PAN Advocacy For Translational Research: An Increased Focus for NIH

In the scientific community, basic research uncovers basic scientific discoveries, but translational research is what translates those discoveries into therapies, medicines, and, hopefully, cures.  While both are critical in biomedical research, getting drugs to market more quickly – and not letting important discoveries languish and disappear into science’s “Valley of Death” – has long been a focus of the Parkinson’s Action Network (PAN), the unified voice of the Parkinson’s community.

In early 2008, after many months of research, data gathering, and conversations with the PAN Board of Directors, PAN CEO Amy Comstock Rick and then-Deputy CEO Mary Richards decided it was time to take on the issue of improving the federal approach to translational research.

“We decided if we're not a part of the solution, we're part of the problem, so let's try and fix this,” said PAN CEO Amy Comstock Rick.  “Until then, we'd never forced ourselves to say:  if we wanted to do something different, what would this solution look like?  What would work?”

Over the next few months they hunkered down, crafting various ideas for how to expand how the National Institutes of Health (NIH) focuses on translational research.  Using their knowledge of how the NIH works, they tried to answer the questions:  What should a focused approach to translational research look like at NIH?  How can we accomplish this legislatively?  Should there be a separate agency?  Who else can we work with to get this done?  At the time, Senator Joe Lieberman (I-CT) had introduced legislation on the Hill to create a separate translational research agency outside the Department of Health and Human Services (HHS).  PAN liked the overall goal, but thought that separating a new agency from HHS (where NIH and the Food and Drug Administration are located) would ultimately undermine coordination.

Rather, Rick and Richards came to the conclusion that centralizing translational research from across the NIH into one new Institute or Center was a really good solution, and one that might actually work.

After first sharing their proposal with the heads of the other national Parkinson’s organizations, PAN’s leadership began talking with trusted colleagues and a few like-minded scientific researchers.  Soon, others began hearing about PAN’s initial legwork on and foray into this issue and contacted the organization with their ideas.  With that, PAN became the advocacy organization to lead the effort to put a new plan on the table.

“As can often be the case, people were more than willing to point out all the flaws in our plan, or tell us the many ways in which the government would never change so how could this possibly work, and we would just say, ‘so how would you address this issue’,” said Rick. 

PAN also knew approaching this issue from just one disease standpoint wasn’t powerful enough, so it encouraged other disease groups and broader biomedical research groups like Faster Cures to get the broader community involved and singing from the same proverbial song sheet.  PAN didn’t feel the need to form an official coalition or separate organization.  It was important to just get all the right people involved and get the work done.

By February 2009, PAN had created a comprehensive, detailed proposal of a separate NIH translational institute.  Even though it was a budget-neutral proposal, it was not met with great acclaim.  Some said it created yet another government silo.  Others said it would never happen – that NIH couldn’t and wouldn’t change. 

“One of the most basic tenets of advocacy is that what you’re asking people to support has to be able to fly,” said Rick.  “If you can't even convince people that this is a good thing, then it's not going to work.  The goal is not to have the most intellectually perfect plan; the goal is to get something done.”

The plan for a separate institute at NIH was shelved for the time being, and PAN went back to the drawing board to create a new proposal to address the need for NIH to be more of a leader in translational research.

When Dr. Francis Collins was appointed NIH Director in the summer of 2009, PAN saw a noticeable shift.  Collins knew and understood the importance of bolstering translational research and began to advocate from within NIH for change in this arena.  PAN continued to work on the issue, speaking at conferences, raising awareness and educating at every turn, and was pleased that it became one of Dr. Collins’ key goals for his term at NIH.  In January 2010, Collins published an article outlining his five goals as NIH Director, one of which was strengthening and supporting translational research.

Then-Senator Arlen Specter (D-PA) had done his own work in this area and had introduced legislation similar to Senator Lieberman's, proposing a separate agency outside NIH and HHS.  PAN reached out to his office to let his staff know that its research showed that, in PAN’s view, this effort needed to be done within HHS.  PAN proposed shifting the Cures Acceleration Network (CAN) so that it would be part of NIH and be comprised of representation in both the public and private sector.  This, PAN believed, would help coordinate the efforts of all stakeholders in the drug development process – including patient advocacy organizations, the Food and Drug Administration, and other agencies outside of NIH – to more quickly move discoveries from the lab into the next generation of therapies. 

Senator Specter needed a vehicle to get CAN through Congress.  Using his significant seniority in Congress and his great commitment to the importance of translational research and NIH, he was able to have CAN inserted into the recently introduced health care reform package, which ultimately passed and was signed into law as the Affordable Care Act in March 2010.  During the monumental health care reform debate, ensuring CAN remained part of the legislation was important to PAN and the other organizations working on this issue.  

