Innovating in the biopharmaceutical field is becoming more and more challenging. Our recent report, in conjunction with Thomson Reuters, clearly shows that some companies are successfully dealing with the current environment, while others are struggling to innovate. One of the key themes to come out of last week's FT Global Pharmaceuticals and Biotechnology Conference was the need to shift from a therapy area focus to a focus on science – identifying specific pathways and targets, whether these be proteomic or epigenetic . This means first developing an understanding of the science of a specific biological pathway and then identifying the therapeutic areas that can be targeted through manipulation of this pathway. Many of the speakers highlighted how important targeting is becoming for any new life sciences drug; targeting the right science, targeting the right patients and targeting outcomes.
When it comes to targeting the right science, success in treating certain cancers is increasingly down to the ability of researchers to identify appropriate genetic or protein targets and subsequently develop therapies which interact with those targets to prevent disease onset or disease progression. One Nature Review paper highlighted at the conference examined factors driving R&D success and demonstrated that therapeutic areas are not in themselves a driver of success. The paper cites infectious diseases, on which a large amount of research has been focussed, as a positive indicator of success. In contrast a focus on neuroscience, which remains a poorly understood area of scientific research, was seen to exert a negative influence. What appeared to make the most difference was scientific acumen and good judgement, particularly with respect to earlier terminations and initiating proof of concept promptly.
In terms of targeting the right patients this has two key implications for the industry. Firstly, targeting the right patients typically means a companion diagnostic will be required. This involves not only identifying an appropriate therapeutic target, but ensuring that it is possible to identify a diagnostic marker for which a test can be developed.
The second implication of targeting specific patients is in terms of potential revenue generation. Targeting a discrete population of potential responders will likely mean lower volumes of patients. However, the upside for all stakeholders (the industry, regulators, payers, patients and physicians) is that patient outcomes are optimised and resources (money and time) are not wasted on patients who fail to respond.
The development of personalised medicines provides an opportunity for the industry and regulators to explore new ways of collaborating, which could benefit everyone. Furthermore, such therapies typically target areas of high unmet need, for which there is either no current treatment or treatments that fail to deliver effective results.
Targeting outcomes refers to utilising the vast amount of available real world data to track treatment response and provide insight into the clinical and social benefits offered by medicines. Ultimately this will aid in demonstrating the value of medicines and approval of reimbursement relative to this. Unlocking the potential of targeted outcomes requires the healthcare system to shift towards a more transparent and collaborative data sharing model, which will provide mutual benefit to all.
A number of speakers at the conference gave fascinating insights into the importance of following the science. These included breaking down internal divisions to ensure that pharmaceutical researchers and diagnostics experts can work together in an intellectual property (IP)-free environment, enabling R&D teams to look at therapeutic targets from a combined drug and diagnostic stance. Another approach was to simplify the organisational structure, change the attitudes of staff and develop much closer links to academia, creating innovation hubs which bring the company closer to science.
There was a lot of discussion at the conference about risk-sharing models, specifically around the area of adaptive licensing. Such mechanisms would allow innovative therapies to be launched earlier into the market, as long as agreement can be reached around potential risk, availability and pricing. The advantages to the industry include:
- gaining earlier marketing authorisation
- the ability to collect real world evidence by obtaining timely feedback on a therapy's impact in the real world setting as opposed to using controlled clinical trials
- sharing risk with regulators
- removing the need for expensive and time-consuming late stage trials
- generating returns earlier in the development lifecycle
- more rapid uptake of innovation.
The advantages for patients, payers and regulators include:
- earlier access to innovative new medicines
- flexibility around pricing – the idea being as part of the risk-share, drugs are launched at a lower price
- real world data which is more reflective of patient outcomes is collected earlier
- the true value of the drug becomes apparent earlier.
Overall this suggests that the direction of travel for biopharmaceutical innovation should be a win-win for everyone concerned.
The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.
