The fullness of a week in Washington

It has been a very full week, one that began with funeral services on Sunday for a cancer friend.  During our last stint on the oncology unit in late 2017, while Lauren was in treatment for osteosarcoma and Brent was trying to bridge to his 18^th birthday in order to be eligible for an adult clinical trial for relapsed leukemia, little Viv and her family lived in the hospital with us.  She was receiving a bone marrow transplant for her own aggressive leukemia. This tiny child brightened those dreary halls and filled a room with joy and hope, just by being there. Vivian Rose was beautiful, sassy, and adored by all.

Last week, she died just hours short of her 4^th birthday.

 

I drove to Washington DC on Monday, to attend an Expanded Access Summit at the National Press Club with Viv and Brent heavy on my heart.  For three days, I listened to presentations and talked with folks from FDA, pharma and research institutions about the challenges found in balancing the competing interests of safety and opportunity for patients.  There was thoughtful discussion about the practical limitations that small start-up biotech companies face.  We analyzed the risks and costs that make it difficult for new companies to provide drugs to patients outside of clinical trial.  I recognize and appreciate these considerations. They are the very issues that brought me to this conference last year.  

I was most encouraged when Janet Woodcock of FDA endorsed a trial design that contained an ‘Open Access Arm.’  This additional arm, which mimics expanded access/compassionate use, would provide much needed “Real World Data” to researchers.  The traditional arm (which has more restrictive inclusion criteria) would better answer questions about product safety and side effects. The data from a more representative cohort could guide countless physicians who wonder how a drug will affect their much different patient after it is approved. The experiences of patients in the open access arm would inform the decisions for patients who have similar comorbidities. Currently, this more complicated type of patient is seldom seen in clinical trials. 

As an advocate, I have long called for this dual arm trial design, which might have provided opportunities for Brent and Viv, who did not fit the narrow criteria of clinical trials that existed while they were alive. I believe that there are things to be learned from every willing patient.  We should not squander that opportunity.

I still worry about Lauren, who becomes more complicated as time goes on.  As she approaches 18, I am less concerned about her age as the criteria that limits her options, than her extensive cancer history (especially the brain tumor that she had at age 9 and again at 12). Regardless of how well she is doing, this single factor would disqualify her from participation in many clinical trials. I am working to change these limitations for her, and patients like her. 

I celebrate Jane Woodcock’s call for this alternate trial design and would urge that data derived from the ‘Open Access’ arm to be given less weight in the approval process, similar to the consideration that expanded access data receives, so as to not negatively impact the application.  I hope that the signal from FDA endorsing this trial design will be widely adopted by pharmaceutical companies. However, given the costs associated with expanding clinical trials in this way, it will likely take more than just a suggestion from FDA for them to incorporate this idea into trial design, even with the benefit of having trials accrue faster.

Creative discussions are critical for change, and I certainly witnessed some of those during the conference early in the week.  I will applaud progress, even when it takes time.

There was more good news.

On Thursday, I was fortunate to meet with a member of Sherrod Brown’s staff, after he was called to Senate impeachment hearings.  I was encouraged by their effort to develop a clinical trial navigation program, as well as their transparency and continued follow up with me about the process.  I am excited for the proposed system which would educate patients about clinical trials, offer medically appropriate opportunities and partner with social work to help connect patients in need with non-profit organizations to manage the other obstacles to participation (travel and housing). Again, I believe that helping individual patients to participate in research will help advance science overall.  I look forward to reading the legislation when the policy review process has been completed, perhaps as early as next month.

After this meeting, I sat outside of Union Station, enjoying the sunshine with a cup of coffee.  A rather disheveled man holding a paper map approached me asking, “Where is the monument?” I tried to imagine what monument he meant, since there are so many in DC.  I took only few steps toward him, to try to help him navigate his map and find his destination, before a friendly local jumped in, helpfully showing him the way.  “You are going to want to take Louisiana to get to the Washington monument….”  I watched the two men and smiled. This seemed like a fine analogy of what I am working toward in clinical trials, the well informed local, helping this lost tourist find his way to the must-see monument.

The next day I drove back to Ohio, pleased with this overall progress.  But there was even more to be hopeful about.

I stopped in the afternoon to take a call with the Coalition Against Childhood Cancer, to review FDA draft guidance for the Pediatric Research Equity Act, which mandates that pediatric clinical trials be offered for new drugs that act on molecular targets that are found in both adult and pediatric cancer.  I have watched the pediatric community come together and coordinate their efforts in order to bring about this much needed change.  

The interpretation of this law by FDA has been strongly supportive of progress in pediatric cancer.  This legislation (and the FDA's interpretation) according to draft guidance would have eliminated the delay that Brent experienced, and would have required that there be a pediatric clinical trial option for little Viv. 

We can only move forward and try to make things better for others. 

I am so grateful for all those who have worked together to address the needs of the most vulnerable patients. It is a complicated problem, requiring cooperation between many groups: academia, industry, regulatory and patient advocacy.  I am grateful for the opportunity to be part of this effort, which honors Brent, serves Lauren and consequently, is terribly meaningful to me.