug at the right time.

Can you recall some of the highlights of those first couple of conferences?

The first conference was titled “Personalized Medicine: Promises and Prospects.” That was in 2005. In 2005, far-sighted people were looking ahead and asking: “What can we do with this new resource, these new understandings, these new technologies? How will they change medicine in the future?” We were, back then, talking about the promise of personalized medicine and what it might look like in 2020. It has been a very interesting evolution.

You would need to be a visionary back then to talk about the promise of personalized medicine, since the cost to sequence a genome was in the tens of millions of dollars.

That is exactly right. There were very few drugs on the market that had biomarker strategies within their labels. I think we counted no more than five percent of the new molecular entities approved by FDA were personalized in 2005. If you look at this year’s Personalized Medicine at FDA: A Progress and Outlook Report, which PMC published in January, you see that 42 percent of all drug approvals in 2018 had biomarker strategies in their labels. That is a proxy for the enormous progress made since 2005.

How has the conference changed since it was first launched in 2005?

I recently took a look at our past programs, and I was struck by the fact that we are still facing many of the same challenges, in that we don’t have a political system that is four-square behind integrating personalized medicine into healthcare. In part, this is because there is anxiety about its costs, and therefore about its accessibility. If anything, this has only increased in the current environment.

So, we still have these public policy challenges, never mind the challenge of educating providers, some of whom may be slow to adopt new ways of doing things. Those are issues that PMC still focuses on.

What is different is we are investing more in developing an evidence base that will convince payers and providers to embrace this paradigm change. I would argue that this is nothing less than a change in the way we practice medicine, because we are proposing to move away from the population averages upon which medicine has been based since the dawn of modern medicine, to a system that appreciates individual variation and treats it accordingly. That’s a big leap.

What do you see as the mission of the conference itself and of PMC, in a world where sea change is occurring?

Well, it actually feels more like an evolution than a revolution when you are in the middle of it. We have seen, and continue to see, incremental progress. We need greater evidence that this approach works not only for patients, but also for health systems. We are trying to provide that evidence. This remains challenging, especially without strong advocates in the administration.

I miss the rhetorical focus the Obama administration placed on the power of personalized medicine to improve healthcare, though I will also note that it focused most of its effort on research and not on implementation. But in the Trump administration, there is an overriding focus on how to cut costs, and not on whether we can integrate innovation to solve medical problems and make the health system more efficient.

Part of the conference this year is designed to examine the issues that have been teed up around cost and accessibility, and to figure out what we can and should do to make sure that the progress we have seen most recently in cell and gene therapy — never mind artificial intelligence — is not arrested in its development. That will be a big question at the conference: are we ready for cell and gene therapy?

We are very fortunate to have Carl June in a facilitated discussion with Stephen Eck, who is PMC’s chairman and is involved in immunotherapy research on his own. We also have Scott Gottlieb, whose leadership helped put FDA four-square behind personalized medicine.

You mentioned Scott Gottlieb. What role can the FDA play to help advance personalized medicine, and how would you rate their recent work in this area?

I think the message has gone out quite clearly in the last five years or so, even predating Scott Gottlieb, that the FDA wants to see evidence of efficacy and safety not just for population averages but for particular patients. The pharmaceutical industry has gotten that message, especially in oncology. The industry understands that if you want to get a drug approved, it better have a biomarker strategy and it better address unmet medical needs. The industry has responded very well to that.

The challenge the industry faces, however, is to develop the business model to make sure it works. If you are developing drugs for smaller populations, it is likely that the price of those drugs will increase, though the efficiency of the healthcare system should also increase. I don’t think that message has come across as well as it should.

The FDA has indeed been the catalyst that has led to this evolution in development of personalized medicines. The 21stCentury Cures Actwas very important in giving FDA the platform to push its agenda forward.

What do you consider the most significant advances since the founding of PMC in 2004?

One is the declining cost of genomic sequencing. We haven’t yet seen the phenomenal ramifications of this development, but I have no doubt that we will.

What do you think those ramifications will be?

I think the ability to collect enormous amounts of data at relatively low cost could and should inform thinking, and then practice.

Also, next generation sequencing provides the ability to use gene panels to figure out how individuals are different from one another in order to, for example, prescribe the right treatment.

