AIDS TREATMENT NEWS #136, October 11, 1991
John S. James

We believe that the controversy over this bill illustrates the need for a forum or institution where national policy on treatment issues in AIDS and other diseases can be discussed and formulated. (One proposal for such a structure was presented last week at the AIDS Treatment Activist Conference in Washington, D. C.) If a national policy-making body could find consensus on leading issues, then initiatives based on that consensus could start with widespread support and progress smoothly and rapidly toward implementation.

What the Bill Provides

H. R. 2872, officially titled the "Access to Life-Saving Therapies Act," begins by stating several findings of Congress:

"(1) For many Americans with life-threatening or seriously debilitating diseases, new pharmaceutical products may offer the best, and sometimes the only hope of treatment;

(2) Patients with life-threatening or seriously debilitating diseases are often denied the most advanced therapies because the drug approval process lacks flexibility;

(3) Patients with life-threatening or seriously debilitating diseases are often willing to accept greater risks with respect to the safety and efficacy of drugs than our current drug approval system allows;

(4) The current mechanisms for expanded access to experimental therapies through Parallel Track, Group C Designation, and treatment INDs do not adequately meet the needs of patients with life-threatening or seriously debilitating diseases and may create disincentives for the development of drugs for patients with life-threatening or seriously debilitating diseases;

(5) Health insurers now unfairly discriminate against experimental therapies, even when there is no alternative therapy;

(6) There is no more efficient way to expand access to a new drug than to permit it to be marketed; and

(7) The drug approval laws should be amended to permit the marketing of drugs for any life-threatening or seriously debilitating disease using similar criteria used to permit drugs for HIV infection to be distributed under Parallel Track."

The bill then directs the FDA to approve "a drug or biologic needed to treat or prevent a life-threatening disease or seriously debilitating illness (including AIDS, cancer, Alzheimer's disease, cardiovascular diseases, and Parkinson's disease)" if nine conditions are met. These require reasonable safety and promising evidence of efficacy, no satisfactory alternative treatment, and ongoing research by the drug's sponsor, including one or more clinical trials to test for efficacy, and in addition, special monitoring for adverse effects in patients who purchase the drug. H. R. 2872 also requires that drugs approved under its provisions be available only by prescription.

In other provisions, H. R. 2872:

* Gives the FDA 120 days to review an application for expedited approval; if the FDA does not act within 120 days from submission of the completed application, it is automatically approved;

* Forbids public and private health-insurance plans from distinguishing between these drugs and other FDA-approved drugs for reimbursement purposes;

* Requires withdrawal of approval if two or more post- approval studies fail to confirm the safety and efficacy information on which the approval was based; and

* Requires written informed consent of the patient or patient's representative.

History of H. R. 2872

H. R. 2872 was first drafted by Jim Driscoll, an AIDS activist who is president of the PATH Foundation and a member of ACT UP/Golden Gate. Driscoll worked with the staff of Representative Campbell's office to improve the draft and express it in the legal language and format appropriate for a new bill.

Driscoll and others have continued to build support for the bill. At this time 35 members of Congress have signed on as cosponsors -- and most of them are Democrats, despite the fact that the bill was introduced by a Republican. The cosponsors cover the entire ideological spectrum, from the most liberal to the most conservative. With this level of support, H. B. 2872 may be crossing the threshold from an "idea" bill to serious legislation.

Issues Behind the Controversy

Few persons or organizations have yet taken positions on H. R. 2872, and many of the arguments on both sides have not been expressed in writing. For this reason -- and also to focus on the issues involved, instead of the personalities -- we chose to write our best understanding of the case on each side, based on conversations we have had with advocates of each. The arguments below do not always represent our own position; for our view, see the Comment section. (We hope that anyone quoting from the "for" or "against" sections of this article, below, will make it clear that we are summarizing the arguments on both sides, not taking our own position on the bill.)

