We’ve now officially closed the door on 2017 and we’re looking forward to what the New Year has to bring for pharmaceutical and biotech companies. As the CEO of NDA Group I have the privilege to listen to many of the people at the forefront of drug development. Based on these conversations there are a few trends that I’d like to highlight that I believe will bring opportunities as well as challenges to the industry.
ADVANCED THERAPIES ARE A REALITY – BUT THE COMPETITION IS FIERCE
2017 saw the first approvals of gene therapies to treat cancer. The modification of the patient’s own T-cells using Chimeric Antigen Receptors has created treatments that arm the body’s own immune system to deal with the cancer cells. CAR T-cells, as they’re called, holds the promise of revolutionising treatment of rare forms of cancer, but is also potentially useful in many other areas.
Interestingly, even though the first two CAR T-cell treatments to achieve approval originates in the US, the majority of products in clinical trials come from China based companies. According to a recent article in the Annals of oncology[1], out of 162 products in clinical development, 98 originates with 46 Chinese companies.
Whether the intention of the Chinese is to take their pipelines global or to focus on the domestic market, expect there to be a rapidly increasing competition in a field that is currently lauded as the cutting edge of science.
EFFICIENT DEVELOPMENT AND ACCELERATED PATHWAYS
In all areas, but maybe most prominently in the enormous pipeline of oncology products, novel trial designs and development as well as regulatory acceleration is going to continue to be a hot topic. Driven by the regulators’ wish to get lifesaving products to patients faster and faster, and the industry’s strive to keep development costs to a minimum before commercialising, as well as getting a fast-to-market advantage, new ways to look at generating and analysing clinical data will become increasingly important.
Some people put the emphasis on data generated in the real world, but the whole story is not revealed until you look at data generation as a continuum. Real World Data is going to play a vital part in ensuring that the product stays within reasonable benefit/risk brackets as well as continue to play a key role in building the case for label expansions and future research. The sooner the product aims to reach the market, the more important it is going to be to have a robust post authorisation package in place.
Innovative adaptive trial design is however going to be another crucial driver for acceleration. The more data can be derived through innovative programs, seamlessly moving from safety to efficacy data collection based on pre-defined stage gates, the faster the process of achieving adequate data for early approval becomes.
These types of designs have now moved from being tested at best and merely conceptual or unrealistic at worst, to being endorsed by the major regulatory agencies. As one example in a July 6, 2017 article[2] members of the FDA outlined the use of master protocols to study multiple therapies and multiple diseases to improve the efficiency of a (single multicentre) trial.
DEVELOPING FOR VALUE
Having a novel therapy, particularly one that responds to an unmet medical need, has historically been a sure card to achieve reimbursement or end up on the prescription lists. As the cost for health care goes up, the willingness to pay high premiums has gone down and new gate keepers are put in place all over the world to keep a watch on the drug prices.
In the new age of advanced and treat-to-cure-therapies, additional hurdles are making themselves known. How should we reimburse a product with unprecedented efficacy? How do we price a “cure”? How do we finance reimbursement when the assessment arrives at a value, but there is not enough money in the budget to pay for it?
As the regulators push for earlier introduction of vital medicines, this leaves the payers and Health Technology Assessors with increasing uncertainty and less data on which to make their decisions.
I believe that 2018 will be a critical and formative year where many first steps will be taken around the world to address how these novel therapies should be evaluated and priced. The results of this process will affect us all for years to come – I suggest you watch this space.
CONCLUSIONS
2018 is going to be a remarkable year for the drug developing industry and for people all over the world suffering from life threatening and debilitating disease. The landmark victories science achieved in the last few years herald incredibly important developments of disease modifying treatments. This is good for all of us.
However, it is not a time without its challenges. The complexity of science and the introduction of novel approaches carries with it the need to educate, learn, transform and adapt. This is not a very fast process, but once we’ve absorbed the experience that this will generate for all of us, humanity will have taken another important step forward.
As an expert consultancy on novel drug development, therapies and regulation, all of us at NDA are looking forward to continuing being at the forefront of this development. Working side by side with our clients we are part of their history, and the history of the patients whose lives they change for the better every day.
References
[1] https://www.cancerresearch.org/scientists/clinical-development/clinical-accelerator/landscape-of-immuno-oncology-drug-development
[2] http://www.nejm.org/doi/full/10.1056/NEJMra1510062#t=article