Thanks: Sandra, Reagan, Abby, Julia and N. all helped me enormously on this story. Truly, I cannot even begin to express my appreciation in anything close to a fitting way. Simply put, without them there would be no story
The One He Chooses by valentine |
Excerpt from the National Organization for Rare Disorders (NORD) Testimony before the Senate Health, Education, Labor and Pensions Committee:
In 1983, to address a longstanding, unmet need to develop new treatments and cures for rare diseases and disorders, Congress enacted the Orphan Drug Act. Congress sought through the Act to encourage the development of new ''orphan'' treatments and cures for the millions of Americans with rare diseases. As defined by the law, an "orphan disease" is one that affects fewer than 200,000 people in the United States. These include such obscure disorders as Lesch-Nyhan Syndrome, Tay-Sachs Disease and Wilson's Disease, as well as more common disorders including Hemophilia, Sickle Cell Disease and Cystic Fibrosis. Historically, such relatively small patient populations were considered too small to justify financial investment by the private sector pharmaceutical research enterprise. Rare disorders represent very small markets and potential treatments were deemed "drugs of limited commercial value." Even with the economic incentives provided under the Orphan Drug Act, it remains very difficult to entice private companies to spend money on research for most rare diseases.
Even in the face of this continued reluctance, the Orphan Drug Act has proven extremely effective. In the ten years prior to the Act, 38 "orphan drugs" were developed compared to the 220 such drugs developed in the nearly 20 years since. Despite this success, patients with rare diseases or disorders still continue to face challenges in receiving appropriate and adequate treatment, and many of these afflictions have no approved treatments at all. Unfortunately, significant opportunities for rare disease research remain unmet because companies will not or cannot commercialize them. The Rare Diseases Act of 2002 (H.R. 1379) is intended to build on the successes of, and improve upon, current law in generating urgently needed treatments and cures for rare diseases and disorders. This Act seeks to further stimulate the research and development of orphan drugs, by giving statutory authorization to the Office of Rare Diseases (ORD) at the National Institute of Health (NIH), and authorizing new funds for the Orphan Products Research Grant program.
Sunday Night
Charlie heard the TV click off in the family room and looked over at the clock -- ten after eleven. It was getting close to Deena's unofficial bedtime. "Unofficial" because he didn't like to be terribly strict with his sister. It was a difficult line to walk sometimes, between brother and de-facto parent. But he sensed that Deena knew that and gave him much more latitude than she ever had their mother. Still, it was always good when they could just avoid the whole parenting interaction altogether.
Sometimes when Charlie looked at his sixteen-year-old sister, he could see what his mother must have looked like in her high school days. The strong hands, the way she always threw her shoulders back and held her head up, the small dimples that appeared when she laughed. Sometimes it broke his heart just to look at her.
Hearing her door close he turned back to reading the reams of Senate testimony he had to get through before his next class. The American Political Systems professor had asked them each to choose a current bill and follow its progress through congress.
The reading, unfortunately, was not doing a very good job of keeping him awake. Feeling his eyelids getting heavier with each passing minute he finally gave up, reaching over to set his alarm before turning out the lamp. He allowed the soft patter of rain outside his window to lull him to sleep.
Within minutes, it seemed, he was startled awake by a presence in his room.
"Charlie?" He could barely hear the faint voice.
"What's do you need Dee?"
"My knees hurt."
Charlie sat up; all vestiges of sleep gone immediately.
"Like last time?"
"Worse."
"Okay," he rubbed his eyes, trying to abate the frustration and concern growing in him. "I told you not to play so hard Dee. You can't overdo it like that."
"Do you think we could skip the lecture this time Charlie? It's gotten kind of old."
"Yeah, okay." His frustration wasn't so much directed at her, it wasn't something she could control anyway. "Well, what do you think?"
"I think I need to go to the clinic."
Charlie cringed at the fear in her voice. It amazed him sometimes that this never seemed to get any easier. He thought that if life had been fair, it should. Then again, if life was fair, Deena wouldn't be sick and his mother wouldn't be dead.
