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Review

Practical considerations in clinical strategy to support the development of injectable drug-device combination products for biologics

, PhD & , MD, PhD
Pages 18-33 | Received 05 Sep 2017, Accepted 09 Oct 2017, Published online: 07 Nov 2017

References

  • Pharmaceutical Research and Manufacturers of America. Medicines in Development: Biologics; 2013 [last accessed 2017 October 06]. http://phrma.org/sites/default/files/pdf/biologicsoverview2013.pdf
  • Food and Drug Administration. CDER New Drug Review: 2015 Update [last accessed 2017 October 06]. http://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/UCM477020.pdf
  • Cai HH. Therapeutic monoclonal antibodies approved by FDA in 2015. MOJ Immunology. 2016;3(2):00087 [last accessed 2017 Oct 06]. http://medcraveonline.com/MOJI/MOJI-03-00087.pdf. doi:10.15406/moji.2016.03.00087.
  • Shih HH, Miller P, Harnish DC. Chapter 1. An Overview of discovery and development process for biologics. In: Wang W and Singh M, editors. Biological Drug Products: Development and Strategies, First Edition. John Wiley & Sons; 2014. p.3–30.
  • Kozlowski S, Swann P. Current and future issues in the manufacturing and development of monoclonal antibodies. Adv Drug Deliv Rev. 2006;58(5-6):707–22. doi:10.1016/j.addr.2006.05.002. PMID:16828921
  • Jefferis R. Aggregation, immune complexes and immunogenicity. MAbs. 2011;3(6):503–4. doi:10.4161/mabs.3.6.17611. PMID:22123066
  • Roberts CJ. Protein aggregation and its impact on product quality. Curr Opin Biotechnol. 2014;30: 211–7. doi:10.1016/j.copbio.2014.08.001. PMID:25173826
  • Hmiel LK, Brorson KA, Boyne MT 2nd. Post-translational structural modifications of immunoglobulin G and their effect on biological activity. Anal Bioanal Chem. 2015;407(1):79–94. doi:10.1007/s00216-014-8108-x. PMID:25200070
  • Frost GI. Recombinant human hyaluronidase (rHuPH20): an enabling platform for subcutaneous drug and fluid administration. Expert Opinion on Drug Delivery. 2007;4 (4):427–440. doi:10.1517/17425247.4.4.427. PMID:17683255
  • Goswami S, Wang W, Arakawa T and Ohtake S. Developments and Challenges for mAb-Based Therapeutics. Antibodies. 2013;2 (3):452–500. doi:10.3390/antib2030452.
  • World Health Organization Report: Adherence to long-term therapies: evidence for action. 2003 [last accessed 2017 July 27]. www.who.int/chp/knowledge/publications/adherence_report/en/
  • Badkar A, Wolf A, Bohack L, Kolhe P. Development of biotechnology products in pre-filled syringes: technical considerations and approaches. AAPS Pharm Sci Tech. 2011;12 (2):564–72. doi:10.1208/s12249-011-9617-y.
