Orphan Drug Exemption Loophole

Loophole 1:

I am in Washington D.C advocating for Congress to pass the RACE for Children Act. I bet some of you are wondering what is the RACE Act, why is it important and why is the Swifty Foundation supporting it.
The RACE for Children Act is an update of the Pediatric Research Equity Act (PREA) which requires drug companies to develop their drugs not only for adults, but for children as well.
Today I thought I'd share some of the information we share with our congressional representatives and why the RACE Act is so desperately needed. There are two loopholes in PREA that we are addressing with the RACE Act. One of the two loopholes that we're trying to fix in PREA is known as the orphan drug exemption. This exemption allows drug companies to receive a waiver for conducting PREA studies for an orphan drug. An orphan drug is a treatment developed for an orphan disease, which is a condition that affects less than 200,000 people in the United States. It doesn't seem like this should apply to cancer, considering how unfortunately common it is in our society today. However, since cancers are so sub-classified, many very specific cancers are designated as orphan diseases. When they occur in children, drug companies do not have to test them as required by PREA. From our own research, we found that 90% of cancer drugs developed in the past 5 years have been designated as orphan drugs. That right there kicks out 90% of these possibly life-saving drugs from being tested on children. The RACE for Children Act would completely eliminate the orphan drug exemption so that cancer drugs could be studied in pediatric populations. It’s only fair, right?   Kira is currently in Washington representing Swifty as a Youth Advocate for Kids v Cancer. Her focus is educating legislators on the urgency in getting the RACE for Children Act passed. You can follow her updates on our Facebook page. www.facebook.com/swiftyfoundation

Making Michael Proud

  I start every meeting by opening my padfolio (that frankly took way too long to find at Office Max). In it, I have a few talking points scribbled down that I make sure to cover in every conversation I have about RACE. I try not to make my pitch seem too scripted, but I almost always have to glance down at my notes at some point during each meeting. However, there is one part of my spiel that never requires me to break eye contact with the person across from me.  

"The reason I'm here is because my best friend, Michael, was diagnosed with medulloblastoma at the age of 10..."

As I begin to tell Michael's story, I see the immediate change of expression of the staffer I'm meeting with. Sometimes it's a half frown. Other times it's an awkward grimace. No matter what face is made, though, it always makes the atmosphere somewhat uncomfortable.

"...Michael was treated with drugs that were 30-40 years old, which is unfortunately a very common practice when it comes to pediatric cancer..." I usually get a nod after this, suggesting some sort of an understanding of this horrible issue. This seems to create common ground between me and the staffer, so things get a little more comfortable. "...Michael's cancer ended up metastasizing to his spine, and he passed away at the age of 15." Any ounce of comfort shreds away as the staffer anxiously tries to come up with an appropriate response as quickly as possible. I don't blame them; most people are extremely uncomfortable with addressing loss. That being said, I receive my fair share of apologies and awkward hums. I always prepare for these responses so that they don't throw me off in the middle of my meetings. But wow was I thrown off by the response from a woman I met with on Friday.

"Michael is very lucky to have a friend like you. He is definitely so proud of you."

I was completely taken aback. I couldn't verbalize to that woman just how much that meant to me, and I can't find the words to explain it here, either. Hearing those words just made my heart light up, and I think my face followed suit. At the end of the day, I'm just trying to honor the life of the boy that impacted mine so much. I don't do any of this for recognition, but making Michael proud is perhaps the greatest feat I could ever achieve. I just hope that woman was right.

Hope on the Hill

I've been at this for a couple weeks now, but I still hold my breath every time I walk into a meeting. I fear that my knowledge of this issue isn't actually as extensive as I think it is. I fear that I will mess up so badly in my explanation that my credibility will be completely shot. I fear that a question will be thrown at me that I just cannot find an answer to. Mostly, I fear that the cause I am so passionate about will not have the same impact on the person sitting across from me. But alas, I leave every meeting with a smile on my face and a certain warmth in my heart. As soon as I open my mouth to begin my little speech, all my fears fade to the background. I look across the table and see the face of someone who has been affected by pediatric cancer; indeed, it is nearly impossible for this disease to not touch someone's life in one way or another. In every meeting, something is said that affirms everything that I'm doing here, and this refuels me for the next meeting on the docket. There are a lot of words I could use to describe the feeling I get on my way out of these meetings. If you've been watching my Facebook Live videos, you know I revert to the term "excited" quite a bit (probably way too much). I could also say "encouraged", "inspired", "vivacious", etc. However, there is really only one word that can truly encompass the wave that comes over me. That word? Hope.

First Week on the Hill

  My first week on the hill is officially in the books! When I reflect on this past week, I am almost speechless, which anyone who knows me would tell you is quite the rarity. All I keep thinking is "Wow!" Unfortunately, while that perky little exclamation completely encompasses my current emotions, it doesn't really give you much insight into what I've actually been doing this week. Lucky for you, I took it upon myself to come up with three words that describe my DC experience thus far. Those words are: chaotic, exciting, and meaningful.

Chaotic.

