Research & Development

Target Organs & Systems

Like previous years, solid tumor oncology remains the top target organ for respondents’ research and development. However, antiviral and immunology are now the next priorities, likely to be as a result of the COVID-19 pandemic.

Clinical Phase

Most of the respondents said that their most advanced therapeutic product was still in the discovery phase, while 16% said their product had reached phase II of clinical trials. Interestingly, only a small number had reached phase III, lower than 15% of those who responded to our survey in 2019. 12% of respondents said that their product had been approved, a higher proportion than just 5% of respondents back in 2019.

New Therapeutics

By average, efficiency of delivery was seen as the biggest challenge facing the development of new therapeutics. Most respondents who selected this were from companies with a focus in oligonucleotide therapeutics, vaccines and other products. Meanwhile, manufacturing capacity was seen by average as the second biggest challenge, mostly by those in companies working on mRNA therapeutics and genome editing.

Interestingly, clinical trials were selected by the least respondents, of whom were working in pharmaceutical companies and CMOs. This number is lower than in 2019 when this answer was selected by 12% of all respondents. Safety and specificity were also recognized as much less of a challenge than in 2019, when it was selected by average as the second biggest challenge.

Similarly, the biggest R&D/technical challenges faced by our respondents’ companies have not changed dramatically since 2019. However, partnering has now taken the place of regulatory challenges in 5th place.

Drug Development

In this open question, the most important points for making a good drug have not appeared to be very different from those in 2019. Efficacy and safety remain the top priorities suggested by our respondents. However, cost was the third highest suggestion, as opposed to delivery in 2019.

The Biggest Challenges

Quality control and control of impurities has remained the biggest challenge for respondents developing peptide therapeutics since our last report 2019. This is now the biggest challenge for those developing mRNA therapeutics too.

Respondents focused on genome editing reported risk-benefit analysis as their top challenge for regulatory compliance. Delivery systems were the top mentioned by those working on oligonucleotides, a shift away from safety which was the top mention in 2019.

What are the biggest challenges in meeting regulations related to:

Peptides

Top Response: "Quality Control"

"Efficacy, cost, patient adherence"

"Oral delivery"

"Long peptides = synthetic proteins, manufacturing at acceptable cost"

"Standardization"

"Biodistribution"

Oligonucleotides

Top Response: "Delivery Systems"

"GMP procedures, Scale and foreign dependency on raw materials"

"Intracellular reach"

"Evaluation of ADMET for longer nucleotides: Pharmacokinetic bioanalysis and immunogenicity for example"

"Setting limits of impurity specifications"

What are the biggest challenges in meeting regulations related to:

mRNA therapeutics

Top Response: "Quality Control"

"Cost"

"Weird German law that makes even the API an FDF"

"Cell targets"

"Drug Delivery"

"Immune response"

Genome Editing

Top Response: "Risk-benefit Analysis"

"CMC"

"Viral shedding evaluation in virus vector"

"Changing targets and payloads (e.g., guide RNAs) without going through full review"

"Cost of personalized medicine"

"Delivery"