The two central disputes seem to be:

* If an experimental drug (for example, ddI or ddC) is urgently needed, and already has enough evidence for safety and efficacy to justify distribution to thousands of people (but not enough to justify full approval under today's system), should the FDA approve that drug for marketing through regular channels, or only for free distribution under expanded-access arrangements like those for ddI or ddC? (The central purpose of the Campbell bill is to allow such marketing.)

* Is it tactically wise to seek drug-approval reform through Congress at this time, or would it be better to wait for FDA Commissioner David Kessler to implement such reforms administratively?

The Case For the Bill

The U. S. drug-approval system is the most severe in the world, requiring years of development and well over $100,000,000 per new drug approved. Because of the delays, many patients with life-threatening conditions are denied treatments which are clearly their best or only hope. To relieve this intolerable situation, the FDA has devised a series of programs, with names like "compassionate use," "treatment IND," and "parallel track," to allow early access to critically needed drugs which are not ready for general marketing approval. The reason there are so many different programs is that none of them met the need very well; as each one fails, a new one is devised to replace it.

The problem with all of these expanded-access programs is that the company which owns the drug has little or no incentive to agree to provide it -- and does have reasons not to, since these programs can be quite expensive to administer. With AIDS drugs at least, the pattern we have seen again and again is that companies first flatly refuse to provide the drug. Under great public pressure they may relent, but then they use unnecessary medical requirements and burdensome paperwork to limit participation; many physicians refuse to enroll their patients because of this burden, and public clinics almost never provide these drugs, because their hurried physicians do not have time for the paperwork. Another barrier to participation is that patients may have to pay for special laboratory tests out of their own pockets; insurance programs usually refuse to pay on the ground that the treatment is "experimental." These restrictions, imposed by the pharmaceutical companies largely to save themselves money, make "expanded access" distribution even less equitable than standard marketing of drugs, even more restricted to those with private physicians and their own financial resources to pay for care.

After activists have generated the public pressure to gain initial access to a drug, they have to generate another pressure campaign to get the restrictions relieved to the point that the program is usable. But some drugs, for example peptide T, are being developed by small companies which could not possibly afford a large expanded-access program, so these drugs will never be available through expanded access.

And now we are beginning to see another problem -- less visible than the ones above, but worse. It appears that companies are beginning to conceal important advances, even to the point of not doing the research which could lead to demands for a large expanded-access program, often considered a costly corporate nightmare.

Worse yet, expanded access may poison the process by which critically important new medicines are developed, by creating a disincentive for developing life-saving therapeutic advances, thus shifting pharmaceutical research efforts toward producing copycat drugs -- projects which are safe, can be profitable, and will never lead to a demand for expanded access. This disincentive extends all the way back to preclinical development and even to basic research, changing the climate in which innumerable internal corporate battles are won or lost. No one will ever know what critical treatments never came into being, and who died needlessly as a result.

The Campbell bill corrects these problems by turning the demand for early access to life-saving drugs from a corporate nightmare into a positive incentive. Instead of punishing companies that develop important advances, it rewards them by making it easier to get to market with a major advance which is important to human beings than with a safe, copycat drug. And yet it does this with no compromise to human health and safety, since it only applies only to drugs which are safe and promising enough to be distributed to thousands of people under current standards, even without H. R. 2872.

Today we are in the middle of a revolution in biotechnology, a revolution representing one of the most important scientific advances of all time. In the near future it will become possible to develop new drugs and other treatments in vastly faster and more efficient ways than could be done in the past. Yet today's drug-approval system was designed 30 years ago, in the early 1960s; and the natural accretions of bureaucracy have left it more cumbersome now than it was then.

The Campbell bill will be critically important even if it never becomes law. For it sheds new light on what is shaping up to be one of the major debates of our time. On one side are the forces opposed to change because they benefit from the current system. These include big companies that want to protect their markets by keeping small companies out, investors and financial analysts who appreciate the predictability of being able to see new drugs in the pipeline years before they reach the market, and consumer protectionists understandably fearful of losing ground in their ongoing war against corporate abuse. Also, the current price estimated at $100,000,000 to $200,000,000 or more for each new drug approved is all paid to someone -- mostly to professionals and associated businesses, who therefore have much incentive to maintain the current system. Until recently it was very difficult to organize a constituency for reform, because almost everyone close enough to the drug-approval system to understand it also made money from it.

