British Journal of Clinical Pharmacology
DOI:10.1111/bcp.12249
Letter to the Editors
The licence for a custom-made medicinal product is an adaptive licence John D. Johnston c/o 35 Indescon Square, London UK
With reference to proposals for new European pharmaceutical legislation to permit access to custom-made medicinal products [1]: 1 It is envisaged that each recipient of a custom-made medicinal product will enter into a post-licensing pharmacovigilance exercise to gain data on use of the product through its entire lifespan. 2 Exposure of the recipient to the custom-made medicinal product represents a trade-off between early access vs. increased (acceptable) uncertainty over benefits and risks. 3 Communication to the public and practitioners will emphasize uncertainties, the evolving nature of knowledge of the product and the provisional nature of any benefit–risk assessment. 4 Communication will include consent of the recipient being required for each exposure and more participation of patients in determining what is ‘acceptable risk’. 5 Patients will be selected on the basis of genomic sequence thereby ensuring that the custom-made medicine is reserved for a well-defined population. 6 Restrictions and monitoring of prescribing will be ensured by use of a ‘statement’, as described, to accompany the custom-made medicinal product from manufacturer to recipient. 7 The collection and analysis of observational data during exposure to the custom-made medicinal product will permit progressive reduction of uncertainty coupled to increased knowledge that facilitates decisions on treatment. The proposed licence for a custom-made medicinal product sits at one extreme of the spectrum of adaptive licensing, as described by Eichler et al. [2]. The custommade medicinal product represents an evolution, not a revolution. 1088
/
Br J Clin Pharmacol
/ 77:6
/
1088–1089
Competing Interests The author has completed the Unified Competing Interest form at http://www.icmje.org/coi_disclosure.pdf (available on request from the corresponding author) and declares no support from any organization for the submitted work, no financial relationships with any organizations that might have an interest in the submitted work in the previous 3 years and no other relationships or activities that could appear to have influenced the submitted work. The author is grateful to Dr Peter Feldschreiber, Barristerat-Law, London.
Contributors The views expressed in this article are the personal views of the author and may not be understood, interpreted or quoted as being made on behalf of, or reflecting the position of any other companies, agencies or parties cited in this article.
REFERENCES 1 Johnston JD, Feldschreiber P. Proposal for new European pharmaceutical legislation to permit access to custom-made anti-sense oligonucleotide medicinal products. Br J Clin Pharmacol 2014; 77: 939–46. 2 Eichler HG, Oye K, Baird LG, Abadie E, Brown J, Drum CL, Ferguson J, Garner S, Honig P, Hukkelhoven M, Lim JC, Lim R, Lumpkin MM, Neil G, O’Rourke B, Pezalla E, Shoda D, Seyfert-Margolis V, Sigal EV, Sobotka J, Tan D, Unger TF, Hirsch G. Adaptive licensing: taking the next step in the evolution of drug approval. Clin Pharmacol Ther 2012; 91: 426–37. Available at http://www.nature.com/clpt/journal/ v91/n3/full/clpt2011345a.html (last accessed September 2013). © 2013 The British Pharmacological Society
Letter to the Editors
RECEIVED
CORRESPONDENCE
11 September 2013
Dr John D. Johnston, c/o 35 Indescon Square, London E14 9DR, UK. Tel.: + 079 0408 3279 E-mail: [email protected]
ACCEPTED 24 September 2013
ACCEPTED ARTICLE PUBLISHED ONLINE 8 October 2013
Br J Clin Pharmacol
/ 77:6
/
1089
DOI:10.1111/bcp.12249
Letter to the Editors
The licence for a custom-made medicinal product is an adaptive licence John D. Johnston c/o 35 Indescon Square, London UK
With reference to proposals for new European pharmaceutical legislation to permit access to custom-made medicinal products [1]: 1 It is envisaged that each recipient of a custom-made medicinal product will enter into a post-licensing pharmacovigilance exercise to gain data on use of the product through its entire lifespan. 2 Exposure of the recipient to the custom-made medicinal product represents a trade-off between early access vs. increased (acceptable) uncertainty over benefits and risks. 3 Communication to the public and practitioners will emphasize uncertainties, the evolving nature of knowledge of the product and the provisional nature of any benefit–risk assessment. 4 Communication will include consent of the recipient being required for each exposure and more participation of patients in determining what is ‘acceptable risk’. 5 Patients will be selected on the basis of genomic sequence thereby ensuring that the custom-made medicine is reserved for a well-defined population. 6 Restrictions and monitoring of prescribing will be ensured by use of a ‘statement’, as described, to accompany the custom-made medicinal product from manufacturer to recipient. 7 The collection and analysis of observational data during exposure to the custom-made medicinal product will permit progressive reduction of uncertainty coupled to increased knowledge that facilitates decisions on treatment. The proposed licence for a custom-made medicinal product sits at one extreme of the spectrum of adaptive licensing, as described by Eichler et al. [2]. The custommade medicinal product represents an evolution, not a revolution. 1088
/
Br J Clin Pharmacol
/ 77:6
/
1088–1089
Competing Interests The author has completed the Unified Competing Interest form at http://www.icmje.org/coi_disclosure.pdf (available on request from the corresponding author) and declares no support from any organization for the submitted work, no financial relationships with any organizations that might have an interest in the submitted work in the previous 3 years and no other relationships or activities that could appear to have influenced the submitted work. The author is grateful to Dr Peter Feldschreiber, Barristerat-Law, London.
Contributors The views expressed in this article are the personal views of the author and may not be understood, interpreted or quoted as being made on behalf of, or reflecting the position of any other companies, agencies or parties cited in this article.
REFERENCES 1 Johnston JD, Feldschreiber P. Proposal for new European pharmaceutical legislation to permit access to custom-made anti-sense oligonucleotide medicinal products. Br J Clin Pharmacol 2014; 77: 939–46. 2 Eichler HG, Oye K, Baird LG, Abadie E, Brown J, Drum CL, Ferguson J, Garner S, Honig P, Hukkelhoven M, Lim JC, Lim R, Lumpkin MM, Neil G, O’Rourke B, Pezalla E, Shoda D, Seyfert-Margolis V, Sigal EV, Sobotka J, Tan D, Unger TF, Hirsch G. Adaptive licensing: taking the next step in the evolution of drug approval. Clin Pharmacol Ther 2012; 91: 426–37. Available at http://www.nature.com/clpt/journal/ v91/n3/full/clpt2011345a.html (last accessed September 2013). © 2013 The British Pharmacological Society
Letter to the Editors
RECEIVED
CORRESPONDENCE
11 September 2013
Dr John D. Johnston, c/o 35 Indescon Square, London E14 9DR, UK. Tel.: + 079 0408 3279 E-mail: [email protected]
ACCEPTED 24 September 2013
ACCEPTED ARTICLE PUBLISHED ONLINE 8 October 2013
Br J Clin Pharmacol
/ 77:6
/
1089
