Executive Summary DIABETES MELLITUS AND GLUCOSE CONTROL BIOTECHNOLOGY INNOVATION IN DIABETES: INSULIN ANALOGS EXECUTIVE SUMMARY

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1 Executive Summary DIABETES MELLITUS AND GLUCOSE CONTROL Diabetes is widely acknwledged as an immense and grwing public health prblem. The disease is assciated with increased risk f death, decreased quality f life and cstly, dangerus cmplicatins. At the rt f these prblems is the lss f the bdy s natural ability t prduce and use insulin t maintain nrmal levels f glucse in the bld. Fr many patients with diabetes, ne f the central means f maintaining bld glucse cntrl is thrugh the external delivery f insulin. Fr a patient whse bdy prduces little r n insulin, external insulin delivery must simulate tw types f internal insulin secretin: a blus secretin, which is a rapid-nset, highlevel secretin f insulin in respnse t meals, and a basal secretin, which prvides a cnstant, lw-level f insulin fr the bdy s between-meal metablic demands. By delivering insulin at the right time and in the right amunt, patients can keep their bld glucse at near nrmal levels, which will limit the develpment f serius, lng-term cmplicatins such as blindness, kidney failure and limb amputatin, 1 and subsequently decrease risk f death and increase quality f life. BIOTECHNOLOGY INNOVATION IN DIABETES: INSULIN ANALOGS Advances in Insulin Therapy One f the mst significant areas f recent innvatin in diabetes treatment has been the develpment f new frms f insulin, called insulin analgs, which allw the bdy t mre clsely mimic the natural regulatin f bld glucse that ccurs in peple withut diabetes. Insulin analgs prmise imprvements in bld glucse cntrl by decreasing the frequency f hypglycemic (lw bld sugar) events and reducing hemglbin A1C i levels. A1C levels are an established prxy fr average bld glucse level ver time and a strng indicatr f lngterm cmplicatin develpment. Insulin analgs are als mre cnvenient fr patients t take than regular insulin and thus have the ptential t imprve patient cmpliance with treatment regimens. This reprt analyzes tw classes f insulin analgs: rapid-acting insulin analgs and lng-acting insulin analgs. Rather than examining the differences amng specific prducts within each class, this analysis fcuses n the benefits f each class f insulin analg as a whle: Rapid-acting insulin analgs, such as lispr, aspart and glulisine, take effect and lse effect faster than regular insulin (RI), which helps patients i The A1C test measures the percentage f glycated hemglbin (hemglbin with glucse bnded t it) in the bld, which in turn crrespnds t a patient s average bld glucse ver a perid f tw t three mnths. An A1C level between 4 percent and 6 percent is cnsidered typical fr peple withut diabetes. 3

2 INSULIN ANALOGS cntrl the rapid change in glucse levels (blus) that accmpanies a meal. Lng-acting insulin analgs, such as glargine and detemir, imprve upn Neutral Prtamine Hrmne (NPH) by prviding the mre cnstant, lw-level f insulin (basal) that the bdy needs between meals. Insulin analgs can be incrprated int a patient s treatment regimen in a variety f different ways, depending n factrs such as the stage f a patient s disease, lifestyle cnsideratins, and the specific times when the patient s bld glucse reaches abnrmal levels. Fr patients wh require nly blus r basal insulin, analgs can ften replace the patient s traditinal insulin. Fr patients wh require bth blus and basal therapy (knwn as insulin replacement), analgs have been used in tw ways. Independent use describes the use f a rapid-acting analg t replace RI as the blus insulin, while maintaining NPH as a basal insulin, r the use f a lng-acting analg t replace NPH as a basal insulin, while maintaining RI as a blus insulin. Recently, studies have als explred the use f rapid- and lng-acting analgs in cmbinatin use a regimen f rapidacting and lng-acting insulin replacing a regimen f RI and NPH. 2 Benefits In general, the use f rapid- and lng-acting insulin analgs is expected t have majr benefits in tw critical areas f diabetes treatment: Bld Glucse Cntrl: Landmark clinical trials have shwn that intensively managing bld glucse levels t remain within a nearnrmal range can dramatically decrease the risk f sme lng-term cmplicatins assciated with diabetes. Insulin analgs prvide benefits in intensive insulin therapy by: Reducing the variability f bld glucse levels ver time. When used as independent agents, bth rapid- and lng-acting analgs have generally been fund t be as effective as traditinal insulins in cntrlling bld glucse levels (represented by a reductin in A1C levels). A recent study has als shwn that insulin analgs can prvide an even greater reductin in A1C than a regimen f traditinal insulins when the analgs are used in cmbinatin. 2 Reducing the frequency f hypglycemia. Clinical trials f rapidand lng-acting insulin as independent treatments, and in cmbinatin therapy, shwed that treatment with analgs generally yields lwer rates f hypglycemia than treatment with traditinal insulin. 3 Patient Cnvenience: Patient cnvenience is critical t the selfmanagement f diabetes, as it greatly impacts patients decisins abut hw aggressively t manage their cnditins. Therefre, the cnvenience 4

