Quality-of-life research is briefly described and said to be in its infancy. However, the authors observe that such studies may, e.g., make it possible to better match patients and therapies. They also predict that, as scientific difficulties are overcome, the pharmaceutical industry will become more active in planning, implementing and evaluating such research

Breitsprecher, Andreas

Hiltbrunner, Beat

English

UNH Scholars' Repository

RISK: Health, Safety & Environment (1990-2002)Volume 2Number 1 RISK: Health, Safety & Environment Article 5January 1991Pharmaceutical Risk and the Quality of LifeBeat HiltbrunnerAndreas BreitsprecherFollow this and additional works at: https://scholars.unh.edu/riskPart of the Pharmacology, Toxicology and Environmental Health Commons, and the Pharmacyand Pharmaceutical Sciences CommonsThis Article is brought to you for free and open access by the University of New Hampshire – School of Law at University of New Hampshire Scholars'Repository. It has been accepted for inclusion in RISK: Health, Safety & Environment (1990-2002) by an authorized editor of University of NewHampshire Scholars' Repository. For more information, please contact ellen.phillips@law.unh.edu.Repository CitationBeat Hiltbrunner, Pharmaceutical Risk and the Quality of Life, 2 RISK 19 (1991).Pharmaceutical Risk andthe Quality of LifeBeat Hiltbrunner and Andreas Breitsprecher*Introduction"I am giving you medicine, not a poison." What appears to be anoath from the pen of a doctor or pharmaceutical representative is in factfrom the drama "Nathan der Weise" by the German poet Lessing andwas written in 1779. However, this quotation is still relevant today.Apart from their benefits, drugs always entail a risk. Thus, closelyassociated with the quotation from Lessing are questions with regard todrug safety and the ratio of accompanying risks and a possible increasein the chances of cure or improvement in the quality of life.Individual acceptance of risks generally, or those posed bypharmaceuticals specifically, is a function of more than their frequencyand severity. It is also a function of what benefit one expects to gainfrom assuming a risk, and both the evaluation of risk and the evaluationof benefit tend to be quite subjective.Recently, investigators have attempted to get a better grasp of riskand benefit perceptions through "quality-of-life" studies which askpatients to complete questionnaires. 1 The main objective of this paperis to briefly review what it is that these instruments attempt to measure* Dr. Hiltbrunner is head of the quality of life research group at Ciba-Geigy Ltd.,Basel, Switzerland. He received his medical training at the University of Lusanne andhas completed residencies in pediatrics and neurology in the United States. Mr.Breitsprecher is the Head of Public Communication Pharma, Ciba-Geigy, Ltd.1 See generally, e.g., QUALITY OF LIFE ASSESSMENTS IN CLINICAL TRIALS(B. Spilker ed. 1990). See also, Merz, An Empirical Analysis of the MedicalInformed Consent Doctrine: Search for a Standard of Disclosure, infra 27, at 54.2 RISK -Issues in Health & Safety 19 [Winter 1991]and the potential for use of such research in the evaluation of drugs.Risk Acceptance GenerallyDuring our life, we are confronted with an indeterminable number ofrisks. We have to estimate them and then decide whether we areprepared to take these risks in the light of the expected benefits. It iswell known that there is no such thing as zero risk. Nevertheless,there's a growing tendency in society to avoid taking risks. A sort of"zero-risk society" is emerging - a society that is attempting to protectitself as far as possible from everything forever. This is in fact aparadox. It is precisely in the western industrialized countries withgrowing technical advance and affluence that we are living in anenvironment where risks are increasing at a similar rate. One would bejustified in claiming that industry tends to increase the number of risks,not only for some individuals but for society as a whole. One can thinkof the dangers involved in industrial production and use and in theproblems of subsequent disposal of wastes. No one would wish todispute the fact that the use of the car - a symbol of technical progress- involves enormous risks. The same applies to the possibleconsequences of everyday stress, incorrect nutrition or the consumptionof nicotine and alcohol and environmental pollution. Viewedobjectively, the risk-shy industrial citizen is thus exposing himself tovery serious risks.We have to accept some risks. However, we are confronted with theproblem of trying to fit risk acceptance into a scientific framework.What is acceptable in the individual case, and what is not? And bywhich criteria and with which means can the individual determine this?In this context, what certainly plays a role is the fact that peopleassociate certain things with the subjective idea of high or low potentialrisks - regardless of the actual existing dangers. Thus, in statisticalHiltbrunner and Breitsprecher: Pharmaceuticals and Qualify of Life 21terms, the probability of dying in a car accident is significantly higherthan death from the effects of a drug.2 Nevertheless, drugs as a wholeare classed as being more dangerous. The benefit/risk evaluation appliedto drugs gives a