Change Happens

You don't have to look far in medical and scientific research today to feel the shifting sands and see signs of change. At times it seems we are simultaneously tilling the soil with a set of old yet tried and true tools while making new ones. Each year, as my organization prepares to bring together leaders in medical research, we confront all of this change head on.

As much as we all like to wring our hands and lament the challenges, the solutions are not always simple but are often elegant. We are supposed to embrace change and seek it out, but it can be a pain. What we have seen is that to reinvent, you have to understand where we are and then find those best practices and case studies that show a path forward, thus easing the pain a bit.

As change unfolds, let us imagine the possibilities:

• Imagine a research culture built on partnerships designed to meet the needs of patients. For years, we have looked at the venture philanthropy sector for models of collaboration -- and now we see more evidence than ever that partnerships between the disease foundations and other sectors in research can and do bear fruit. These groups clearly have an outsized impact. They've catalyzed change and paved a path toward real models for all sectors.



• Imagine if we invested in a strong infrastructure for our essential health systems. This includes the public health, healthcare, and medical innovation systems, so that when a need comes forward, like the current Ebola crisis, we are at the ready to respond with all the systems.



• Imagine if we assured predictable and sustainable funding for the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). Predictability would allow our scientific leaders, our NIH institute directors, and our young investigators to get back to doing what they do best: finding ways to solve even more problems. You don't need to look far to see evidence of the payoff of investing in science. The promise of new immunotherapies in oncology gives concrete hope that diseases like melanoma that hadn't seen new treatments in decades may now see progress. When Debra Black, the founder and chair of the Melanoma Research Alliance, received her melanoma diagnosis, she became a tenacious advocate for change and scientific progress. And it's not just patients who gain hope from these types of breakthroughs. These examples teach us what is possible. Everyone gets a high five when a product gets approved-- scientists, companies, nonprofits and patient groups, regulators, clinicians, and the heroes who participate in research. Imagine the positive things that come with success in one disease: It gives the entire system the stamina and hope to keep pushing on.



• Imagine protecting and nurturing our basic science infrastructure, the seed corn for future discovery. Our university research system is under extreme pressure. There is consensus about the value of this investment, yet we continue to watch resources leave this vulnerable area. We need to snap out of it. There is no question that the university-based research model is under stress, and we need to take a close look at how that model can continue to grow, thrive, and evolve.



• Imagine a system that is patient-centered in word and in deed. We must go past the anecdotes, the sound bites, and the excuses. The process of gathering patient preference and benefit-risk data has demonstrated intense and purposeful cross-sector collaboration. There's tremendous momentum gathering as industry, government, academia, and disease groups are finding ways to create mechanisms to ensure patient perspectives aren't just heard but are seen and used to inform decision making. When I speak about medical research, I often show an image of farm silos. I can still visualize the grain elevator that my grandmother was the accountant for in Michigan. In that case, the silo/elevator served a purpose. It contained the grain from nearby farms and stored it so that it would remain in a pure state for the buyer. Well, in the search for cures, no one goes it alone, and we may want things to be a bit less pure -- is there a purpose to silos in medical research anymore? I think that mindset is being tested and challenged. In fact, in a few weeks at our Partnering for Cures meeting in New York, we look forward to seeing more than 1,000 leaders from across sectors and across diseases and across disciplines do just that.



• Imagine if innovation can come from and thrive in nontraditional places. At a recent Institute of Medicine meeting on the potential for disruption in drug development, John Wilbanks of Sage Bionetworks (and a FasterCures senior fellow) noted that we could see a scenario where scientists bypass traditional infrastructure and set up lab space in their garage or in a centralized facility. Citizen scientists leaping over the silo and into a garage? We've heard tell that it has happened before (see "The HP Garage" on the list of 14 sights you must see in Silicon Valley). Do we want research done in a garage? How much of science can we democratize?

As we imagine these possibilities -- all of which are totally within our grasp -- I want to provide the context we're in. Right now, we are witnessing exactly how our biomedical and healthcare systems are coming to grips with the shifting sands of change. Case in point: What if we cure a disease? Outright cure it. Guess what? With the recent hepatitis C product development pioneered by Gilead, we have seen it this year.

And yet, we are witnessing a public debate about the very cost of pioneering innovation and the reality of introducing something new into a system not built for it. Many groups are working hard to predict what happens when the next cure comes down the pipeline, and hopefully the next after that. And it thrusts groups that were totally comfortable in their silo of medical research smack into the doctor's office and the payer decision tree about what innovation costs and whether we can absorb it given the steady uptick of the cost of healthcare.

There is so much to imagine, to celebrate, to be optimistic about in terms of our readiness for change. But we have a vast to-do list to accept the change and advance it. We must be determined to get it done. While the promise of science has never been hotter, the enormity of human need for solutions to disease is starting to weigh more and more. I recently testified in a roundtable convened by the U.S. House of Representatives Energy and Commerce Committee as part of the 21st Century Cures initiative, and I cited the statistic that there are at least 7,500 rare diseases and only 500 treatments. This statistic illustrates how far we have to go. Chairman Fred Upton and Congresswoman Diana DeGette, who are at the helm of the 21st Century Cures initiative, are busy trying to create a piece of legislation that will break through some of the inefficiencies in today's system and help us achieve faster cures.

Change happens. Will it be enough so that we can save and transform more lives? I sure hope so. I'm so looking forward to watching some of it unfold at Partnering for Cures and beyond.