"Okay, grab your bag, let me call Dr. Richards."
As Deena left the room, Charlie looked out the window, trying to decide what he would tell his boss in the morning.
Monday Morning
By the time Sam managed to hobble into the White House, the sun had been up for a good hour, meaning he was late for his morning appointment. He considered himself fairly fit for a man his age; and, more notably, for a man in his line of work. Maybe he couldn't run a six-minute mile anymore, but he could more than hold his own in the athletic arena. At least most of the time he could. There were just some things that he shouldn't do anymore. Playing a pickup basketball game with the President's twenty something body man and his younger sister was high on that list at the moment. Not only were his quads aching but it had also been raining steadily since late the previous night. Any other day he'd have been a little more wary of puddles on the marble floor. Any other day, he was sure, he would have avoided the whole incident. But today it was raining and he was late, and before he could really understand what had happened, he found himself flat on his back in the middle of the White House lobby.
"Ow," he gave silent thanks that the lobby was empty except for the security guard, who could be heard trying, unsuccessfully, to stifle his laughter.
"Sam?"
Sam suddenly found himself looking up into the hair-obscured face of what appeared to be, at least from his current vantage point, a rather tall brunette woman.
"I'm sorry..." he mumbled pushing himself into a sitting position. Cursing the offending puddle that had, by now, soaked through his jacket and shirt, he looked harder at the woman before him. "Do I know you?"
Laughing, the woman set her briefcase next to him on the floor. She squatted down, pushing the hair behind her ears. Looking him directly in the face, she laughed again, high and light and echoing like a bell in the high ceilings.
"You look like someone pulled the rug out from under you there, Sam. Say something."
"Linda," Sam exclaimed, finding his voice again.
"Sam."
"Linda!"
"Yes, and you're Sam. And you're wet, not to mention on the floor."
"Oh, god, I'm sorry...I..."
"Here." Linda rose to her feet again and offered her hand.
"Thanks. I'm sorry, I'm not normally this clumsy." He grimaced slightly as he struggled to his feet.
"You've always been this clumsy."
"I have not." Sam turned to look at her again, glad his initial impression of her stature hadn't been accurate. Drawn up to his full height, he was a good half-inch taller than her, "I am not."
"Oh come on, I specifically remember a time involving a boat-"
"It was slick and that wave caught me off guard!"
"Whatever."
"It's true."
"All right," she raised her hands in mock surrender before bending to retrieve her briefcase. "So what's your excuse today. Did that tile there sneak up on you?"
"Funny! No, I was just in a hurry, I'm supposed to be meeting with this guy from the National Organization for Rare Disorders." He gestured for her to follow him through the doors to the bullpen.
"I know."
"I have this...wait, how did you know?"
"I'm your guy."
"You're my guy?"
"Are you sure you didn't smack your head? You're usually much quicker on the uptake."
"No...yes...wait. I'm meeting with you?" Sam guided her into his office and set about removing his wet jacket. He vaguely remembered hearing that Linda had become a lobbyist for NORD a few years ago, but was sure he hadn't seen her name on his calendar.
"Yep."
"How come your name isn't on my schedule?"
"It is, see," she leaned over to point to the appropriate place on his calendar. "L. Wiley, National Organization for Rare Disorders."
"But your last name is Ryan," Sam stated, still confused.
"It was Ryan, I went back to my maiden name."
"When?"
"About two years ago," she replied softly, carefully not looking down at her wedding band.
"Oh." Sam was silent, unable to look Linda in the eye. He remembered hearing about Collin's death, but couldn't remember if he'd sent flowers or even a card. He'd never really even seen the two of them since leaving Dewey Ballantine. Linda and Collin were a few years older than he and Lisa and didn't really frequent the same social circles. He suddenly wished that he'd made a greater effort to keep in touch. "Listen Linda, I'm sorry, I mean I should have come..."
"It's okay."
"No, I... really...he was a good man. You two were good."
"Thanks," she whispered. Staring down, she absently tuggied at the hem of her jacket.
