Summary

This module is unique for students interested in translational research on novel therapies (e.g. enzyme replacement therapy, gene therapy and RNA therapy) and drug development. You will learn about the steps required for experimental therapies to progress from bench-to-bedside and get adopted into routine medical practice. The module includes the latest development in next-generation therapies, utilization of biomarkers, clinical trials management, commercialisation strategies and intellectual property protection, industry perspective into novel therapies, and ethical and regulatory legislation. Examples of trials/translation from industry, specific aspects of dealing with regulatory agencies and the basic principles of health economics relevant to novel therapies will also be covered.

Learning outcomes and objectives

On completion of the module, you will:

1. Understand the trajectory of new therapy development

2. Understand the principles of setting up and managing a clinical trial

3. Gain an industry perspective of gene, cell and molecular therapies

4. Appreciate the ethical implications in setting up clinical trial

5. Understand the cost effectiveness focusing on cost utility analysis

6. Understand health economics and evaluation in healthcare

Who is this module for?

This is a compulsory module for the MSc Personalised Medicine and Novel Therapies and optional module for MSc Cell and Gene Therapy, MSc Precision Medicine and other relevant UCL MSc Programmes

 

Teaching and Learning methods

 

Using the theme of clinical translation of novel therapies “from bench to bedside”, the topics will be delivered using a variety of teaching methods including a blend of taught lectures, interactive group sessions, problem solving activities, applied case studies and tutorials. There will be ample time for independent study and distance learning through the e-learning environment, Moodle.

 

Selected reading list

1. Bennett CF. Therapeutic Antisense Oligonucleotides Are Coming of Age. Annu Rev Med. 2019 Jan 27;70:307-321. (review)

2. Anguela XM, High KA. Entering the Modern Era of Gene Therapy. Annu Rev Med. 2019 Jan 27;70:273-288. (review)

3. Ioannidis JPA, Bossuyt PMM. Waste, Leaks, and Failures in the Biomarker Pipeline. Clin Chem. 2017 May;63(5):963-972. (review)

4. Rare Diseases: Common Issues in Drug Development Guidance for Industry (FDA guidance - https://www.fda.gov/media/119757/download)

5. Marsden, G. and Towse, A. Exploring the Assessment and Appraisal of Regenerative Medicines and Cell Therapy Products: Is the NICE Approach Fit for Purpose? (consulting report; Feb 2017; https://www.ohe.org/system/files/private/publications/Final%20report%20NICE%20CAR%20T%20February%202017.pdf?download=1 )

6. Spinraza® and Zolgensma® for Spinal Muscular Atrophy: Effectiveness and Value ICER’s final report on treatment for SMA (ICER’s final evidence report; April 2019; https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Final_Evidence_Report_052419.pdf ) 7. Intellectual Property Primer (July 2012; https://www.lw.com/presentations/Intellectual-Property-Primer )

8. Kim J, Hu C, Moufawad El, Achkar C, Black LE, Douville J, et al. Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease. N Engl J Med. 2019 Oct 24;381(17):1644-1652. (N-of-1 clinical trial)

9. Schulz A, Ajayi T, Specchio N, de Los Reyes E, Gissen P, Ballon D, Dyke JP, Cahan H, Slasor P, Jacoby D, Kohlschütter A; CLN2 Study Group. Study of Intraventricular Cerliponase Alfa for CLN2 Disease. N Engl J Med. 2018 May 17;378(20):1898-1907.

10. Johnson S, Buessing M, O’Connell T, Pitluck S, Ciulla TA. Cost-effectiveness of Voretigene Neparvovec-rzyl vs Standard Care for RPE65-Mediated Inherited Retinal Disease.  JAMA Ophthalmol. 2019 Oct 1;137(10):1115-1123.

 