completely different result compared with the evaluationin the case of a car.Apart from a lack of information, the reason is certain also to befound in the tendency toward different evaluation of perceptions, whichoften confuses the desirable with the actual. The daily report onautomobile accidents is certainly not as spectacular as an air crash or adramatic drug offense report. Nevertheless, in the decision-makingprocess, everyone daily makes his own, generally subjective, riskevaluation on the basis of information available to him. This also appliesto the sensitive area of drugs.Drug RisksThe pharmaceutical industry contributes to the existence of risk intwo ways. First, research and technical and administrative activities giverise to possible risks which affect safety of the workplace, theinhabitants of the surroundings or of the whole environment. These arefactors which are becoming more and more important in society. Thisbranch of industry invests considerable sums of money and humanresources in the investigation of industrial risks and in the developmentof suitable technologies to reduce risks posed by its activities. Second,the products of the pharmaceutical industry are possible sources of risk.As noted earlier, drug safety has long been a matter of concern.Medicaments must meet in particular the three main criteria of "quality","efficacy" and "safety" - safety clearly being the most sensitive area.The ethical responsibility of science, medicine and thepharmaceutical industry to minimize drug safety is undisputed. A true2 Heilmann, Das Arzneimittel als Risiko, PHARMA DIALOG 82 (1984).2 RISK - Issues in Health & Safety 19 [Winter 1991]awareness of the existence of drug-related risks among the broadpopulation first emerged in the 1960's as a result of the thalidomidetragedy. This case initiated a radical change in the public's perception ofrisks associated with the effects of drugs.In the past, evaluation of the risk of drugs was based onspontaneous reports of side effects, post-marketing surveillance studiesor literature evaluations. Nowadays, much more precise studies of drugrisks are required. They include extensive compilation of side effects inthe international context, safety evaluations in clinical studies, andbiochemical and genetic studies. Pharmacoepidemiology, a relativelyrecent branch of science, tries to achieve progress along the path togreater drug safety by international networking and worldwide dataexchange.Yet, it would be utopian to believe in the possibility of developing,in the foreseeable future, medicaments without more or less severeadverse effects. One has to live with the risk associated with drugs, butthe risk has to be viewed as a function of the therapy. Serious risks tendto be rare and minimal risks comparatively frequent. The central aspectin the determination and evaluation of drug risks is to achieve themaximum possible benefit in conjunction with the minimum possiblerisk of adverse effects to the patient.Which risk of adverse effects is acceptable or unacceptable in theindividual case? What standards must be set? In the case of life-threatening diseases, such as AIDS or cancer, answers are fairly easy toobtain. However, in the large number of chronically stationary diseases,the degree of a successful treatment tends to vary over ranges which aredifficult to measure.Hiltbrunner and Breitsprecher: Pharmaceuticals and Qualify of Life 23Risk and Quality of LifeThere is no doubt that the introduction of effective antibiotics againsttuberculosis since the middle of our century has resulted in animprovement in the quality of life of tuberculosis patients. However, anattempt rigorously to measure quality of life has only recently takenplace.3 The importance of such evaluations in connection with drugtreatment is increasing insofar as people are living longer, and thequality of life is of increasing priority to older people.4Improved quality of life has traditionally been regarded as aninevitable consequence of declining disease or life-prolonging measures.However, it has been shown that the quality of life, as patients assessesit, may deteriorate despite an "objectively" successful treatment.If the risk of dangerous side effects is high compared with thebenefits, the drug usually has to be withdrawn from the market. If therisk is comparatively small and the benefits are large, a risk/benefitassessment is also straight-forward. However, the risk/benefit ratio ofmost marketable medications fall between these two extremes. In suchcircumstances, the results of quality-of-life studies can aid doctors andpatients in choosing to pursue a method of treatment.Also, quality-of-life information may be unnecessary if, usingconventional disease parameters, the therapeutic efficacy of a new drugis clearly superior to existing treatments. Yet, such information may bevery valuable where conventional clinical parameters cannot distinguishalternative therapies. Quality-of-life measurements of apparently equallyeffective methods of treatment may, if the profiles of the side effects orthe action mechanism of a new medicament differ, help patients todecide between possible treatments.3 Luce, QoL and the Pharmaceutical Industry: A Perspective, QUALITY OF LIFEAND CARDIOVASCULAR CARE 191-192 (1988).4 Wertheimer and