The Waxman Report

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The Waxman Report Page 6

by Henry Waxman; Joshua Green


  At that time, the law offered little protection for someone with AIDS. There were no federal health privacy laws, no confidentiality standards, and nothing to prevent discrimination (the Americans with Disabilities Act would help rectify this, but not for two more years). People genuinely feared losing their jobs, housing, or custody of their children, so a confidentiality provision and a guarantee against discrimination were obvious additions to the bill. The idea was to bring people in by eliminating disincentives and then to pay for testing and counseling. Republicans balked. To a person, those on my subcommittee told me, “I’m with you on counseling, testing, and confidentiality, but I can’t support you on a nondiscrimination clause because that would look like a ‘gay rights’ bill.” So we reluctantly dropped nondiscrimination protections at the outset.

  Though it lacked much Republican support, the bill made it through subcommittee and was narrowly approved by the full House. Ted Kennedy had sponsored an AIDS bill that cleared the Senate, but dealt only with funding for research. That spring the House and Senate bills went to conference, where I aimed to insert testing, counseling, and confidentiality into the final bill, and Republicans kept up their efforts to kill it. One morning during the markup, Dannemeyer arrived, arms bursting with videocassettes and comic books from the Gay Men’s Health Crisis in New York City, apoplectic that these materials “erotized” safe sex—never mind that, lacking any federal funding, they did not fall under congressional purview. (Once, seated next to him at a hearing, I couldn’t help myself and asked, “Bill, what are your thoughts on masturbation?” With a grave look, he turned to me and replied, “I don’t think there’s anything we can do about it.”)

  It was not Dannemeyer, however, but North Carolina senator Jesse Helms who brought down a comprehensive AIDS bill that year. Negotiations between the House and Senate teams had progressed to the point that an agreement looked imminent, when Helms placed a “hold” on the bill, preventing it from moving forward until all but the research provision had been stripped. “I don’t want any confidentiality on these records,” Helms declared. “Nothing at all.” I was furious and let him know: “A lot of lives are going to be lost because of what you’ve done.” He replied that they would be lost because of a “gay disease.”

  The following year, we began anew. AIDS had gained a morbid kind of momentum by now, to the point that metropolitan emergency rooms were being overwhelmed. As Koop and others had long predicted, AIDS did not limit itself to the gay community. Now it was straining the entire health system and clearly needed to be addressed.

  As activists and interest groups came together to lobby for attention and support, the public perception of AIDS slowly began to shift. An important catalyst for this change was Ryan White, a thirteen-year-old boy from Kokomo, Indiana, who had acquired AIDS from a blood transfusion. White had been banned from public schools, whereupon he and his mother had responded by launching a public education campaign. An enormously compelling figure, Ryan White soon became the literal poster boy for the disease, changing many people’s perception of AIDS because he so clearly did not fit the stereotype of the victim as a gay man or a drug user.

  In 1989, we wrote a new bill that began with counseling, testing, confidentiality, and treatment, and later came to include research funding to study pediatric AIDS, since virtually none was available. On the Senate side, Kennedy introduced emergency impact assistance for hospitals. To gain Republican support, he approached Orrin Hatch of Utah. Lacking big cities, Utah hospitals were not yet being overrun, so they did not stand to receive emergency assistance. As a compromise with Hatch, Kennedy included block grants to the states so that even areas as yet unaffected by AIDS could also build an infrastructure to handle the epidemic when it arrived.

  I, too, was concerned about getting Republicans on board.

  Though bipartisanship seemed to fall into disfavor during the Bush years, my experience has always been that it is nearly impossible to pass major legislation without support from both parties. As I’d learned from watching my predecessor, Paul Rogers, getting everyone involved in a bill vastly improved the chance that it would become law.