When she looked up again, Sam was struck by how she looked almost exactly the way she had in New York. Her hair was a little shorter and her eyes more haunted, but it was still the Linda Sam knew all those years ago. He blushed suddenly, realizing that she'd caught him staring. She smiled broadly at him, releasing the tension in the room.
"So how's Lisa?"
"Uh, she's good," Sam laughed, not quite sure if she was serious. "You did know we broke up...like three years ago?"
"Oh God, you know, I did know that. I'm sorry...just force of habit you know. You guys were always just Sam and Lisa," she laughed nervously, her face flushing with embarrassment. "Sorry."
"It's no big deal," he smiled, watching as she distractedly tucked stray hairs behind her ear.
"So why don't we just both admit we suck at this keeping in touch thing and get on with it. No more apologies," she offered.
"Sounds good."
"Good."
Sam smiled. "So what can I do for you today Linda?"
"Well, since you asked, I'd like 50 million dollars."
"Way to jump right in there."
"I'd thought I'd just, you know, get right to the point, avoid all the extraneous material, if you will."
"As much as I appreciate that..."
"There is a bill in the Senate right now, The Rare Diseases Act."
"Increased funding for the Orphan Products Research Grant Program?" Sam asked, trying to recall as much as he could about the act. He'd read over the bill when it was first introduced into the House, several weeks ago. The research grants were to go to small clinical trials that focused exclusively on drugs that would only benefit a small population of patients, earning them classification as orphan drugs.
"Yeah, 25 million for that and we're also asking that the National Institute of Health Office for Rare Diseases be made permanent with an increased appropriation."
"Doesn't Rare Disease already have an office within the NIH?"
"Yes and no. In 1993 the NIH voluntarily established the Office for Rare Diseases but because it's not been written into law the office has no official authority."
"And probably no legislative mandate or congressional appropriation," Sam added, making the connection.
Linda nodded. "That's what the other 25 million is for."
"I thought this was sewn up."
"Funny enough so did we," Linda shrugged.
"And you're here now because?"
"NORD would like the White House to back the legislation."
Sam leaned forward on his desk, removing his glasses. Pinching the bridge of his nose, he considered her request.
"Linda, I'm not sure this is something we're going to want to get stuck in. You had the votes, I'm sure you can find them again without our help."
"It's not that we don't have the votes Sam. It passed the House in a landslide and if it came to the floor today it would pass the Senate by almost the same margin. The problem is that Senator Terry's got it in committee and it doesn't look like he's going to let it through anytime soon."
Sam cringed at the mention of Mark Terry's committee. Health, Education, Labor and Pensions would also be the committee in charge of reviewing the Patient Bill of Rights when it came to the Senate. Touted as a bipartisan effort the passage of the bill could prove a huge boost for the President going into November. As Chairman of the committee, they needed Terry on their side to make that happen. Sam wasn't sure how much political capital they could afford to spend on lesser bills right now.
"I'm sorry, I'm not sure we can help you here."
"Sam, I'm not asking for an aggressive campaign from you guys, really. All I'm asking is a well placed phone call or two."
"With the legislation we've got coming to the Senate, with reelection...it's just bad timing."
"I know...I know this is a bad time and we're not indifferent to that, but I wouldn't be here is this wasn't important."
Sam leaned back in his chair, studying her.
"Hey, all I'm asking is that you look into it." Linda reached into her briefcase and retrieved several thick folders, laying them on the desk between them. "Here are some things we've put together."
"I can't promise anything."
"Just consider it. That's all I'm asking."
Sam reached forward to scoop up the files, looking up just in time to catch her knowing smile.
"No promises," he warned.
"Okay," she replied, still smiling.
"I'm serious Linda."
"I know."
"Okay just so long as we're clear on that."
"Great." Linda rose from across the desk and grabbed her coat. As she turned to the door Sam made his way to her.
"So I'll let you know as soon as I get a chance."
"Thank you Sam." She leaned in to place a peck on his cheek before heading out of the office.