  • Majumdar S, Ford BM, Mar KD, Sullivan VJ, Ulrich RG, D'souza AJ. Evaluation of the effect of syringe surfaces on protein formulations. J Pharm Sci. 2011;100(7):2563–73. doi:10.1002/jps.22515. PMID:21319164
  • Bai S, Landsman P, Spencer A, DeCollibus D, Vega F, Temel DB, Houde D, Henderson O, Brader ML. Evaluation of incremental siliconization levels on soluble aggregates, submicron and subvisible particles in a prefilled syringe product. J Pharm Sci. 2016;105(1):50–63. doi:10.1016/j.xphs.2015.10.012. PMID:26852839
  • Jezek J, Darton NJ, Derham BK, Royle N, Simpson I. Biopharmaceutical formulations for pre-filled delivery devices. Expert Opin Drug Deliv. 2013;10(6):811–28. doi:10.1517/17425247.2013.780023. PMID:23506207
  • Jenke DR. Extractables and leachables considerations for prefilled syringes. Expert Opin Drug Deliv. 2014;11(10):1591–600. doi:10.1517/17425247.2014.928281. PMID:24955780
  • Schellekens H. Biosimilar therapeutics—what do we need to consider? NDT Plus. 2009;2(suppl 1):i27–i36. doi:10.1093/ndtplus/sfn177. PMID:19461855
  • Lee JF, Litten JB, Grampp G. Comparability and biosimilarity: considerations for the healthcare provider. Curr Med Res Opin. 2012;28(6):1053–1058. doi:10.1185/03007995.2012.686902. PMID:22519391
  • Mellstedt H, Niederwieser D, Ludwig H. The challenge of biosimilars. Ann Oncol. 2008;19(3):411–419. doi:10.1093/annonc/mdm345. PMID:17872902
  • Choy E, Jacobs IA. Biosimilar safety considerations in clinical practice. Semin Oncol. 2014;41 Suppl 1:S3–14. doi:10.1053/j.seminoncol.2013.12.001. PMID:24560025
  • Webber JTK. Demonstrating comparability for well-characterized biotechnology products. BioProcess Int 2008:32–43 [last accessed 2017 Oct 06]. http://www.bioprocessintl.com/analytical/product-characterization/demonstrating-comparability-for-well-characterized-biotechnology-products-182256/
  • Lubiniecki AS, Marcia Federici M. Comparability is not just analytical equivalence. Biologicals. 2006;34(1):45–7. doi:10.1016/j.biologicals.2005.08.016. PMID:16330221
  • Federici M1, Lubiniecki A, Manikwar P, Volkin DB. Analytical lessons learned from selected therapeutic protein drug comparability studies. Biologicals. 2013 May;41(3):131–47. doi:10.1016/j.biologicals.2012.10.001.
  • European Medicines Agency. Guideline on Comparability of Biotechnology-Derived Medicinal Products after a Change in the Manufacturing Process: Non-Clinical and Clinical Issues. November 2007. EMEA/CHMP/BMWP /101695/2006. [last accessed 2017 Oct 06] http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003935.pdf
  • Food and Drug Administration. Draft Guidance: Human Factors Studies and Related Clinical Study Considerations in Combination Product Design and Development. February 2016 [last accessed 2017 July 07]. https://www.fda.gov/downloads/regulatoryinformation/guidances/ucm484345.pdf
  • Kivitz A1, Cohen S, Dowd JE, Edwards W, Thakker S, Wellborne FR, Renz CL, Segurado OG. Clinical assessment of pain, tolerability, and preference of an autoinjection pen versus a prefilled syringe for patient self-administration of the fully human, monoclonal antibody adalimumab: the TOUCH trial. Clin Ther. 2006;28(10):1619–29. doi:10.1016/j.clinthera.2006.10.006. PMID:17157117
  • Phillips JT, Fox E, Grainger W, Tuccillo D, Liu S, Deykin A. An open-label, multicenter study to evaluate the safe and effective use of the single-use autoinjector with an Avonex® prefilled syringe in multiple sclerosis subjects. BMC Neurol. 2011;11:126. doi:10.1186/1471-2377-11-126. PMID:21999176
  • Wray S, Armstrong R, Herrman C, Calkwood J, Cascione M, Watsky E, Hayward B, Mercer B, Dangond F. Results from the single-use autoinjector for self-administration of subcutaneous interferon beta-1a in patients with relapsing multiple sclerosis (MOSAIC) study. Expert Opin Drug Deliv. 2011;8(12):1543–53. doi:10.1517/17425247.2011.628656. PMID:22032264