A third breakthrough is the willingness to consider real-world evidence in changing the way medicine is practiced in the future and in controlled randomized clinical trials, which are inevitably slow.

And a fourth is the re-emergence of gene therapy and the emergence of cell therapy as potential curative treatments for unmet medical needs. These are potentially phenomenal breakthroughs that we have seen just in the past two years.

How important are some of the larger integrated health systems like Geisinger, Intermountain, and others in their focus on providing personalized medicine?

They are critically important. Some institutions, however, overstate their capacity at times. We are presently doing a landscape analysis that is trying to evaluate how well personalized medicine is integrated into what is offered to patients. What we are finding — and will be reporting at our November conference — is that this is uneven.

Is it because for some it is still early days?

It’s because some are more committed in rhetoric than in reality.

What are the challenges to broader adoption?

Well, ensuring that the latest advances in science and technology are actually integrated into what patients actually get. That is dependent on education, a willingness to change, and a willingness to incorporate real-world evidence to what one offers patients. That may require changing guidelines, etc.

It also requires getting the multiple stakeholders in healthcare to work together to share value. And this is one of our big themes: to share value so that change becomes a win-win situation and not a zero-sum game, as it is too often presented today.

Are you talking about getting providers, payers, pharmaceutical companies, and diagnostics companies to share data?

Sharing data is one thing, but it’s also getting payers, providers, and manufacturers to work together to make sure that innovation benefits everyone. It can’t just benefit the manufacturer. It also needs to benefit the payer, who has to see lower downstream costs, and the patient, who stands to benefit from improved care. That requires an integrated system that we don’t now have.

The year’s conference theme is “The Paradigm Evolves.” Why was that chosen and how does the programming reflect that?

The programming reflects the fact that the paradigm has evolved. Fifteen years ago, we were talking about the promise and prospects. Today, we are changing the way medicine is practiced and focusing on what we need to do to accelerate this process. We are moving toward personalized medicine, albeit, I would argue, not as fast as patients would like.

As you look at the challenges in the landscape, what is PMC’s role to help mitigate these challenges and bring the stakeholders together?

Essentially, we need to explain what personalized medicine is in order to get the stakeholders to work more closely together around the concept of sharing value to improve care. That is number one.

Number two, we have to convince the political system not to put obstacles in the way and to encourage linking therapy to diagnostics. Right now, that is challenging because people don’t want to pay for something they don’t understand.

And we have to develop the evidence base to encourage clinical adoption. Providers and payers aren’t going to move off the dime unless they see a better future.

When it comes to collecting that evidence, where does the burden lie?

I think it lies with everybody, don’t you?

PMC is a relatively small nonprofit, but we are developing the evidence base. Our work has shown, for example, that there is a practice gap in using next-generation sequencing in non-small cell lung cancer; that the value of next-generation sequencing would be higher if physicians put in place actionable opportunities where they could. We know from our analysis that patients aren’t always getting the therapies that the sequencing suggests they should get.

We also have another project to demonstrate the value of next-generation sequencing for patients with suspected rare diseases.

Would you say that cancer care is the most advanced in providing personalized medicine?

Yes, and I say that with some regret because I think the implications of personalized medicine apply outside of cancer and genetic diseases.

Do you think that diseases like diabetes, heart disease, and metabolic diseases will see significant attention for personalized medicine in the future?

Those are the big areas where we would like to see progress. And I would add one to that list. The opioid crisis is one that begs for an understanding of who is most likely to get addicted to opioids. At the conference, we will hear about research efforts at the Coriell Institute, which is working to help answer that question.

FDA has been more active recently in trying to regulate pharmacogenetic testing, and many in the industry believe it is overreach. What is PMC’s position on this?

We are working with FDA directly to better understand its concerns and establish a path forward. We recognize that there are likely problems out there, in that claims are sometimes being made that may or may not be supported by evidence. At the same time, the question of whose evidence and what evidence is going to be acceptable is ambiguous at the moment, causing enormous confusion in the field. This will also be a subject of conversation at the conference.

Looking into the future, where will we be in 2025 with personalized medicine?

Of course, it is difficult to make predictions.

What I am sure of is that we will continue to make incremental progress. My hope is that the public will get excited about the opportunities that we see not on the horizon, but in the present.

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