Why the 21st Century Cures Act is a Good Thing

A Q&A with Mary Woolley, President and CEO of Research!America

Originally published Feb. 8, 2017

Q: You attended the December signing by President Obama of the 21st Century Cures Act and are recognized to be a strong supporter. Yet harsh criticism of it has quickly appeared in JAMA, BMJ, a variety of other venues, as well as on these pages. Please tell our readers why this is good legislation and how the public health will be protected from exploitation in this very different regulatory world.

A: The bi-partisan 21st Century Cures Act is grounded in a commitment to assuring that our nation’s research ecosystem has the capacity to accelerate the pace at which safe and effective medical advances reach patients. The Act will expand the efficiency, reach and impact of medical discovery in a manner that sustains crucial safeguards against unsafe or ineffective products. The law finances more research, helps to reduce the administrative cost surrounding basic research, and takes additional steps to overcome challenges the Food and Drug Administration (FDA) faces. Patient groups, health care professionals, academic leaders, industry leaders and the FDA and the National Institutes of Health (NIH) were frequently consulted regarding provisions of this bipartisan bill, and their insights were incorporated. We at Research!America were closely involved throughout development of the bill, and are pleased that it crossed the finish line last December.

After years of automatic spending cuts and flat-funding, researchers have been stressed as they work to find solutions to deadly and complex diseases. The 21st Century Cures provides some relief in that regard with an initial $352 million in FY17 to support the NIH Precision MedicineBRAIN, and Cancer Moonshot initiatives. Congress recognizes that these dollars are targeted and temporary; they do not supplant the need to grow NIH’s annual budget. As reflected in surveys that Research!America commissions regularly, Americans recognize the importance of federally-funded research and support streamlining the pursuit of medical research and innovation.

The FDA, which has for years been underfunded, will also receive new funding with an initial $20 million in FY17 to improve efficiencies in the R&D pipeline. This new funding, in combination with other provisions of the law, is particularly meaningful as it will give the FDA more flexibility to recruit additional experts needed to assure that our regulatory system can properly evaluate rapidly evolving science in areas such as immunology and regenerative medicine.

One important example of rapidly evolving science is the potential to diversify the evidence base used to evaluate the safety and efficacy of medical advances by leveraging “real world evidence” (RWE). The Cures Act defines real world evidence as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials.” While concerns have been raised that the RWE provisions would force the FDA to relax critical safety and efficacy standards, these provisions were developed with agency input. This section of the law is designed to empower, not require, the FDA to capitalize on real world data. Real world data will be used when — and only when — it is appropriate to do so.

Faster medical progress saves lives. The 21st Century Cures Act will fuel faster progress. It’s incumbent upon research advocates to engage elected officials to build on the Cures Act, and ensure that adequate funding is provided to make the promise of science and innovation a reality in our lifetime.

Copyright: This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.


How I Survive Cancer

A Q&A with Erin Maloney: Intrepid explorer, Amateur photographer, Aspiring leader; Toronto, ON

Email: erinLmaloney@gmail.com

Originally published Nov. 1, 2017

 

Q: You have recently disclosed that you have had a diagnosis of cancer and described your experience in some detail on Medium. What does it mean to you to be a “Cancer Survivor”?

A: Calling myself a survivor sometimes feels like an exaggeration. In 2016, I was diagnosed with Stage 1A2 squamous cell carcinoma of the cervix. It began with a routine pap smear and led to a robotic laparoscopic radical trachelectomy seven months later. Every procedure was challenging, and surgery was particularly arduous. However, I did not have to endure radiation, brachytherapy, or chemotherapy. I got to keep my hair, didn’t have to cope with nausea or worry about the lifelong maintenance required after radiation. So, in many ways, calling myself a survivor feels fraudulent. I have it too easy.

The physical recovery was relatively smooth but there is no preparation available for the mental toll of the Big C diagnosis. After the gynecologist shared the news, everything moved quickly. Within 10 days I had an MRI and was in consultation at Princess Margaret Cancer Hospital. It did not leave much room for preparation. In a very brief timeframe, I went from being a healthy 32-year old with no plans to have children to being a 33-year old who might not have a say in the matter.

People expect others to react in a binary way to that kind of news. There is a belief that one can choose to be optimistic or pessimistic, but that is a false dichotomy. Some days optimism abounds; most days, the fear brews below the surface unacknowledged. I mentally created a list of unanswerable questions (Would a hysterectomy be better? Would I feel like less of a woman? What if it comes back? How do I cope with this forever? Is this what kills me?). I researched and armed myself with information – a coping mechanism that allowed me to ignore my own terror.

I am not the same person I was a year ago. Prior to this, I was entrenched in the pitfalls of my Type A personality: a planner and organizer, domineering and determined, opinionated and unwilling to compromise. I had big ambitions and undaunted confidence. But our best-laid plans can be laid waste by a simple two-word sentence: “It’s cancer.” I wished I believed that this was part of some larger plan but it wasn’t. Instead, what it meant for me was that I had to change. My attitude needed to be different if I was going to come out the other side not completely broken.

Cancer took away my control. I could think of little else. Administrative inefficiencies rendered me powerless. I couldn’t get the answers I was seeking faster than they were willing to give them. For example, making decisions for myself was challenging when I needed answers from a busy surgeon. If I tried to use the same approach as I normally would, I would probably have ended up insane. So, I made the decision to adapt. I had to learn patience, to be more open to spontaneity, flexibility, and work on being able to ‘go with the flow.’ I try to allow humour and positivity to flow into impossible situations. I practice kindness and thoughtfulness as often as I can. I strive to think of others and notice their needs. Above all else, I try to treat people the way I want to be treated; to be more open and to deliver honesty without being cruel or callous.

There are many days when I fail at most of those things. Self-improvement is never easy. Imposed self-improvement as a survival mechanism is even more difficult. Finding and maintaining true positivity in the face of overwhelming terror has been the hardest task of all.

The depression that surfaced during recovery was different; it was borne out of a deep desire to live and the constant fear that I might not. I still don’t have certainty. The fear that surrounds each appointment will never abate. In a strange way, that has been inspiring. I want to live. I want a full, vibrant life that is technicolour. It doesn’t mean every day, and it doesn’t mean I get to tick off all those dreams immediately. Ultimately it means that I want to live better and that’s what makes me a survivor.

Copyright: This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.