As economic pressure builds to controlcosts, drugs used in relatively smallpopulations will become prime targetsfor cost containment. Drug costs continueto increase yearly (Figure 1), andbecause of the aging baby boomers, the numberof individuals in need of medical servicesand prescription drugs, including recombinanthuman growth hormone (hGH), willincrease dramatically in coming decades(Figure 2).
The current yearly increase in prescriptioncosts may drive up employer healthcarecosts per employee by 250% over the next 5years, an unsustainable increase. To controlemployer costs, the financial burden fromdrugs will continue to shift to the employee,and there will be a heightened scrutiny ofnew drugs and new indications for existingdrugs by managed care organizations andemployers to ensure that they are medicallynecessary and cost effective.
An example of a high-profile drug used bya small population is hGH. Recombinant GHis expensive, costing $18 000 to $30 000 peryear. Currently, the hGH market for adultsis almost twice that for children, underscoringthe need to efficiently manage the use andcosts of hGH in adult patients with growthhormone deficiency (GHD). Health planshave seen exponential growth for hGH inrecent years. This therapy accounts for a significantportion of the overall injectable drugper member per month. Even at-risk medicalgroups are actively managing requests toallocate the appropriate resources. The costof hGH therapy in adults must also be balancedwith the ethical requirement of providingpatients with optimal care. The 4fundamental principles of bioethics providean objective approach to balance cost andcompassionate therapy and are particularlyrelevant today in the treatment of adultGHD. These principles1 are:
Efforts to manage pharmacy cost for hGHand other high-cost therapies will inevitablyconfront these basic bioethical principles.From a pharmacy benefit management perspective,the challenge is to balance theseforces while accepting resource limitations,such as helping the sick, respecting thepatient's autonomy, and sustaining thepatient's trust. In this daunting context,cost-effective and ethical resource managementrequires prospective decision makersto provide services that are not only consistentwith institutional commitments to managecost appropriately, but that also permittreatments that ensure the consistent andfair treatment of patients.2
In clinical practice, ethical issues encompassmedical indication, patient preference,quality of life (QoL), the family, and socialcontext.3 The Table presents some of theethical issues and questions that may arisewith the use of hGH. In choosing a medicalindication for a drug, beneficence and nonmaleficenceare salient ethical principles–the goals of treatment are always to promotehealth and avoid harm to the patient.Obstacles to achieving these goals includediagnostic uncertainty, conflict betweendivergent treatment goals, and use of treatmentsthat may worsen the patient's conditionor the patient's inability to respond tohGH (eg, liver disorders). The implementationof evidence-based medicine can be animportant step in avoiding the obstacles tooptimal care versus treatment; however, thisapproach may narrow the range of treatment options available and is best used asguidelines for treatment. With hGH, controversystill surrounds its appropriate use inadults and transitional patients. Patientswith appropriately diagnosed GHD and itssequelae are candidates for hGH therapy,and these uses are described extensively inpeer-reviewed literature. However, widespreadinappropriate use (eg, cosmetic, antiaging,fatigue) raises serious ethical issues.The central ethical question is: Do the benefitsof this form of therapy outweigh the risksin a particular patient?
In ethical discussions, patient preferenceis always relevant. Patient participation inthe choice of treatment can foster a sense ofpatient autonomy, bolster the physicianpatientrelationship, encourage compliance,and improve clinical outcomes. Yet, the clinicianshould ensure that the patient is acandidate for hGH treatment and the treatmentchosen is based on the best availableevidence. Incorporating the patient's preferenceinto the healthcare decision-makingprocess shifts the approach to medicinefrom a paternalistic to an autonomous perspectiveand recognizes the patient's rightto choose.
In addition, QoL issues also have ethicalimplications, especially when there is anotable divergence between the physicianand patient in what constitutes an accurateassessment of QoL. There are emerging QoLassessments, which are specific to adult GHDand these should be used to measure changesbefore and after hGH therapy. Also, thepatient may have an unrealistic expectationof the short-term improvements in QoL thatcan be obtained from hGH therapy.Ethically, the adult patient with GHD shouldbe informed that improvements in QoL,however defined, will often be a gradual,long-term process with hGH.
Finally, ethical issues should be viewed ina larger context that encompasses institutional,financial, and social networks that surroundthe individual patient. Clearly, family,social, religious, legal issues, and healthcareprovider issues often influence treatmentdecisions for particular patients. Payer financialconsiderations that restrict the allocationof resources bring into play a question of justice–how can healthcare resources be fairlyand equitably distributed to optimize careand manage costs? This is a fundamentalethical dilemma with the use of expensiveagents such as hGH, and approaches differamong healthcare plans. Throughout theethics decision process, all key parties have tolook for solutions that meet the needs of allparties. For example, to reduce cost alternatives,delivery systems can be employed usinghome care or specialty pharmacy providers.This option may also improve patient managementwith a reduction in costs. Additionalsavings can be achieved by switching fromHealth Care Procedure Coding System JCode(for injections) to National Drug Codebilling. This will allow preferred product formulariesfor injections and contractingopportunities. Further, categorical priorapproval may be required for all hGH products,and not all US Food and Drug Administrationindications automatically resultin approval–only those that were deemed bythe plan as medically necessary.
The final step in the ethical decision-makingprocess is to review performance.The performance review should refine themedical policy based on the assessmentfindings, assure access is maintained, andprevent inappropriate use. The result will bea dynamic medical policy that meets thehealth plan's goals as well as member needs.
Principles ofBiomedical Ethics.
1. Beauchamp TL, Childress JF. 4th ed. New York, NY: OxfordUniversity Press; 1994.
2. Boyle PJ, Moskowitz E. Making tough resource decisions.A process for considering both values and costs. 1996;77:48-53.
Clinical Ethics:A Practical Approach to Ethical Decisions in ClinicalMedicine.
3. Jonsen AR, Siegler M, Winslade WJ. 5th ed. New York, NY: McGraw-Hill/Appleton& Lange; 2002.