Sam smiled as he watched her turn towards the lobby, then he headed the opposite direction across the bullpen. He tried again to remember more details about the RDA. From what he remembered it seemed like a good bill. Nothing monumental, but nothing out of line either. He couldn't imagine why Mark Terry's committee was taking so long on this. He only knew that NORD and Linda were anxious to get it moving.
"Sam!"
He was still thinking about Linda when he heard his name. Turning he found himself gazing up at CJ.
"Hey CJ."
"Hey yourself."
They passed through the communication's bullpen, heading for Leo's office.
"So I just had this meeting with a woman from NORD."
"National Organization for Rare Disorders?" CJ asked.
"That's the one. Anyway, they want us to get involved in H.R. 1379."
"The RDA? I thought that was a done deal."
"Apparently not."
"Well we shouldn't."
"Why?"
"Why? Because this isn't the time Sam. We don't want him to be perceived as too focused on medical issues right now. Not unless it's ramping up to the Patient's Bill of Rights."
"I told her I'd make a call."
"Well I'm glad my advice had such an impact."
"It's just...she's a friend."
They rounded the corner into Margaret's office, where Toby and Josh were waiting.
"Oh by the way," CJ continued, a smile crossing her face, "I heard you got spanked last night."
"Okay, seriously, what is it with you and spanking?" Sam asked, his voice indignant as possible. "Anyway, I wouldn't call it that."
"Oh? What would you call it? Trounced? Whupped? Beaten to a bloody pulp and made to cry for your mommy?"
In the background he could hear the barely contained snickers coming from Josh. When he turned to fix the other man with a scowl, he swore he could see Toby's mouth crinkling in a suppressed laugh.
Turning back to CJ, he raised his hands in concession. He hated to admit to anyone that he'd lost, but he had and now it was just as well to face up to it.
"I got beat, okay? I lost to the better player. There's no shame in that."
"No, you're right Sam, there's no shame in losing to a girl and it takes a big man to admit that." Toby was smiling, almost openly, at his deputy's misfortune.
"Hey!" CJ said, fixing Toby with a glare. "I bet she could take you too, my friend. That girl's got game."
"You know, someone your age really shouldn't be using phrases like that?"
"My age?"
"Or, you know, people of your experience and distinction," Toby back-peddled quickly.
"Better," she said, smiling slyly.
"But back to Sam now," Josh offered.
"Can I just say in my own defense that Deena really is very good, and basketball really isn't my game?" Sam pleaded.
"What's your game? Croquet? Shuffleboard?" CJ smirked.
"No, it's true," Josh spoke up, coming to his friend's defense. "He really does suck."
"Thanks."
"No problem," Josh shrugged.
"Morning," Leo greeted them as he swept past into his office. Following him Josh, Sam, Toby and CJ all took up positions surrounding the Chief of Staff's desk and waited for him to begin the meeting.
"So," Leo looked up from the briefing book he'd been carrying, "Josh, you're meeting with the representatives from Nevada?"
"Yeah, the Secretary of Energy is days away from recommending the Yucca Mountain site for construction as a high level nuclear waste repository. These guys just want to sound off once more for good measure."
"We're about to start dumping high level nuclear waste in their mountains," Toby pointed out. "I really can't imagine why they'd have concerns."
"It's not like the guys from Energy went around and had the states draw straws," Josh argued. "We spent 20 years and 40 billion dollars on this."
"And I'm sure that will come as great comfort when the trucks full of plutonium start rolling through their cities."
"You know-" Josh started before he was cut off by Leo.
"Toby's right. They have legitimate concerns and you're going to listen to them. Besides," Leo grinned at his Deputy's obvious discomfort, "Nevada's important."
"Nevada is so *not* important."
"I'm saying they have five electoral votes, so be cordial."
Josh scowled but nodded his agreement.
"Good. Toby how's the speech for the NIH dinner?"
"It's coming along."
"It's not done?"
"The conference isn't until Friday, it'll get done."
"Okay. Anything else?" Leo asked, clearly hoping that there wasn't.
"I took a meeting this morning," Sam announced. "NORD would like us to light a fire under Terry."
"What's he doing now?" Leo sighed.
"He's refusing to let the RDA move to a vote."
