population share, there were more people in the US and Europe recruited into clinical trials and provided with access to new medicines than in APAC13 or Africa. 15% of the worldwide popu- lation lived in Africa yet recruited only 2-3% of the worldwide patients into clinical trials. 57% of the worldwide population lived in APAC yet they, too, were significantly under-represented in clinical trials. Figure 1 reveals that the US, with about 5% of the world’s population, was carry- ing out more than 25% of the world’s clinical tri- als. However, China and India, accounting for approximately 35% of the world’s population, collectively performed only about 12% of the world’s clinical trials. However today, through improved patient reg-

istries in those regions of the world, and with the deployment of digitally-enabled clinical trials, sub- stantially more patients as a percentage of their population are able to participate and gain access to innovative medicines. The challenges of clinical trial enrolment, a long-

standing problem for drug developers, have been to a large extent overcome by the deployment of digital technologies. These technologies have empowered physicians, and indeed patients them- selves, to find relevant clinical trials based on dis- ease, age, gender, location and biomarkers.

Stem cell and gene-based therapies: By the mid- 2020s, stem cell14 research and cell therapy such as CAR-T, genetics and gene therapies including CRISPR-Cas9, etc were all providing break- throughs in numerous disease areas, but implemen- tation was not being fully realised. These new ther- apeutic modalities had created a new market for the products and services needed to support tissue engineering therapies, such as the culture of tissues for therapeutic purposes, the management of stem cell banks, or the highly regulated and precise delivery of cell-based therapies directly to patients. Discovery research in pharma had for many dis-

ease areas moved to 3D tissue models15. Today, there is a substantially reduced need for animal models in drug R&D and the move has both increased the precision of efficacy and toxicity pre- diction and improved the ethics of drug testing. Such developments have dramatically reduced the drug attrition rates typical in the industry and reduced time to market. Additionally, the use of new diagnostic imaging techniques has further reduced attrition rates in later-stage clinical trials. Together these two factors alone have reduced the overall costs and time required to obtain regulato- ry approval.

Drug Discovery World Winter 2019/20

Oncology: Tumours are now treated with well- established, mainstream, chronic therapies includ- ing immunotherapy16 and most cancers are being identified and treated earlier, not least due to progress in Liquid NGS17. Research initiatives, such as ASCO’s Center for Research & Analytics (CENTRA)18, has provided the infrastructure to analyse the RWD and deliver deep insights into the etiology, diagnosis, treatment and prognosis of cancers. However, the ongoing cost pressures of therapeutics remains an issue and new pricing models are being experimented with. Proteolysis- targeting chimeric molecules (PROTACs)19, pro- tein-slaying drugs, look destined to become the next wave of blockbusters.

Genomics: Genetics has moved centre-stage in recent years and personalised medicine is avail- able across the globe. The markets for cell and gene therapy20 are immense. CAR-T21 and stem cell therapy22 have matured and become avail- able for treating serious disease. Gene therapy and gene editing are now seen as being able to cure rare, monogenic diseases such as severe com- bined immunodeficiency due to adenosine deami- nase deficiency (ADA-SCID)23, or the one-time gene therapy to restore functional vision in chil- dren and adult patients with biallelic mutations of the RPE65 (retinal pigment epithelial 65kDa pro- tein) gene24. The vastly-improved knowledge in the field of genomics coupled with phenomics allowed the understanding, tackling and the cure of some polygenic diseases to be contemplated. Cure as a primary endpoint for some new thera- pies was possible. However, it came at a cost (Table 1). The business model for such curative therapies

had been difficult to implement. As Bloomberg Opinion noted: ‘Wall Street Wants the Best Patents, Not the Best Drugs’25.

Table 1: Figures relate to the late 2010s DISEASE

Hepatitis C

ADA-SCID (‘boy in the bubble syndrome’)

Inherited retinal disease Beta thalassaemia Solvadi Strimvelis Luxturna Zynteglo Infant spinal muscular atrophy Zolgensma


TREATMENT COST $ 84,000 665,000 850,000 1,800,000 2,125,000


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