  • Paul C, Stalder JF, Thaçi D, Vincendon P, Brault Y, Kielar D, Tebbs V. Patient satisfaction with injection devices: a randomized controlled study comparing two different etanercept delivery systems in moderate to severe psoriasis. J Eur Acad Dermatol Venereol. 2012;26(4):448–55. doi:10.1111/j.1468-3083.2011.04093.x. PMID:21557778
  • Xie L, Zhou S, Wei W, et al. Does pen help? A real-world outcomes study of switching from vial to disposable pen among insulin glargine-treated patients with type 2 diabetes mellitus. Diabetes Technol Ther. 2013;15:230–236. doi:10.1089/dia.2012.0253. doi:10.1089/dia.2012.0253. PMID:23336845
  • Pivot X, Gligorov J, Müller V, Barrett-Lee P, Verma S, Knoop A, Curigliano G, Semiglazov V, López-Vivanco G, Jenkins V, et al. Preference for subcutaneous or intravenous administration of trastuzumab in patients with HER2-positive early breast cancer (PrefHer): an open-label randomised study. Lancet Oncol. 2013;14(10):962–70. doi:10.1016/S1470-2045(13)70383-8. PMID:23965225
  • Freundlich B, Kivitz A, Jaffe JS. Nearly pain-free self-administration of subcutaneous methotrexate with an autoinjector: results of a phase 2 clinical trial in patients with rheumatoid arthritis who have functional limitations. J Clin Rheumatol. 2014;20(5):256–60. doi:10.1097/RHU.0000000000000117. PMID:25036562
  • Seddighzadeh A, Hung S, Selmaj K, Cui Y, Liu S, Sperling B, Calabresi PA. Single-use autoinjector for peginterferon-β1a treatment of relapsing-remitting multiple sclerosis: safety, tolerability and patient evaluation data from the Phase IIIb ATTAIN study. Expert Opin Drug Deliv. 2014;11(11):1713–20. doi:10.1517/17425247.2014.944159. PMID:25073663
  • Hupperts R, Becker V, Friedrich J, Gobbi C, Salgado AV, Sperling B, You X. Multiple sclerosis patients treated with intramuscular IFN-β-1a autoinjector in a real-world setting: prospective evaluation of treatment persistence, adherence, quality of life and satisfaction. Expert Opin Drug Deliv. 2015;12(1):15–25. doi:10.1517/17425247.2015.989209. PMID:25430947
  • Paul C, Lacour JP, Tedremets L, Kreutzer K, Jazayeri S, Adams S, Guindon C, You R, Papavassilis C, JUNCTURE study group. Efficacy, safety and usability of secukinumab administration by autoinjector/pen in psoriasis: a randomized, controlled trial (JUNCTURE). J Eur Acad Dermatol Venereol. 2015;29(6):1082–90. doi:10.1111/jdv.12751. PMID:25243910
  • Blauvelt A, Prinz JC, Gottlieb AB, Kingo K, Sofen H, Ruer-Mulard M, Singh V, Pathan R, Papavassilis C, Cooper S, FEATURE Study Group. Secukinumab administration by pre-filled syringe: efficacy, safety and usability results from a randomized controlled trial in psoriasis (FEATURE). Br J Dermatol. 2015;172(2):484–93. doi:10.1111/bjd.13348. PMID:25132411
  • Pivot X, Spano JP, Espie M, Cottu P, Jouannaud C, Pottier V, Moreau L, Extra JM, Lortholary A, Rivera P, et al. Patients' preference of trastuzumab administration (subcutaneous versus intravenous) in HER2-positive metastatic breast cancer: Results of the randomised MetaspHer study. Eur J Cancer. 2017;82:230–236. doi:10.1016/j.ejca.2017.05.009. PMID:28648618
  • Borrás-Blasco J, Gracia-Pérez A, Rosique-Robles JD, Casterá MD, Abad FJ. Acceptability of switching adalimumab from a prefilled syringe to an autoinjection pen. Expert Opin Biol Ther. 2010;10(3):301–7. doi:10.1517/14712590903530633. PMID:20059372
  • Roth EM, Bujas-Bobanovic M, Louie MJ, Cariou B. Patient and physician perspectives on mode of administration of the PCSK9 monoclonal antibody alirocumab, an injectable medication to lower LDL-C levels. Clin Ther. 2015;37(9):1945–1954.e6. doi:10.1016/j.clinthera.2015.07.008. PMID:26278513
  • Korytkowski M1, Bell D, Jacobsen C, Suwannasari R, FlexPen Study Team. A multicenter, randomized, open-label, comparative, two-period crossover trial of preference, efficacy, and safety profiles of a prefilled, disposable pen and conventional vial/syringe for insulin injection in patients with type 1 or 2 diabetes mellitus. Clin Ther. 2003;25(11):2836–48. doi:10.1016/S0149-2918(03)80337-5. PMID:14693308
