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Executive Summary

New drug research in a cost sensitive health system could lead to development of higher safety/lower efficacy agents intended specifically for home use, Bristol-Myers suggested in a recent meeting with FDA Com. Young and General Counsel Tom Scarlett. In a prepared presentation, the company stated that "we are on the threshold of a new era in American medicine -- thrust upon us by the necessity for health cost containment. New factors now come into play in guiding new drug development. No longer can we allow route and frequency of administration to be low priority items in our drug development wish list. Safety and efficacy will have to share the spotlight with ease and infrequency of administration, as well as with rapidity of effect." The firm suggested that under a health system that emphasizes cost containment, the objectives of drug research will be shifted to the development of agents which reduce cost rather than those which represent therapeutic breakthroughs. Maintaining that such a shift in research goals will have an impact on the approval process, the company explained in its prepared statement: "Currently our mission is clear. That is, we look for new drugs that are significant improvements over existing drugs both by way of efficacy as well as safety. Perhaps we should now be starting to look for drugs that possess that constellation of characteristics which would make them ideal for home care use." The firm continued that "today we would usually not accept significantly less efficacy along with greater safety. However, this might be exactly the kind of drug that would be useful in home care." The growth in home health care "might lead to a new class of drugs that are preferred for use in this setting," the Bristol-Myers presentation noted, "even though they would not be recommended for either outpatient use or hospital use." If new drug products in the future are developed on the basis of cost concerns, FDA's regulatory review standards, currently based on safety and efficacy considerations alone, may have to evolve along with drug research programs, Bristol-Myers suggested. For example, "can we expect to see in a package insert of the future, in the indications section, that a new drug is indicated for the treatment of disease in the home bound patient?" From the point of view "of the practicing physician, the type of monitoring available on the home bound patient might very dictate the kind of therapy that would be utilized," the company said. "With good monitoring and competent nursing care, the home bound patient might be treated almost the same as a hospitalized patient. To what extent could we expect FDA to become involved in these types of decisions?" The statement notes that "traditionally, FDA has stood back from any implication that they might be telling the physician how to practice medicine. However, as times change, so do obligations, and perhaps FDA and the industry will have to make this kind of recommendation." The answers to such questions, the execs said, are "essential for our guidance in current new drug development. Afterall, the drugs that we are just starting with now will be the drugs in widespread use in the middle 1990's." The issue of future drug development in the context of cost containment is one to which FDA Com. Young may be particularly sensitive. Young came to the agency last July saying that his charge from HHS Secty. Hickler was to "prepare the FDA for the 21st Century." His major project has been the development of an action plan to guide the agency in the coming years. The issues raised by the Bristol-Myers presentation are the sort which may be addressed in that action plan, reportedly near to public release. Marketing Upon Receipt Of "Approvable" Letter Should Be Permitted If Firms Agree To FDA Conditions, Bristol-Myers Says The Bristol-Myers representatives met with Young and Scarlett April 24 at the company's request to discuss issues relating to the new drug approval process. A text of the company's presentation was included in FDA's memo of the meeting. In addition to Exec VP William Miller, company representatives included Senior VP Abramo Virgilio; VPs Giulio Vito and Dan Couglin; VP-Medical Affairs, Pharmaceutical R&D Div. Erwin Whitman; VP and Associate General Counsel Rodolphe Hamel; and VP-Govt. Affairs Bill Greif. The firm maintained that another way that drug development will be influenced by cost pressures is in the use of hospitals as locations for clinical trials. The exec's presentation said that several investigators have been told by hospitals that "under the prospective payment system of DRG's [Diagnosis-Related Groups] the hospitals will not be reimbursed for that portion of the patients" involved in research. In order to defer the increasing costs of clinical research, the company suggested "perhaps the FDA could help somewhat by accepting, under specified conditions, the treatment of the home bound patient as equivalent to that of the hospitalized patient." Bristol-Myers made several specific recommendations for improving the drug approval process. VP Erwin Whitman suggested that FDA reviewers could work more closely with NDA sponsors in reviewing drug applications. Whitman recommended that "when an NDA is submitted to the agency, the sponsor be permitted to send along with the NDA to the medical reviewing officer a representative of the sponsor who really knows the NDA." Whitman said the sponsor's representative "could then spend as much time as is required with the reviewing officer during the first few days and weeks, to go over the entire NDA, and to much more rapidly familiarize the reviewing officer with the NDA, its content, how it is put together, how it's indexed, and so on." He added that later in the review process the FDA reviewing officer could invite the sponsor's representative to periodic meetings to discuss the status of the review. "It is our suggestion that these meetings and subsequent periodic meetings be made a practice and policy of the FDA," Whitman asserted. He said such practice would cut down on phone calls and visits from the sponsor to FDA and likely "result in quicker and cleaner answers to any questions the reviewing officer might have." Whitman said if any deficiencies are found in the study during the review, the sponsor could complete the work to answer the deficiencies before review of the application is completed. Bristol-Myers currently has four NDAs pending: the anxiolytic Buspar (buspirone); the anti-arrhythmic Enkaid (encainide); the analgesic Stadol (butorphanol); and butorphanol with acetaminophen. Three additional drugs are in Phase III trials: the antihypertensive bucindolol; the anti-cancer cisplatin analog carboplatin; and the anti-allergy agent tiprinast. With respect to the IND phase of the approval process, Whitman suggested that agreements reached between the agency and sponsor at an end of Phase II meeting should be binding. "Just as there are various points at which data are locked in, I would like to see the plans made at the end of Phase II meeting similar locked in," Whitman stated. Guideline Revisions Through Joint Industry, FDA, And Academic Review Should Be Made Periodically, Firm Says He noted that at an April 11 meeting between FDA and members of the Pharmaceutical Mfrs. Assn., FDA Office of Drug Research & Review Director Robert Temple, MD, commented that only about 10-20% of NDA applicants take advantage of the opportunity for a conference at the end of Phase II trials. Whitman suggested that more sponsors would request such meetings if they were binding. Commenting on the role of advisory cmtes. in the approval process, Whitman recommended that if no action is taken by FDA on an official advisory cmte. recommendation "within perhaps 180 days" the matter should be "automatically brought up again before the advisory cmte. to determine why action has not been taken." He said the process "could be repeated at six month intervals until the matter is settled." Whitman also objected to FDA's conflict of interest policy that precludes cmte. members who have previously worked with a drug from voting on any questions about it at cmte. meetings. In further comments, Whitman said Bristol-Myers would like to see guidelines "clearly labelled as providing those criteria that the agency feels are necessary to achieve a designated goal" and "subjected to periodic review and revision, involving the agency, industry and academe." Whitman added that "some people have even suggested that guidelines be subjected to some type of sunset regulation, which indicates the date of the termination of a given guideline, thereby requiring the issuance of a new guideline by that particular date." Whitman also said the company would like to see the standardization from division-to-division and reviewer-to-reviewer of such things as carcinogenicity testing and inclusion criteria for patient admission into studies. "We recognize that if the sponsor disagrees with the marketing conditions specified by the agency in the approvable letter, there will be meetings and discussions, and consequent delays in the final approval for marketing. However, what if the sponsor decides to accept all of the agency's conditions contained in the approvable letter? Should it not then be possible automatically to allow the sponsor to market the drug upon receipt by the agency of a letter to that effect from the sponsor? This would eliminate the substantial delay that can occur between issuance of approvable and approval letters."

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