But now, on the side of change, a new "triple alliance" is coming into view:

* First, the AIDS, cancer, Alzheimer's, and other grassroots activists have a common interest in access to (and better and faster development of) life-saving treatments. The future of AIDS activism is with this alliance, since many people believe that AIDS does not concern them, but everyone knows that they could get cancer or another deadly disease, and their life may depend on access to new treatments. So the alliance includes not only all the disease groups, but everyone.

* Second, there is great interest today in reducing the cost of medical care. The best way to reduce cost without reducing quality is to improve technology. The Campbell bill would reduce the cost of developing the most important new drugs, thereby reducing pressure on prices. In addition, by allowing more drugs to be developed, it would further reduce prices through increased competition. And perhaps most importantly, it would reduce costs by speeding the process of supplanting poor medical technologies with better ones.

* Third, this alliance can further expand to include those concerned U. S. industrial efficiency and competitiveness. Biotechnology has the potential to become the most important area of U. S. scientific and industrial leadership. By imposing the 1960s system of drug approval -- a system found nowhere else in the world -- on the technology of the 1990s and beyond, we will seriously damage a vital national resource and harm this country's future.

The AIDS treatment movement can and should help to build this alliance for reform.

The Case Against H. R. 2872

The issue is not whether reform is needed, but whether this legislation now is the best way to get it.

FDA Commissioner Kessler very much wants to speed critical drug approvals, and is already preparing a plan to do so -- a plan based on new regulations (or on a policy statement) under existing law, not new legislation from Congress. Most experts believe that the FDA already has the power to do what the Campbell bill would ask it to do. Why, then, is new legislation necessary? Most of the arguments in favor of the bill, above, could just as well be used in favor of Kessler's proposal.

And if legislation is unnecessary, there are many reasons not to seek it:

* Legislation takes time. The FDA can act -- and is ready to act -- much more quickly on its own.

* Even introducing legislation can impede FDA reform, as opponents can argue that the FDA should wait to see what Congress does before taking its own action. Furthermore, if the Campbell bill is defeated -- and the great majority of bills introduced in Congress never become law -- then the FDA may be unable to implement its own reforms, since Congress will be seen as having rejected the concept of "weakening" the efficacy standard to allow faster access to life-threatening therapies. [The bill's advocates answer that congressional opponents of FDA reform could kill regulations more easily than they could kill a bill, which would not get to them until other members of Congress had already given their support.]

* The way this bill was introduced was, in several ways, not politically astute. It was introduced by a Republican, when the Democrats control Congress; the bill should have been bipartisan. (This argument was made before the bill had a majority of Democrats as sponsors, as it does today.) Democrats are likely to consider it a Republican deregulation scheme -- not popular these days. Few members of Congress will study the bill on its merits; most will follow their leadership and vote against it. And it doesn't help that the bill was drafted by an AIDS activist, Jim Driscoll, who twice attacked the leading Democrat on health issues -- Representative Henry Waxman of Los Angeles -- in two op ed pieces in the Wall Street Journal (March 6, 1991, and June 20, 1991). Waxman is exceedingly important to the AIDS community in restraining the senior Republican on health issues in the House of Representatives -- Representative William Dannemeyer of Orange County, California, who is obsessed with homosexuality (he has authored a book about it -- Shadow in the Land: Homosexuality in America, published in 1989) and has impeded constructive national response to AIDS. Dannemeyer, ironically, might be supportive on treatment-access issues; but the chance of an alliance between him and the AIDS community is zero.

* Another political problem with this particular bill is that it was created by a single activist who then found a member of Congress to introduce it. Few AIDS activists knew about the bill before it was introduced, so they had no chance for input or to have their concerns considered.