  • Stockl K1, Ory C, Vanderplas A, Nicklasson L, Lyness W, Cobden D, Chang E. An evaluation of patient preference for an alternative insulin delivery system compared to standard vial and syringe. Curr Med Res Opin. 2007;23(1):133–46. doi:10.1185/030079906X159524. PMID:17257475
  • Sheikhzadeh A, Yoon J, Formosa D, Domanska B, Morgan D, Schiff M. The effect of a new syringe design on the ability of rheumatoid arthritis patients to inject a biological medication. Appl Ergon. 2012;43(2):368–75. doi:10.1016/j.apergo.2011.05.014. PMID:21696704
  • Schiff M, Jaffe J, Freundlich B, Madsen P. New autoinjector technology for the delivery of subcutaneous methotrexate in the treatment of rheumatoid arthritis. Expert Rev Med Devices. 2014;11(5):447–55. doi:10.1586/17434440.2014.929492. PMID:24934630
  • Deleu D, Alsharoqi I, Al Jumah MA, Al Tahan AR, Bohlega S, Dahdaleh M, Inshasi J, Khalifa A, Szólics M, Yamout BI. Will new injection devices for interferon β-1a s.c. affect treatment adherence in patients with multiple sclerosis? An expert opinion in the Middle East. Int J Neurosci. 2011;121(4):171–5. doi:10.3109/00207454.2010.541572. PMID:21329472
  • Devonshire VA, Verdun di Cantogno E. Review of subcutaneous interferon β-1a, delivered via the electronic self-injection device RebiSmart™, for the treatment of multiple sclerosis. Ther Deliv. 2011;2(11):1455–65. doi:10.4155/tde.11.116. PMID:22844681
  • Kesson CM, Bailie GR. Do diabetic patients inject accurate doses of insulin? Diabetes Care. 1981;4:333. doi:10.2337/diacare.4.2.333a. PMID:7011752
  • Puxty JA, Hunter DH, Burr WA. Accuracy of insulin injection in elderly patients. BMJ (Clin Res Ed). 1983;287:1762. doi:10.1136/bmj.287.6407.1762.
  • Food and Drug Administration. 21 CFR Part 4 [Docket No. FDA–2009–N–0435]. Current Good Manufacturing Practice Requirements for Combination Products; January 2013 [last accessed 2017 Oct 06]. https://www.gpo.gov/fdsys/pkg/FR-2013-01-22/pdf/2013-01068.pdf
  • Food and Drug Administration. Final Guidance: Current Good Manufacturing Practice Requirements for Combination Products; January 2017 [last accessed 2017 Oct 06]. https://www.fda.gov/downloads/RegulatoryInformation/Guidances/UCM429304.pdf
  • European Medicines Agency. Concept Paper on Developing a Guideline on Quality Requirements of Medicinal Products Containing a Device Component for Delivery or Use of the Medicinal Product; November 2016 [last accessed 2017 Oct 06]. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2017/02/WC500221747.pdf
  • Food and Drug Administration. Draft Guidance: Demonstration of comparability of human biological products, including therapeutic biotechnology-derived products; April 1996 [last accessed 2017 Oct 06]. http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm122879.htm
  • International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human use. ICH Harmonised Tripartite Guideline Q5E. International Conference on Harmonization/ICH Expert Working Group. Comparability of biotechnological/biological products subject to changes in their manufacturing process; November 2004 [last accessed 2017 Oct 06]. http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Quality/Q5E/Step4/Q5E_Guideline.pdf
  • Food and Drug Administration. Guidance: Q5E Comparability of biotechnological/biological products subject to changes in their manufacturing process; June 2005 [last accessed 2017 Oct 06]. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM073476.pdf
  • Food and Drug Administration. Draft Guidance: Rheumatoid Arthritis: Developing Drug Products for Treatment; May 2013 [last accessed 2017 Oct 06]. https://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm354468.pdf