  This presented an obvious challenge: Bill Dannemeyer, the subcommittee’s most vociferous member, wasn’t about to cooperate on a bill, block grants or not. He continued to offer “grenade amendments” requiring mandatory AIDS testing. So we went above him, to the full committee level, and approached Norman Lent, a New York Republican who was ranking member of the House Energy and Commerce Committee, and whose district in Long Island was very much affected by AIDS. But it remained a tough sell to get a Republican’s support for such legislation. One reason that Paul Rogers was so effective a chairman was his willingness—uncommon in Washington—to parcel out credit to other members. At around this time, an activist named Elizabeth Glaser approached my staff with a proposal regarding pediatric AIDS. I suggested that she take it to Lent, who could offer it as an amendment to our bill. A major impediment to Republican support was that AIDS still conjured images of drug users and gay men among many of their constituents. However, pediatric AIDS, as Lent understood, was a noncontroversial exception. By adding his amendment, we were able to build bipartisan support around Dannemeyer, further strengthening the bill.

  Even then, its fate was unclear. Reagan was gone by now, but his successor, George H. W. Bush, did not support the legislation. And the Senate prospects once again looked iffy. The person who finally tipped the balance was a Republican senator from Indiana, Dan Coats. Coats had not planned to support the bill. But legend has it that Ted Kennedy persuaded him by offering to rename the legislation the Ryan White Comprehensive AIDS Resources Emergency (CARE) Act in honor of Coats’s young constituent, a powerfully savvy ploy on two fronts. By the stroke of a pen, it shifted public attention from gay men and drug users to a thirteen-year-old boy who many already knew of and admired; renaming the bill after Ryan White also subtly pressured Coats to support it or risk looking callous in a way sure to be noticed back home. When Coats switched his vote, several others did, too—the support of so staunch a conservative as Coats provided political cover.

  Ryan White died on April 8, 1990. Four months later, President Bush signed his act into law. The Ryan White CARE Act was initially adopted for a five-year period, and has since been reauthorized three times. It continues to provide assistance to hundreds of thousands of low-income and uninsured people living with HIV and AIDS and, as the profile of the disease has shifted, spreading into rural areas, it has been updated to keep pace.

  CHAPTER 4

  The Orphan Drug Act

  ONE FALL MORNING IN 1979, BILL CORR, A MEMBER OF MY subcommittee staff, was sitting at his desk when the phone rang. A panicked caller named Muriel Seligman told him that her son Adam suffered from a rare neurological disorder known as Tourette’s syndrome, for which no treatment was available in the United States. Adam’s doctor had told them that a drug called Pimozide, sold in Canada, helped alleviate the involuntary motor tics, cursing, and guttural noises that are symptoms of Tourette’s. So Muriel Seligman, feeling she had no alternative, had asked a friend to fly to Canada to obtain Pimozide for her son. She was frantic because earlier that day customs agents at San Francisco International Airport had intercepted the friend as he returned and seized the medication because it was not approved for use in the United States. As a constituent, she was calling to demand my help. “They took the drug that my son needs,” she said. “What are you going to do about it?”

  A frequent complaint about Congress is that it does not respond to people’s needs. But Mrs. Seligman’s phone call demonstrates that this is not always the case. The concern for her son that prompted a call to her congressman set in motion a chain of events that culminated in legislation that addressed not only Adam’s plight but those of millions of other Americans just like him who were silently suffering from rare diseases.

  Disturbed by the Seligmans’ story, my staff began by looking into the obvious question:
If Canada offered a safe and effective treatment for Tourette’s syndrome, why wasn’t one available in the United States? The answer soon became clear. Tourette’s was an ailment that afflicted so few people that it did not hold sufficient profit potential to entice any U.S. pharmaceutical company into the costly process of developing and gaining approval for a treatment. Tourette’s fell into the broad category of “orphan diseases” whose victims had little hope of ever finding a treatment or cure because their numbers were so few. The situation was especially tragic, we learned, because scientists who discovered promising new treatments for orphan diseases often could not interest profit-minded drugmakers. These products thus became known as “orphan drugs,” and pharmaceutical companies rarely pursued them, even in cases when a foreign drug like Pimozide demonstrated clear potential.

