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W A S H I N G T O N R E P O R T By Jill Wechsler . Challenging FDA Authority THE USER FEE PROGRAM is forcing Food & Dru; Adminis- tration (FDA) officials to rethink the way they do business,just as a leQal challenge may finally prompt the agency to reevaluate its restrictions on industry conm1 unications. Meanwhile, congressional action threatens continued growth of the orphan drug program. Those situations raise questions about how much FDA regulation industry really needs to ensure quality health care. The main effect of user fees will be to shift FDA's "efforts ori- entation" to an "outcomes orientation," observed Center for Drug Evaluation and Research (CDER) deputy director MtuTay Lump- kin at the June annual meetinR of the Drug Information Association. In the past it was good enough for the agency to "try hard" to accomplish its tasks, he explained; it could "pop oft" and tell an applicant to choose between FDA doing it rivht-or on time. Now FDA will have to meet specific timetables-to -get it ri;ht and on time. Lumpkin emphasized. To meet specific deadlines for pro- cessiniZ submissions to the agency, the reQulators find themselves busy setting up new information systems and espousing all kinds of "2ood management" jargon in an effort to become more effi- cient and absorb an expandina work force. It remains to be seen, I F-It remains to be seen whether FDA will be ~ble to increase productivity and ensure ~valid decision »zaking, or whether the process will become so complex that it W• iscourages innovation. though, whether the chanRe in culture at FDA will really be able to increase productivity and ensure valid decision making, or whether the process will become so complex that it discourages innovation. Leading the campaign for improved management systems at FDA is Deputy Commissioner Mary Jo Veverka, who is busy devel- oping a strategic plan and articulating a clear sense of "vision" for the entire agency. She emphasizes the need to maintain strong sci- ence, provide fair enforcement, develop staff, and improve effi- ciencv throuQh new information systems. To accomplish those lofty nals. Veverka seeks to encourage more interaction among FDA's various centers by implementing a series of "cross-cutting strate- gies" and "best practices." The aim is to sain more consistency in review and enforcement activities throuRhout the agency-as well as diminish the power of separate fiefdoms that traditionally have controlled the aSency. FDA now has several intercenter workinR I8 7HpqMnC€U'nCAL EXECUTIVE AUGUST 1994 c7roups to collaborate on application reviews and share information in areas such as oncolo"7y, wound healin,, and nuclear medicine. More cooperative programs are planned, some with outside organi- zations such as the National Cancer Institute. Most of the new manazement activity is taking place in CDER and the Center for Biolooics Evaluation and Research (CBER), which now have to meet specific performance targets. The main ini- tiatives include: "Refuse to file" (RTF) policy. FDA centers now eliminate from the queue those applications that lack obviously important elements related to testino and manufactutin-. So far, CDER is experiencing about a 20 percent refuse-to-file rate on new-drug applications (NDAs), althouah that excludes those applications that are with- drawn before being counted as RTFs. Cohort tracking systems. To be able to report exactly how much time it spends processing each application, FDA has beQun to group all applications received during each fiscal year into "cohorts." The 1993 cohort, for example, includes all NDAs and supplements filed with CDER from f October 1992 to 30 Septem- ber 1993. One challenge for CDER is making sure it keeps track of the "cauRht Qeneration" so it doesn't become the "lost Qeneration: " That refers to acroup of more than 100 applications that were filed before the user fee program was enacted in September 1992, but were not yet sufficiently overdue to be included in the baclJoa as of 1 Octo- ber 1992. Lumpkin reports that CDER has been fairly successful in whittling down the 106 NDAs originally in the group to 21 still pending. as well as eliminating most of the supplements "caught" by the timinQ of the new law. Project management. To move the cohorts through the review process on time, CDER has established management teams to coor- dinate review activities. Four of 10 CDER new-drug review offic < now have teams that set a 45-day meeting after the NDA is filed t' determine if CDER should file it and coordinate the primary anLi secondary reviews so that they can run concurrently. Another objec- tive is to ensure "rapid closure" on labeling issues. Good review practices guidelines. As part of the effort to improve the consistency of the review process, working groups in CDER are preparing documents to infonn new medical reviewer> and other professional staffers of the best methods for conductina " review. With a staff of 1,400 in CDER, including 1,000 reviewer`• chemists, statisticians, and other professionals, it is difficult to set policies for all reviewers to follow-particularly when CDER I` under pressure to hire and train more review staff quickly. The trici will be to set standards, but not make reviewers feel hamstn n' lt` a preset checlJist. Coordinating committees. CDER has also established of panels that aim to encourage more interaction and sharin~' new-drua review divisions. There are coordinating committee"