3 factr and shrt-term quality f life imprvements will ultimately drive imprved health utcmes. Insulin analgs: Increase patients cmfrt with pursuing tight cntrl f bld glucse by reducing the frequency f hypglycemia. Ease the burden f insulin delivery n patients by allwing them t take rapid-acting insulin with a meal (r in sme cases within 20 minutes after starting a meal), rather than 30 t 60 minutes befre eating. Reduce the number f injectins f lng-acting insulin, depending n the specific treatment regimen. Value This prgress in the treatment f diabetes culd nt be timelier. As innvatins, like insulin analgs, deliver better care t patients, it becmes increasingly imprtant t examine the benefits f these innvatins relative t their csts. In tday s cst-cnscius health care envirnment, there is a grwing mvement t assess innvatins based n value that is, t determine the benefit f an innvatin relative t its cst rather than t evaluate it n prduct acquisitin cst alne. In this analysis, NEHI has taken tw appraches t examining the value f insulin analgs: A traditinal cst-effectiveness apprach (cst-utility analysis) 4 t determine the value f insulin analg drugs used in cmbinatin therapy. Extraplatin f results frm the largest randmized trial f cmbinatin use in type 1 ii patients demnstrates that insulin analgs are indeed csteffective. A qualitative review f the benefits f insulin analgs t determine whether increased cnvenience f drug administratin imprves the management f diabetes. Patient and clinician experiences suggest that insulin analgs have significant cnvenience and quality f life benefits that are critical t the lng-term management f diabetes. Hwever, these benefits are nt quantified fully in NEHI s value analysis because they have nt yet been adequately measured in the literature. Cst Effectiveness Insulin analgs can be used in a variety f different treatment regimens depending n the stage and type f a patient s disease. A cst-effectiveness evaluatin fr ii In type 1 diabetes, the pancreas is unable t prduce insulin and patients must inject r infuse external insulin in rder t live. 5

4 INSULIN ANALOGS each use is beynd the scpe f this reprt. 5 Instead, NEHI s cst-effectiveness analysis examines the specific case f cmbinatin insulin analg therapy in type 1 patients. This analysis serves as a case study f the value that insulin analgs can prvide. Evaluating the csts and benefits f cmbinatin insulin analg therapy in type 1 patients des appear t be cst effective, with a base case csteffectiveness rati f $59,001 per Quality-Adjusted Life Year (QALY) (see Appendix fr further details). The cst-effectiveness result is sensitive t the cst difference between analgs and traditinal insulin, the cst f a hypglycemic event, and the relative reductin in A1C that can be brught abut by analgs. Despite this sensitivity, there are many reasns t believe that the technlgy may prve t be even mre cst effective and valuable ver time. Fr example, there are cntinual imprvements in the clinical understanding and use f the drugs, as well as new findings regarding the additinal benefits f bld glucse cntrl in reducing macrvascular cmplicatins. 6 Cnvenience One f the majr benefits f insulin analg use lies in patients ability t maintain bld glucse cntrl with fewer restrictins and limitatins n their daily lives. There is a grwing bdy f evidence that insulin analgs can significantly imprve patients treatment satisfactin and cnvenience by imprving meal timing and reducing fear f hypglycemia. 7,8,9 T date, these benefits have nt been well quantified in terms f QALYs, and thus cannt be reflected in a cst-utility analysis f insulin analgs. Frm Cnvenience t Cmpliance Until a cure is develped, NEHI believes that sme f the mst significant advances in diabetes care will cme in the frm f imprved management regimens that patients can easily adpt and maintain. Given the pr rate f adherence with management regimens in current diabetes care, innvatins that allw a greater number f patients t enter int a treatment regimen may have significant scietal value beynd what can be expressed in cst-effectiveness terms. Insulin analgs are extremely imprtant in helping patients achieve higher levels f medicatin cmpliance. Evidence in Practice NEHI s mdeling results suggest that the determinatin f value is dependent n relatively early assessments f the effectiveness f analg drugs. As such, mre wrk needs t be dne t cnfirm the benefits f cmbinatin use bserved in initial trials. Despite this uncertainty, the wide adptin f this class f innvatin in practice indicates that much f the health care cmmunity believes in the use f insulin analgs. Insurers pay fr the analgs at the same level as 10,11 regular insulins and there is evidence f brad adptin amng clinicians. 6

5 SUMMARY This reprt examines the value f bitechnlgy in the treatment f diabetes thrugh a study f ne f the mst significant advancements in insulin therapy insulin analgs. Applying cst-effectiveness analysis t the specific case f cmbinatin use f insulin analgs in type 1 patients, NEHI determined that insulin analg drugs used in cmbinatin therapy are indeed cst effective fr type 1 diabetes patients. In additin t quantitative analysis, the qualitative evidence f imprved patient cnvenience and quality f life indicate that insulin analgs ffer significant value t patients. Mving frward, health care decisin makers need t find ways t quantitatively evaluate imprvements in quality f life and patient cnvenience. These are critical measures that need t be incrprated in the larger analyses in rder t prvide a cmplete picture f the value f an innvatin. In particular, health plicy researchers need t create a mechanism t test the hypthesis that imprved patient cnvenience leads t increased patient cmpliance and results in better clinical utcmes. 7