  When a member of Congress confronts this sort of dilemma, the first challenge is to gain a sufficient understanding of the problem, and then to figure out what the government can do about it. In an effort to learn more about orphan diseases and how we might help those stricken by them, the Health and the Environment Subcommittee scheduled a preliminary hearing on Capitol Hill in June 1980. We invited Adam Seligman, and several doctors, government officials, and representatives of the few organized rare-disease groups we could find to testify.

  What we learned at the hearing was that although the federal government and the private pharmaceutical industry spent hundreds of millions of dollars a year for biomedical research and drug development, our country’s system of discovering and developing new drugs contained an important flaw: It did not account for the inherent financial disincentives to producing orphan drugs, and therefore failed to serve millions of people like Adam Seligman who suffered from rare diseases. Since no government policy addressed this shortcoming, creating a mechanism to facilitate the development of these drugs seemed a promising line of pursuit.

  Every day that Congress is in session, members are constantly being barraged by problems like this one that demand their urgent attention. The number of these competing claims unfortunately far outstrips the time and resources that Congress can apply to them. One benefit of a hearing like the first one we held on the orphan drug problem is that it can take an abstract policy issue like pharmaceutical development and bring it vividly to life with searing human examples of who it affects and why Congress must act on it.

  When his turn came to testify, Adam Seligman took a seat before the members of the subcommittee. At eighteen years old, Adam was slender, dark-haired, and handsome, but the simple act of carrying on a conversation required enormous energy; he was constantly fighting through the frequent outbursts and loud guttural noises that Tourette’s inflicts, which made his willingness to testify before Congress all the more courageous.

  I began by asking Adam to describe what his life had been like. He recounted the story in stages. At age eight, the first signs that something was not right. The tics that began soon after, developed into muscle spasms, and finally into the horrific “dystonic reactions”—the doctor’s term for the sudden jerks that snapped his neck back so violently that he couldn’t breathe. The emergency room visits that ensued, and the procession of mystified doctors who could not even give his mother a name for her son’s affliction. The years of hopelessness and fear continued until, at age fourteen, the genetic clues finally yielded a diagnosis. His eighty-two-year-old grandfather had been the unlocking key. Long ago, doctors had told the old man that the tremors in his hands and feet were caused by Saint Vitus’ Dance. But Adam’s new specialist recognized Tourette’s. Haldol had eased the tremors, but brought fatigue, depression, blurred vision—made it impossible to function. Adam had had to repeat senior year. Pimozide was a magic elixir. He tore through two years of high school in nine months and, just before the hearing, graduated with extra credit. But the Pimozide was gone now, and the symptoms returning.

  “What will you do without Pimozide?” I asked him.

  “I don’t know,” he replied. “When the tics start to get really bad again I will have to go back on Haldol, which I would really not like to do.”

  Amid the sadness of cases like Adam’s were also tales of great heroism and perseverance on behalf of the ill, such as that of Dr. Melvin Van Woert. Like Adam Seligman, Dr. Van Woert’s patient suffered from a rare but treatable condition, in this case a neurological disorder called myoclonus, so debilitating that it had forced her into a wheelchair. Though a treatment existed, no pharmaceutical company considered it commercially viable, and so none would agree to bring it to market. For years, Dr. Van Woert, relying on grants from private foundations, had hand-mixed the drug himself with ingredients purchased from a biochemical supply house that ordinarily serviced veterinarians, and had kept his patient out of her wheelchair.

  The day’s testimony convinced most of us that this was a clear case of a problem that Congress could play a constructive role in solving. Next we needed to figure out the best course of action. Only then could we turn to the greatest challenge of all: figuring out how to build public momentum to fix a medical issue that even many doctors were not aware of.

  * * *

  THAT FIRST HEARING, IN JUNE 1980, DREW A SPARSE CROWD AND little public notice. Only the Los Angeles Times sent a reporter, and only because Adam Seligman was a local resident. But this was enough to deliver an unexpected boost. The next day, a Hollywood writer and producer named Maurice Klugman happened upon the Times article and was moved by what he read. Klugman himself was battling a rare form of cancer. A producer of the hit television drama Quincy M.E., which starred his brother, the actor Jack Klugman, as a crusading medical examiner, he decided to write an episode of the show devoted to Tourette’s syndrome and the orphan disease problem. At the end of the episode, a message explained to viewers that the story was based on real events and invited them to write in if they wanted to help. In the weeks and months after the show aired, thousands of letters poured into the Quincy production studio from viewers eager to help raise public awareness.

