• 1
    Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on Advanced Therapy Medicinal Products and Amending Directive 2001/83/EC and Regulation (EC) No 726/2004.
  • 2
    Snodin D, Ryle PR. Understanding and applying regulatory guidance on the nonclinical development of biotechnology-derived pharmaceuticals. BioDrugs 2006; 20: 2552.
  • 3
    CHMP/SWP/1042/99 (R1) Guideline on Repeated Dose Toxicity.
  • 4
    Baldrick P. Safety evaluation to support first-in-man investigations: I: kinetic and safety pharmacology studies. Regul Toxicol Pharmacol 2008; 51: 230236.
  • 5
    Martin PL, Breslin W, Rocca M, Wright D, Cavagnaro J. Considerations in assessing the developmental and reproductive toxicity potential of biopharmaceuticals. Birth Defects Res B Dev Reprod Toxicol 2009; 86: 176203.
  • 6
    ICH S6 (R1) Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals.
  • 7
    Sistare FD, Morton D, Alden C, Christensen J, Keller D, de Jonghe S, Storer RD, Reddy MV, Kraynak A, Trela B, Bienvenu J-G, Bjurström S, Bosmans V, Brewster D, Colman K, Dominick M, Evans J, Hailey JR, Kinter L, Liu M, Mahrt C, Marien D, Myer J, Perry R, Potenta D, Roth A, Sherratt P, Singer T, Slim R, Soper K, Fransson-Steen R, Stoltz J, Turner O, Turnquist S, van Heerden M, Woicke J, DeGeorge J. An analysis of pharmaceutical experience with decades of rat carcinogenicity testing – support for a proposal to modify current regulatory guidelines. Toxicol Pathol 2011; 39: 716744.
  • 8
    Vahle JL, Finch GL, Heidel SM, Hovland DN, Ivens I, Parker S, Ponce RA, Sachs C, Steigerwalt R, Short B, Todd MD. Carcinogenicity assessments of biotechnology-derived pharmaceuticals: a review of approved molecules and best practice recommendations. Toxicol Pathol 2010; 38: 522553.
  • 9
    CHMP/410869/2006 Guideline on Human Cell-Based Medicinal Products.
  • 10
    CHMP/GTWP/12549/2006 Guideline on the Non-Clinical Studies Required Before First Clinical Use of Gene Therapy Medicinal Products.
  • 11
    Goldring CE, Duffy PA, Benvenisty N, Andrews PW, Ben-David U, Eakins R, French N, Hanley NA, Kelly L, Kitteringham NR, Kurth J, Ladenheim D, Laverty H, McBlane J, Narayanan G, Patel S, Reinhardt J, Rossi A, Sharpe M, Park BK. Assessing the safety of stem cells. Cell Stem Cell 2011; 8: 618628.
  • 12
    Sharpe ME, Morton D, Rossi A. Nonclinical safety strategies for stem cell therapies. Toxicol Appl Pharmacol 2012; 252: 223231.
  • 13
    Baldrick P, Donniger R. Nonclinical aspects of biosimilar development – practical considerations from an industry perspective. Regulatory Rapporteur 2012; 9: 1519.
  • 14
    Jones DR, McBlane JW, Hawkins R. Nonclinical aspects of biosimilar development- a regulatory perspective. Regulatory Rapporteur 2012; 9: 1720.
  • 15
    Concept paper on the revision of the Guideline on Similar Biological Medicinal Products (EMA/CHMP/BMWP/572643/2011).
  • 16
    Concept Paper for the Revision of the Guideline on Similar Biological Medicinal Products Containing Biotechnology-Derived Proteins as Active Substance: Non-Clinical and Clinical Issues (EMA/CHMP/BMWP/572828/2011).
  • 17
    Concept Paper for the Revision of the Guideline on Similar Biological Medicinal Products Containing Biotechnology-Derived Proteins as Active Substance: Quality Issues (EMA/CHMP/BWP/617111/2010).
  • 18
    FDA Guidance for Industry Scientific Considerations in Demonstrating Biosimilarity to a Reference Product February 2012.
  • 19
    Fox JL. Debate over details of US biosimilar pathway continues to rage. Nat Biotechnol 2012; 30: 577.
  • 20
    ICH M3 (R2) Non-Clinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authorisation for Pharmaceuticals.
  • 21
    Directive 2001/20/EC on the Approximation of the Laws, Regulations and Administrative Provisions of the Member States Relating to the Implementation of Good Clinical Practice in the Conduct of Clinical Trials on Medicinal Products for Human Use.
  • 22
    Communication from the Commission – Detailed Guidance on the Collection, Verification and Presentation of Adverse Event/Reaction Reports Arising from Clinical Trials on Medicinal Products for Human Use (‘CT-3’) – 2011/C 172/01.
  • 23
    CHMP. Guideline on Strategies to Identify and Mitigate Risks for First-In-Human Clinical Trials with Investigational Medicinal Products. CHMP/SWP/28367/07.
  • 24
    Detailed guidelines on Good Clinical Practice Specific to Advanced Therapy Medicinal Products ENTR/F/2/SF/dn D(2009) 35810 Brussels, 03/12/2009.
  • 25
    CHMP/BWP/41450/98 Points to Consider on Human Somatic Cell Therapy.
  • 26
    CHMP/BWP/3088/99: Note for Guidance on the Quality, Preclinical and Clinical Aspects of Gene Transfer Medicinal Products.
  • 27
    CHMP/GTWP/405681/2006: Concept Paper on the Development of a Guideline on the Quality, Preclinical and Clinical Aspects of Medicinal Products Containing Genetically Modified Cells.
  • 28
    CHMP/GTWP/60436/07 Guideline on Follow-Up of Patients Administered with Gene Therapy Medicinal Products.
  • 29
    FDA Guidance for Industry: Gene Therapy Clinical Trials – Observing Subjects for Delayed Adverse Events.
  • 30
    Megit S. Immunocore pioneers new safety studies. In MedNous 2011; 5: 1415.