' i research, pharmacology/toxicology, medical policy, and chemistry, more re2ulation of new dru,s under health reform, the proposed manufacturing, and controls sections of NDAs, amonQ others. bill is unlikely to rev up interest in this area. It is unfortunate because Senate approval is expected to prompt action in the House. • PEOPLE PROBLEMS A major obstacle to implementing all those management initiatives is a shortaRe of trained personnel to evaluate applications. CDER and CBER are supposed to expand review staffs by about 600 to be able to meet the user fee review timetable, but hirina freezes and Qovernment downsizing programs have hampered them. The user fee program has been exempted from hiring freezes, but FDA still faces a mandate to cut its overall work force by several hun- dred people in the coming year, So even if CDER and CBER were able to persuade 300 or 500 professionals to accept jobs with the agency, their atrival would require cutbacks in other FDA offices. There's a lot of bud2et legerdemain controlling the process, which has forced some FDA offices to steal staffers from others. As a result, the net -ain to CDER has been only 77 professionals and support staff as of the end of May. To make matters worse, FDA is still fightinQ for an exemption from White House policy to cut senior staffer positions throughout the federal bureaucracy. In addition. the a-ency's budget for fiscal year 1995 is short of the amount needed to support a taroet of 9,700 staffers. Consequently, FDA has adopted a "modified" hirinn freeze that allows it to hire one new staffer for every three that leave. One reason for that defensive move is that FDA officials expect the final budget approved by Congress to be less than what the House has proposed. ABANDONING ORPHANS AlthouRh there is heavy pressure on all sides to boost FDA's user fee collections, some parties want to make an exception for orphan dnies. The aim is to encourage industry investment in orphans, sim- ilar to the objective underlyina separate le`islation movina throueh Congress. In May, the Senate Labor & Human Resources Com- mittee approved a bill to amend the Orphan DruQ Act by a wide margin after years of wrangling over ways to curb high-priced orphan products. Some senators also thouQht that a reform bill would make a nice going-away present for its diain advocate, retir- ina Sen. Howard Metzenbaum (D., Ohio). Industry and consumer advocates aoreed on the compromise measure, even thouch it threatens to reduce incentives for orphan druc, development• The main problem is that the measure cuts the seven-year period of exclusivity for orphans to four years. Manufacturers can renew for the additional three years' exclusivity if the product still has "limited commercial potential." such as a small-less than 200,000-patient population and limited revenues. The Biotech- nology Industry Organization backed the proposal, largely because it Rrandfathers all orphans now on the market or in the pipeline, includinQ the handful of real orphan blockbusters that generated the criticism of the program. National OrQanization for Rare Disor- ders executive director Abbey Meyers supported the agreement because she fears that penny-pinching managed care plans will refuse to cover very expensive orphans. She also believes that the provision in the bill that allows competitor orphans to enter the mar- ket when developed simultaneously will keep orphan prices down. What's more likely to happen is that a lot of drug producers won't bother with orphan druSs at all. Most pharmaceutical comr panies already have abandoned the orphan field. Sen. Christoper Dodd (D.. Connecticut) commented at a committee session. Sen. Judd GreQQ (R., New Hampshire) argued that FDA will tind it impossible to determine which orphans are sufficiently without commercial value to wan•ant extended exclusivity. Althouah it•s likely that the recent decline in orphan applications may be because of the uncenainty, from legislative stalemate plus the threat of e\ en 20 PHARMACEUTIC6L E%ECUTIVE AUGUST 1994 THE SAGA CONTINUES 1Vhile conaressional tampering may actually weaken the orphan druR program, FDA policies are killing off industry-sponsored med- ical and scientific activities. Now it looks like the courts may have an opportunity to review the constitutionality of FDA regulation of communication on pharmaceutical products. The sorry saga of FDA's efforts to reaulate continuina medical education (CNfE) pro- grams has been playing out since David Kessler became FDA com- missioner and made reform of scientific and educational programs a personal crusade. Instead of tryinR to clear up unethical activities, though. Kessler decided lie had the authority to prevent anv manu- facturer involvement in even bona fide educational activities if they involved a discussion of off-label dru, use. First came the "Con- cept Paper" that tried to "carve out" a"safe harbor" by allowing companies to fund CME through third-party providers. The outcry was so loud that FDA revised it and issued its "Draft Policy." which continues to discoura,e industty support for CME. While congressional tampering may -actually weaken tlte orplaara drug progranr, W ~ DA policies are killing off indust~y- ~sponsored medical and scientific activities. ThrouRhout the debate, some critics complained tha: FO-1's efforts to control scientific and educational