  In the meantime, I used my chairmanship of the Health and the Environment Subcommittee to press ahead. I was not the only member of Congress concerned about orphan diseases. Elizabeth Holtzman, a Democrat from New York, had previously introduced a bill calling for the government to develop orphan drugs through the National Institutes of Health. Holtzman’s rationale was that NIH scientists already conducted biochemical research using drugs; she wanted to expand the agency’s responsibilities to include developing them for the market as well. Holtzman retired in 1980, but appealed to a colleague, Ted Weiss, a Manhattan Democrat, to reintroduce her bill after she left. Weiss had done so, but the measure had not gotten far because Congress was reluctant to provide the considerable outlay that a major new government initiative would require and because some members wondered whether the private sector might not do a better job. The fundamental question that we needed to decide in order to put together effective legislation was whether government or the pharmaceutical industry was better suited to the task of developing orphan drugs.

  To learn more about the Holtzman-Weiss approach and the private industry alternative, I organized a second hearing for the spring of 1981. Orphan drugs remained an obscure issue, so we needed to draw more attention to the problem to foster a sense of urgency and pressure Congress to act. The Quincy episode devoted to Tourette’s syndrome was scheduled to run on March 4, 1981. So we decided to hold the hearing the following week and invited Quincy himself—Jack Klugman—to testify, along with pharmaceutical industry representatives, government officials, and a broad group of people with orphan diseases.

  Hollywood celebrities are so prevalent on Capitol Hill these days that they rarely cause much of a stir. But in 1981, the appearance of a bona fide television star like Jack Klugman at a congressional hearing was a major news event. On the appointed day, The New York Times ran a front-page story on Klugman and the orphan disease problem. While our first hearing, nine months earlier, had taken place be
fore a nearly empty room, this time we arrived to find it jam-packed with cameras and reporters.

  This was, of course, precisely the effect we had intended by inviting Klugman to appear. Orphan diseases had been ignored or overlooked for years. Now, suddenly, they were in the spotlight. Klugman’s testimony had a mesmerizing effect, and not just on the news media—in a rare moment of levity, my colleague Jim Scheuer of New York began asking the star witness scientific questions, as if he were a real medical examiner, rather than an actor who portrayed one on television.

  But even Klugman’s star wattage could not overshadow the testimony of those stricken by orphan diseases, who spoke next. After Adam Seligman appeared before Congress the previous summer, the news had spread to people with all sorts of rare ailments that a few people in Washington had at last noticed their predicament and wanted to help. The first hearing had featured testimony from just one other victim besides Adam because we had had difficulty finding others; no national group existed then to organize and advocate on behalf of this underserved population. This time, however, the hearing room was filled with victims, many of them children, of some of the rarest and least understood disorders known to medicine—people with terrible skin ailments, crippling cancers, elephantiasis, and conditions that caused webbed fingers and internal organs. They had in common the exotic nature of their maladies.

  At the time, Tourette’s afflicted only about 100,000 people, not nearly enough to interest drug companies, but still more than many of the other diseases and conditions that were represented that day by victims and their families. They included muscular dystrophy, a congenital disorder that weakens the muscles; cystic fibrosis, a deadly hereditary disorder (40,000); spina bifida, a congenital neurological condition (27,500); Huntington’s chorea, a degenerative disease of the mind and nervous system (14,000); ALS, better known as Lou Gehrig’s disease (9,000). Then there were the truly obscure ailments. Prader-Willi syndrome, a fatal ailment that causes huge weight gain in children, afflicted about 2,000 a year; Wilson’s disease, an abnormal accumulation of copper in the liver and brain, just 1,000; and cystinosis, a genetic disorder that usually causes kidney failure by age ten, struck about 100 children a year.

 

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