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The Fda and Ophthalmic Therapeutics
VOL. 80, NO. 2
305
EDITORIALS
T H E FDA AND O P H T H A L M I C THERAPEUTICS In recent years, many of us interested in progress in ophthalmic therapeutics have been pessimistic about the future. To clear a new ophthalmic medication through all the studies required by the Food and Drug Administration a company must invest an estimated $500,000 to $1,000,000, and be prepared to spend a minimum of two to three years once they have been convinced of safety and efficacy to complete the studies and the review processes required by this organization. For those with uncommon dis eases, the outlook seemed grim. Unless a large market could be generated to repay the manufacturer for the enormous cost of the studies and the risks involved with tre mendous delays and the uncertain approval, areas such as ophthalmology in which the market was limited, were bound to suffer. We saw many examples of this. Drugs such as trifluorothymidine, which are superior to idoxuridine in the treatment of herpes sim plex keratitis, have been available on an experimental basis for 11 years and have been shown by controlled double-blind stud ies to be safe and more effective than idoxuridine. Until recently, no company could afford the large cost of producing them, nor was any company sufficiently altruistic to endure the enormous losses re quired to make them available on a charita ble basis. The therapeutic use of soft con tact lenses required six years to be approved. Fungus keratitis, which is ineffectively treated by most of the presently available antifungals, could be better treated by pimaricin, a drug unquestionably safe and effective in the treatment of topical fungus disease and available in Europe for more than a decade. Its efficacy was confirmed in this country by numerous studies, but no manufacturer could afford the costs and aggravations of making available a drug that might be used for as few as 200 to 300 people a year. It seemed as if this blinding and debilitating, but unpopular disease might
remain untreatable by most ophthalmolo gists, even though treatment of proven su periority was available. It may be that at least some of these fears have been unjustified, and that the FDA, in seeking to meet its mandate to the people of this country, realizes that sins of omission, that is, blindness from untreated or poorly treated disease, may be just as serious as sins of commission, that is, the possibility a drug with some risk factors might be re leased. Those of us interested in thera peutics have sympathized with the plight of the regulators who are faced with constant criticism by laymen of influence and posi tion. It is easy for these critics to point at possible side effects of drugs that have been released, but there has been relatively little consideration for those who may have been maimed or injured by diseases that could better be treated if medications were made available more promptly and expeditiously, and if some mechanism could be found to make the process of approval more rapid and less expensive. A landmark development has recently oc curred in the field of ophthalmology. The FDA has taken the information available on pimaricin from the studies done in this country and abroad, and, through internal motivation and the use of its advisory com mittees has approved this drug for manu facture. It will be made available to oph thalmologists, even though no significant profit is likely ever to be generated from its sale, and this ability to treat patients with fungus keratitis has been made possible through FDA recognition of the need, and a realization that some special consideration must be given to the treatment of less com mon but important diseases. It seems that there is almost constant criticism of the FDA in the press and the Congress. I think it is time that we in oph thalmology praised them for an action that will greatly benefit our patients and may set an important precedent. HERBERT E.
KAUFMAN
305
EDITORIALS
T H E FDA AND O P H T H A L M I C THERAPEUTICS In recent years, many of us interested in progress in ophthalmic therapeutics have been pessimistic about the future. To clear a new ophthalmic medication through all the studies required by the Food and Drug Administration a company must invest an estimated $500,000 to $1,000,000, and be prepared to spend a minimum of two to three years once they have been convinced of safety and efficacy to complete the studies and the review processes required by this organization. For those with uncommon dis eases, the outlook seemed grim. Unless a large market could be generated to repay the manufacturer for the enormous cost of the studies and the risks involved with tre mendous delays and the uncertain approval, areas such as ophthalmology in which the market was limited, were bound to suffer. We saw many examples of this. Drugs such as trifluorothymidine, which are superior to idoxuridine in the treatment of herpes sim plex keratitis, have been available on an experimental basis for 11 years and have been shown by controlled double-blind stud ies to be safe and more effective than idoxuridine. Until recently, no company could afford the large cost of producing them, nor was any company sufficiently altruistic to endure the enormous losses re quired to make them available on a charita ble basis. The therapeutic use of soft con tact lenses required six years to be approved. Fungus keratitis, which is ineffectively treated by most of the presently available antifungals, could be better treated by pimaricin, a drug unquestionably safe and effective in the treatment of topical fungus disease and available in Europe for more than a decade. Its efficacy was confirmed in this country by numerous studies, but no manufacturer could afford the costs and aggravations of making available a drug that might be used for as few as 200 to 300 people a year. It seemed as if this blinding and debilitating, but unpopular disease might
remain untreatable by most ophthalmolo gists, even though treatment of proven su periority was available. It may be that at least some of these fears have been unjustified, and that the FDA, in seeking to meet its mandate to the people of this country, realizes that sins of omission, that is, blindness from untreated or poorly treated disease, may be just as serious as sins of commission, that is, the possibility a drug with some risk factors might be re leased. Those of us interested in thera peutics have sympathized with the plight of the regulators who are faced with constant criticism by laymen of influence and posi tion. It is easy for these critics to point at possible side effects of drugs that have been released, but there has been relatively little consideration for those who may have been maimed or injured by diseases that could better be treated if medications were made available more promptly and expeditiously, and if some mechanism could be found to make the process of approval more rapid and less expensive. A landmark development has recently oc curred in the field of ophthalmology. The FDA has taken the information available on pimaricin from the studies done in this country and abroad, and, through internal motivation and the use of its advisory com mittees has approved this drug for manu facture. It will be made available to oph thalmologists, even though no significant profit is likely ever to be generated from its sale, and this ability to treat patients with fungus keratitis has been made possible through FDA recognition of the need, and a realization that some special consideration must be given to the treatment of less com mon but important diseases. It seems that there is almost constant criticism of the FDA in the press and the Congress. I think it is time that we in oph thalmology praised them for an action that will greatly benefit our patients and may set an important precedent. HERBERT E.
KAUFMAN