discussion % the First Amendment right to free speech. No drugmaker ~~a, ~% i!ling to challenae FDA on the issue, thou2h, and nothing happened until the WashinQton Legal Foundation (WLF), a consen•at \ e puhlic interest law firm, filed a petition last October. It charccl tr;a the aoency's proposed CME policy and similar curbs on gn n; do~ • tors enduring materials such as textbooks and journal, c.: rc ird the agency's statutory authority and violated individual nch: : u.u anteed under the First Amendment. That action brouch• i~ FDA's plan to issue a final policy on CME by the end of I u aQency officials acknowledQed a need to respond serio,:~,% t~' = issues. To support the petition, a number of media and mx, r:.n; orsanizations and some free-speech groups, including tthe \le.: .. Institute, the Association of American Publishers. the S ci:•:, , Professional Journalists, and the Coalition for Health Carr C ummunication. filed comments. Even thoush FDA officials made a lot of noise about addres~ui_' the petition, when the agency failed to reply officially to it after the allotted 180 days had run out, WLF filed a lawsuit in the li.S. Di>• trict Court in June. It souQht an injunction asainst FDA'> propo`cd policy to limit scientific communication, but also asked the court to require FDA to adopt a new policy to "dissipate the chilling et(cct on First Amendment rishts" of the current proposal. \vLF \~ent to court because it feared that FDA might never respond to it; com- plaint, yet would continue to enforce its proposed policy Witho~~t ever issuing a final rule. FDA is expected to seek outriQht di~mi >; l but WLF hopes the judge will ask the aovernment to at least addlc" the issues raised in the petition and the lawsuit by some sp«il •' date. Guiiinul -1 S

Y WLF's filinos are noteworthy because they raise a larger ques- That trend away from CME support is likely to continue, even tion about whether the federal government in general, and FDA in if FDA modifies or abandons its proposed rules. With managed panicular, should regulate communication by medical profession- care oreanizations (MCOs) emerging as the prime force in health als and druia manufacturers unless the infornlation is false and mis- care delivei~•i ditt_ manufacturers are decreasing efforts to market leading. FDA. obviously. has taken a broader view. claiming that to individual physicians. Instead of running ads in medical jour- it has authority to require manufacturers to prove that any use of a nals and sendinR out thousands of sales reps to make calls on doc- medication is safe and effective based on objective supporting data. tors. companies are spending more time and resources on broadcast communications, satellite hookups, computer information systems, CME CONSTITUTIONALITY and telemarketin-. At the same time they are directing more pro- AlthouEh it's interesting to examine constitutional interpretations of motional efforts directly to consumers, who are playina a greater the law, the really important issue on a practical level is what poli role in making treatment decisions. [See "Reach the People;" July cy or process is most likely to keep physicians and scientists best 1993.] informed about new therapies and medical technoloaies so that they The transformation of the U.S. health delivery system, coupled with a revolution in communications and information technology, presents FDA with an opportunity to rethink its traditional role in re~TulatinQ information about druEIs. In the past, it has argued that ~ ''Tl2e tl•a12Sfo1722at1o12 of the U S. health individual doctors could not possibly have enou,h expertise to eval- ~ ~ .._._ .1__ ___ .. ___.1 ___....__.. l1G U~IIC 111G UlUlllt~' alilu LLI.I.Ultll.~' VI l.lalllll.l 11V111 IIILL1[~GI~.IJ, WIIV del2Vely SyStem prese~2ts FDA fvith a22 really just want to increase sales. Therefore, the government had O~J~lortil122ty to 2•etl2U2~{ its traditional role 212 to check everythmg. demand supportin- studies, and evaluate the likelihood of bias and unfair influences. r' 1'ea2LlatU2a222for122atI0J2 avoilt d1•IIQS. With MCOs providing health care to a predicted 80 percent of the population within five years. doctors will be following fonnu- ,.. :_ lary recommendations, clinical practice ,uidelines, and advice from in-house "detailers." In addition, physicians will be able to tap on- line computer systems that can answer questions about comparative can provide the best patient care. So far, the main result of FDA's claims, druR interactions, and costs and benefits. With all that infor- proposals to restrict CiE is that many drug marketers have stopped mation on medical technology available to both doctors and tmderwritin- those educational pro.orams. They've also stopped patients, do we need an FDA that sees its mission as blocking any distributin2journals and medical literature, according to major pub- kind of communication that doesn't fit its rules, or an agency that ~ lishers, because of FDA threats. will promote infonned discussion of medical advances? I ...to step up communications with doctors and patients? ...to provide more patient education and compliance programs? ...to magnify the impact of your sales force? ...to offer value-added services for the managed care audience? Biospherics can help you get ready with cost effective toll free health telecom77iunications programs. Call 1-800-727-0602 for more information. • 22 o,veRlf<ePUnee1 £xHCUTIVE AUGUST 1994