"That's not our problem, besides he's probably just being difficult. I don't want us involved with this."
"I know but maybe we could..."
"We can't afford to piss Terry off," Leo said, his voice stern.
"I know, but I'm just talking about a phone call."
Leo looked hard at Sam, judging how deeply he was involved with this already. The young man seemed fairly excited, though he tried to hide it. Leo couldn't exactly remember when Sam had seemed that eager to go after a piece of legislation. Perhaps it wouldn't hurt just to have him make a call or two.
"Fine. Start with the others on the committee, avoid Terry if you can."
"Thank you."
"Anything..."
They all turned when they heard the door to the Oval Office open. None of them were expecting to see the President standing in the doorway so it didn't immediately register that he was motioning them into the office.
After a moment of silence, the President sighed impatiently. "Well come on, I don't have all day."
"Good morning Mr. President." Leo was the first to recover, stepping quickly to follow the leader of the free world into his office. "So Charlie's got you opening your own doors now. 'Bout time."
"No, as a matter of fact, his sister's sick, and he wanted to stay with her this morning." Jed settled himself behind his desk, watching his staff file in. "And I'm perfectly capable of handling the doors myself, thank you very much."
"Yes, Sir," Leo smirked at his friend, enjoying the lightness that infused the President's mood this morning; a lightness that was all too fleeting these days.
"Deena's sick?" Josh asked. "Because she seemed fine enough last night after tossing Sam around the court."
Sam reached out and swatted Josh's shoulder while he and the others moved in to form a semi-circle around the President's desk. The last thing he wanted was another basketball lecture from the President.
But the President was clearly distracted, not even gracing Sam with a glance as he answered. "Yeah, Charlie said she was just under the weather. So, what do we have going on today?"
"Toby and Sam have almost wrapped up the NIH speech. We have CJ wrestling with the press. And Josh, trying not to piss off Nevada."
"So a pretty typical day."
"Yes sir."
"Okay then."
"Thank you Mr. President," they all chorused, turning to leave.
"Oh, by the way, Sam?"
Sam turned and, seeing the grin on Jed Bartlet's face, braced himself. Getting mocked by CJ was one thing, but the Commander In Chief took it to a whole new level.
"Do you know why you lost?"
"Oh, no sir, but if you could enlighten me..."
"See, it's that kind of attitude that will leave you a mediocre player Sam. Here I am about to share my vast knowledge with you and you go and be a smart mouth."
"I'm sorry sir. Tell me, why did I lose?"
"You underestimated your opponent. *Girls' Varsity* he said with disdain," Jed mocked. "Let me tell you something, I raised three girls, I even married one, and if there's one thing women know how to do, it's compete. And here's a secret: they hate it when men say as way of explanation 'well it's just girls' this or girls' that'. Am I right on this one CJ?"
"Yes, sir."
"Besides I think they like to refer to it as Women's Varsity now."
"I...yes, sir...I didn't really mean."
"Good. Now just stick to the fundamentals next time and she'll only skunk you once."
"Thank you, sir."
Josh reached out and grabbed Sam's coat sleeve as they left the Oval Office. Steering them toward Sam's office, he couldn't help but snicker.
"Wow, that was fairly painful," Sam cringed.
"He had a point you know."
"Hey, as I remember you were there last night and got pretty well beat yourself."
"Yeah, but I never doubted she could kick our collective asses. Anyway, I'm not the one who made that asinine comment."
"This time."
"I know, it's a rare day. I'm going to go revel in it for a couple minutes. You want to get some coffee and join me?" Josh motioned in the direction of the mess.
"No, I think I'll go back to my office and bury my head in the sand."
"Ahkay."
Sam wearily returned to his office only to find, on his desk, a nerf ball and matching basketball hoop. Before he could wonder which of his colleagues had provided the gift he looked back up, out into the bullpen and found himself staring at Ginger, Bonnie, Donna and CJ. All four had Cheshire cat grins and nerf balls. Realizing too late their intent, he flailed his arms out desperately tying, unsuccessfully, to protect his head from the orange balls.
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