Once CAN was authorized in the Affordable Care Act, it needed to be funded.  PAN organized a number of sign-on letters with other disease organizations, industry, venture capital associations, and other organizations because the broad base of support for this kind of change extended way past just the patient advocacy groups.   And still, there were many people saying this plan was going down the wrong path.  They said this kind of translational focus isn't what NIH does, and that NIH can't change.  

“We continued to say that we believed NIH had to and could change, and that we had to try something new because the status quo wasn’t working,” said Rick.  “We knew that because we had support from inside the NIH and on Capitol Hill, we had to keep pushing it and bringing in the right people at the right times and continue the momentum we’d already begun.”

All along, PAN had been educating its grassroots advocates about translational research and the importance of shifting the way it was organized within NIH.  So, when it was time for them to act, advocates already knew why this was so critical to moving drugs and therapies through the pipeline faster.   Though it wasn’t until the push for federal funding that PAN unleashed tens of thousands of advocates to call and email their elected officials, in the meantime PAN’s grassroots leaders set the stage by educating their Senators and Representatives about the need for increased NIH focus on translational research.

As a next step, Dr. Collins tasked the NIH Scientific Management Review Board with looking at how to best increase NIH’s focus on translational research.  After a number of meetings and much deliberation, in December 2010 it came back with the recommendation to create a separate institute within NIH.   The NIH followed through with the recommendation by proposing to Congress the establishment of the National Center for Advancing Translational Sciences (NCATS). 

PAN once again got to work leading joint letters with other organizations, while its extensive grassroots advocate network began calling and emailing to fight for funding for both CAN and NCATS.  Ultimately, NCATS was given $575 million for fiscal year (FY) 2012, from which CAN got $10 million in startup money, which allows the CAN Board to begin its work.

One significant component of the CAN legislation was the creation of its oversight Board.  The CAN Board has industry representation, venture capital, biotech, and patient advocates – all of whom will ensure this new direction will stay on course and continue to grow.  Despite earlier concerns that the CAN Board wasn’t being viewed as significant, PAN is pleased it now appears that the CAN Board will become the national advisory council for NCATS.  This means a broad range of people with expertise in translational research will have oversight responsibility for NCATS, which makes sense since translational research is often best done through private-public collaboration.  CAN has flexible-spending authority and can partner with non-governmental entities and create public-private partnerships.  Even though $10 million is not a lot in terms of the broad scope of work that needs to be done, with flexible-spending authority PAN hopes the CAN Board can show early on how valuable they are and extend that authority to the rest of NCATS.

So what’s next for NCATS and CAN?  NIH is in the process of searching for the Director of NCATS position, and PAN looks forward to seeing the new center move forward in its important work. 

As far as future advocacy, PAN will continue to fight for strong NIH funding, including for NCATS.  And, of course, PAN will be watching to ensure NCATS really does position itself to make a difference in the development of better therapies and cures.

What did PAN learn during this process?

“We learned a lot of lessons in the four years we’ve been working on this issue,” said Rick.  “We expected our biggest hurdle to be explaining why what seems to be an organizational issue at NIH – meaning, the creation of a new institute – actually has huge impact on research and could make a difference.  We envisioned getting feedback from our colleagues like, ‘Really?  Moving an institute or making an organizational change could help cure a disease? How is that possible?’  Much to our surprise, we had support on that notion from the get-go.”

Perhaps the biggest hurdle was and continues to be the fear that basic/academic research centers have that this new translational center at NIH undermines the already-limited resources for basic research. 

“This was the most language-specific advocacy effort PAN has ever worked on,” said Rick.  “We had to make sure everyone knew what we were proposing, so people couldn't define their own demons and be misled.  We had to explicitly say, ‘we're not suggesting all money be moved from basic research into translational and we're not suggesting NIH isn't already doing translational work’,” she added.  “We had to acknowledge the great work they were already doing, but say that it needed to go further.  We were asking NIH to change in a real, substantive way.  Yes, it might affect people’s careers and work, but we also knew that if things stayed the same way, there would continue to be an unacceptably large disconnect between basic research and the development of new therapies.  This would not be okay,” she added.

The origin of the word “advocate” stems from the meanings “to plead cases,” “one called to aid,” “to call,” and “a voice.”  PAN’s role in advocating for CAN and NCATS encompassed all that, and more.  NCATS and CAN would not exist without the collective effort across the broad biomedical research spectrum.  It wasn't siloed by diseases or interest groups, nor was it merely a "here's an idea" coalition-building campaign.  There was a solid year or more's worth of research, work, and discussion before a proposal was even floated for consideration in legislation.  People and organizations that had never worked together before joined forces for a common purpose that, PAN believes, might lead to some new treatments and possibly cures.

“A change this big takes time,” said Rick.  “You have to be thoughtful, inclusive, and do it right. But that's what advocacy is all about.  It’s identifying an area for change, presenting a case, and raising your voice.  Whether it’s from one person, or a chorus of many, it’s not just every person’s right, but responsibility if we want to move anything forward and create a better future.”


Back to January 2012 Monthly Message