* Despite the good intent behind H. R. 2872, the fact remains that the drug-approval law of the United States affects the flow of billions of dollars. Every word of the current law has been litigated; almost three decades of legal precedents have been established. Any change in the current law will make some of these precedents uncertain, unleashing new litigation which itself could harm drug development; change could also upset boundaries and relationships which have developed over the years between major companies and other powerful interests and institutions. The pharmaceutical industry, the insurance industry, and others will certainly come forward while the bill is going through Congress -- if only to defend their technical interests, which the public, the AIDS community, and even the drafters of H. R. 2872 do not understand. No one knows what will come out of this process, how the bill may be changed before becoming law.

Why invoke the legislative process when it is unnecessary, and before we have examined the realities involved or even worked out consensus among ourselves about what we want?

* It has been argued that we need legislation because we cannot trust the FDA; administrative reform can easily be overturned by later officials. But administrative flexibility is necessary so that rules can change as situations change. When Congress plays doctor it does a terrible job of it, as we have recently seen; should Congress now play scientist also?

The right way to achieve reform is to change the professional and public consensus about what is right, and what is wanted, in drug development. A new consensus will guard against arbitrary changes by bureaucrats, giving us the permanence we want -- but no more.

* Another problem is that the bill could create an incentive for sham research. Companies would win approval based on early data, provided they agreed to continue studies. But if two studies fail to support the early approval, the approval would be withdrawn; therefore, it may well be in companies' interest to never finish their studies, or to delay them as long as possible. In addition, there could be a special incentive to price gouge for dubious drugs, since they would have only a short market life until their drawbacks became known.

* The insurance provision in H. R. 2872 -- that companies cannot distinguish drugs approved under its provisions from other FDA-approved drugs -- is likely to be thrown out by the courts, leaving the individual to pay the whole cost burden.

(The bill's proponents might answer the last two objections by saying that a reform to speed access to life-saving new treatments should not be expected to simultaneously solve the underlying problems of the U. S. medical system, such as price gouging and insurance flight. The way to stop price gouging is hardly to keep new drugs from being approved. The FDA, and the medical community -- both inherently skeptical -- will serve as barriers against scams. And if there are problems in the current draft H. R. 2872, it can easily be changed as the bill moves through the legislative process.)

Comment

While both sides of the controversy have persuasive points, we feel that the dispute itself is unfortunate. This bill could help to build coalitions between AIDS groups, other disease groups, and the public as a whole. The lack of consensus in the AIDS community has impeded this progress.

The bill's supporters have gone to Congressional offices never before approached about AIDS, let alone about treatment issues, and have found a very encouraging response; for example, some of the 35 current House cosponsors were not approached by anybody, but heard about the bill on their own, read it, and signed on. This good reception in Congress for H. R. 2872 shows the great potential for broadening the AIDS appeal beyond its customary focus on the rights and needs of those with HIV, to also address issues which speak to everybody. Drug-approval reform could save anybody's life; if properly explained, it is an issue anyone can understand. And anyone can understand that what the AIDS community has learned on this matter could someday translate into a critical benefit for them.

Drug-approval reform therefore provides an unprecedented issue where the work of AIDS activists could save the lives of those who choose to think that AIDS is not their problem. In one stroke it rescues AIDS from being seen as special pleading, and instead begins building a new mainstream around the central, universal concerns of life and death, the widespread distrust of bureaucracy, and the fear of abandonment in adversity if the medicine you need is not available.

The current controversy is unnecessary because the universal message, from all sides, is that we need regulatory reform to speed access to critically important new drugs. This basic demand for change does not specify a "legislative" or an "administrative" solution. It is far easier to organize public support around legislation in Congress than around an internal FDA proposal not even released to the public yet; this is why attention now focuses on the Campbell bill, as it should. But ultimately, policy experts may decide that administrative reform offers a faster, cleaner, and more flexible route to the goal. That is legitimate, too.

Whether or not the Campbell bill ever becomes law (and whether or not it ever should) it will make an enormous contribution if it helps to show a way out from the current national political paralysis on AIDS.

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