Let me tell ya, the hustle and bustle of DC is crazy! Perhaps the most hectic place I've ever been in my life is the Metro Center station, which is where I transfer from the red line to the orange line (sorry for the unnecessary details, I'm just really enamored by public transportation). The sheer number of people moving around is enough to leave me in awe. On top of that, everybody looks super important and professional...it's honestly pretty intimidating to the little 19-year-old that still sleeps with her Simba Pillow Pet. However, I'm learning to find my place in the midst of all these fancy humans. After all, I think the work I'm doing is pretty fancy. My commute is only the beginning of the chaos, though. On Monday, the two other interns (Carly and Anna) and I had four meetings with congressional staffers. We didn't meet with any congressmen themselves, but rather their legislative assistants. Going into our first meeting ever was incredibly nerve-racking. We had practiced our spiel over and over, but no amount of rehearsal could've prepared us for the feeling we got when we entered the office of someone who helps run our country. Luckily, the shock wore off quickly and we had a phenomenal first meeting. The encouragement and positivity we received was so amazing, and it gave us fuel for the rest of our busy day. We ran from Rayburn to Cannon, back to Rayburn, then back to Cannon for our four meetings...needless to say, my Fitbit congratulated me on 10,000 steps by the afternoon. Overall, the response from the staffers was to keep doing what we're doing to support the RACE for Children Act. They said it was people like us who give things momentum in DC, and I couldn't agree more. There was supposed to be a subcommittee markup of RACE on Wednesday, but it got postponed at the last minute, so we had to roll with the punches and work from the office. Aside from lobbying, we have several other projects we are working on for Kids v Cancer. Clearly, we have a lot to juggle, but it feels nice to be so productive especially for such an amazing cause. We continued our work on Friday with five scheduled meetings. They were all right in a row, so we were running all around the House buildings. We also had an unexpected meeting come up, bringing our total of the day to six. It was incredibly tiring, but I feel really confident that we gained a good amount of support.

Exciting.

At this point, I suppose it's appropriate to apologize for the rambling in this blog post. It definitely has everything to do with my uncontrollable excitement! I am just so ecstatic to be here and to have this incredible opportunity. Whenever I walk past the Capitol Building or see the Washington Monument or head into a meeting, my heart flutters a little bit. I'm fairly certain I've been running solely off of adrenaline and turkey sandwiches for the past week. Besides being in DC, what excites me the most about advocating for this bill is all the science involved. Most of you probably know that I am studying to become a pediatric oncologist, but I don't think I'm the typical pre-med kid. I am genuinely enthralled by science and medicine! I love learning about pediatric cancer research and then using this knowledge to help promote RACE. My role in our meetings is to discuss the scientific aspect of this bill, and every time I say the words "genetic mutation" or "molecular target", I can't help but smile. It excites me so much that the work I'm doing with RACE now could potentially affect my future research endeavors. How cool would that be!? I think it's pretty darn neat.

Meaningful.

In all the chaos and excitement, it'd be really easy to lose sight of why I came here in the first place. The other day, when I was making my morning commute, I looked down on my left wrist to check the time on my Fitbit. All I was worried about was getting to my destination as quick as possible. It was in the midst of my senseless rush that my eyes wandered from my left wrist to my right. I traced my fingers over the letters engraved on the orange wristband that became a staple of my wardrobe over four years ago. When I got through all 16 letters that spelled out "Michael Gustafson", I paused for a moment. I realized that it had been quite a long time since I last paused and really remembered my sarcastic, athletic, kind-hearted best friend. I'm so happy that I had the chance to actually stop and think about the huge impact Michael's had on my life. His influence ranges from my career goals to my humor to my outlook on life. It is an honor and privilege to keep his legacy alive in any way I can. That is why the work I'm doing here is so meaningful to me. When I called my mom the other day, I was telling her how great it felt to be doing something with my true passion again. While at college, I wasn't able to do much with Swifty, and I didn't realize how much of a hole this was creating in my life. However, being here and advocating for kids with cancer is repairing that hole and then some. It's times like these that I feel God is confirming the path He's set out for me. I know this is what I am meant to do, and I feel so blessed that I have been endowed with the knowledge and compassion to do God's work on behalf of Michael.

Thoughts from 36,000 ft

I'm about half an hour into my flight to DC, and wow am I excited! Within the next hour, I'll set foot in the place that I'll hopefully leave my mark on in the next 6 weeks. I still can't believe I'm on my way to work as an intern for Kids V Cancer and lobby for the RACE for Children Act; I keep pinching myself to make sure this isn't all a dream! This opportunity is unlike any I've ever had before, and I feel so blessed that all the plans fell into place the way they did.

I had long been searching for a summer internship involving medical research, but none of the fish were really biting there. Then, Pat Pat (more commonly known as Patti Gustafson) called me up in March and told me she found the PERFECT internship for me. She told me Kids V Cancer was looking for an advocacy intern--and before she could finish her sentence, I was online applying. For anyone who isn't familiar with Kids V Cancer, I encourage you to go check them out because they are the epitome of everything a pediatric cancer foundation should be; they're total nonprofit celebrities!

I couldn't believe it when I got an email from them to arrange a phone interview. When the phone call occurred, I must've sounded like a little kid at Disney World because I was just so thrilled to be associating with some of the nation's top childhood cancer advocates. You can imagine my reaction, then, when I got the email offering me the position! I honestly don't think I've stopped smiling since then.

With help from Pat Pat, my wonderful mama, and Elena at Kids V Cancer, I found the family that I will be staying with for the duration of my internship. I can't thank this family enough for so graciously opening their home to me (in exchange for babysitting their absolutely adorable kids). There were so many players in making this internship happen for me, and I'm just so grateful to all of them.

It's gonna be tough being away from my friends and family again after just getting home from college, but I know this is what God meant for me to do at this time in my life. I can't wait to do His work in DC and help all the kiddos out there who are fighting for their lives. Ultimately, that's what this is all about. I'm going to help children with cancer. I'm going to be their advocate. I'm going to be the voice for the voiceless (inspired by Bridget). I'm going to honor Michael. I'm going to make a difference.

As you can probably tell, it was quite a long journey to get to this snug middle seat 6,000 feet in the air, but I know the greatest journey of all is the one that lies before me. I invite you to tag along on my adventure by following the Swifty Foundation on Facebook and checking my blog at www.swiftyfoundation.org. Be on the lookout for updates on my journey!