Nickman, Drug Therapy and Quality of Life, QUALITY OF LIFEAND CARDIOVASCULAR CARE 13-22 (1985).2 RISK -Issues in Health & Safety 19 [Winter 1991]Possibilities and Limits in Practical UseThe term "quality of life" usually covers five dimensions whichconstitute the main areas of human well-being: physical, social,intellectual, emotional and economic. There are two general types ofquality-of-life studies. Some investigate general health studies, andothers examine specific diseases. Both employ questionnaires whichhave to be completed by the patient, but such studies pose a number ofquestions. Particularly difficult is a suitable method of measurement forcomparative evaluations of the quality of life.The use of general health questionnaires is particularly suitable tocompare the quality of life of patients with different diseases ortreatment. However, they do not tend to be sufficiently sensitive todifferentiate between the effects of similar treatments.Disease-specific quality-of-life investigations are used to studyparticular aspects of a disease, for example, the effects of differenttreatments on the quality of life, their different benefits and theirdifferent risks. They cannot be used for other diseases. If the influenceof two different hormone treatments on the quality of life of women inthe menopause are studied, questions regarding the use of a wheelchair,the need for assistance to move from the bed or the inability of theperson to dress herself may be of no importance. In contrast, hotflushes and dryness of the vagina have major impact on the quality oflife of these patients but not of patients with chronic polyarthritis orhereditary diabetes melitus.Quality-of-life instruments which are developed for a particularpatient population are often not transferable to patients across cultures.Adapting instruments to new cultural or linguistic conditions is usually atime-consuming and expensive process.Additional matters further complicate quality-of-life studies. Theycan be influenced by a wide range of health and demographicI-Hltbrunner and Breitsprecher: Pharmaceuticals and Qualify of Life 25characteristics. In order to separate treatment effects on patient quality oflife from other considerations - for example, concurrent diseases - itmay be necessary to select subpopulations of patients. This may lead toloss of general validity of the results, a matter with potentially importantregulatory consequences.Studies to determine treatment related improvement in the quality oflife may be particularly important in the case of chronic diseases such asrheumatoid arthritis, in advanced stages of cancer, or asymptomaticdiseases such as essential hypertension. In the case of progressivediseases, such as cancer, an improvement of the patient's quality of lifeas a result of therapy may be interesting but not satisfactory unless theduration of the improvement is measured. Unfortunately, few validatedquality-of-life instruments can be used repeatedly every two to fourweeks.Studies on the effects of treatments on the quality of life of children,elderly people and the mentally retarded entail special difficulties, andthe first steps to resolve them have only just been taken. Additionalproblems arise from the effects which the treatment of these patientshave on their social environment - especially on parents or spouse.If a treatment leads to an improvement in the parameters of thedisease, as well as in the quality of life, the therapeutic intervention isuseful. If improvement is shown to the advantage of one drug overanother, depending on economic circumstances, health authorities maybe more willing to permit or even to favor its use.ConclusionQuality-of-life research is generally classified under the "softsciences" and may not offer the precision that some people woulddesire. Yet, studies in certain cases, make it possible to recordquantitatively the personal risk assessment of a patient and to take the2 RISK - Issues in Health & Sqfety 19 [Winter 1991]results into account for a risk/benefit analysis. Such studies can be usedto complete a registration dossier as a parameter of efficacy. Because oftheir potential, they can also be used to study the influence of a treatmenton the social environment. Therefore, they may figure into pricenegotiations. However, quality-of-life evaluations are in their infancyand still entail uncertainties and problems.Until recently, quality-of-life research has been primarily done atuniversities. However, over the past ten years, the pharmaceuticalindustry has begun to play a role.5 In the future and particularly assome of the scientific difficulties are overcome, it is expected that thepharmaceutical industry will become even more active in planning,implementation and evaluation of quality-of-life studies.5 Bombardier, Ware, Russell, et al., Auranofin Therapy and Quality of Life inPatients with Rheumatoid Arthritis, 81 AM. J. MED. 565-578 (1986). Croog,Levin, Testa, et al., The Effects of Antihypertensive Therapy on the Quality of Life,314 N. ENG. J. MED. 1657-1664 (1986).

Pharmaceutical Risk and the Quality of Life

https://scholars.unh.edu/cgi/viewcontent.cgi?article=1039&amp;context=risk

Pharmaceutical Risk and the Quality of Life

Abstract

Similar works

Full text

Available Versions

UNH Scholars' Repository