What a year! Biopharma Excellence has been easing the path to market for a whole host of advanced therapies and biosimilars

by Dr. Christian K Schneider Head of Biopharma Excellence


As Biopharma Excellence marks its first anniversary as a distinct brand within PharmaLex, Christian K Schneider, M.D., head of the business, reflects on a very full 12 months addressing the evolving challenges for ambitious biotechs.

The last year has been tremendously exciting – and busy – in the biotech space, particularly in relation to advanced therapies and biosimilars.

Progress continues a-pace in addressing very rare diseases with orphan designations. We’ve seen new approvals for cancer treatment and the advancement of CAR T-cell therapy, whose success story continues.

My hope now is that we will see additional indications come through to target more mainstream conditions – blood pressure, Parkinson’s, dementia – despite the associated development challenges. We’ve seen a number of interesting approvals recently, for example for haemophilia – where otherwise we could do little more than substitute coagulation factors; and also around some very severe metabolic disorders, and other deficiencies, which are crying out for new or even first treatment options.

Less haste, more speed

Bringing ambitious new treatments to market requires both bold thinking and careful planning. Often, companies’ priority is to pursue an accelerated development path (understandable as sponsors strive for market advantage and an expedited return on their investment). Yet this haste carries a risk, especially when the manufacturing process for a product has not yet been well defined. This can result in developments taking longer – due to sub-optimal timing of clinical trial activity, for instance.

Adaptive trials offer a lot of potential meanwhile, but again this activity must be planned, designed and rolled out with a measured approach to maximise the investment and its impact.

Companies need to be aware, too, that if they plan to use their trial results as supporting evidence for marketing approval, they must establish the benefits for the patient. Sometimes, this is assumed rather than established separately – benefit is distinct from a medicine’s activity. (If most patients see a response in their tumours yet die from serious adverse events, then the medicine was very active but clearly there is no benefit to that treatment.)

Again, this is something we’ve been helping our clients get right first time. As much as cutting-edge developments in medicine continue to benefit from huge momentum, it’s vital to ensure that something important isn’t being missed along the way.

Supplementing trial activity with data

Thinking is evolving in relation to the role of controlled clinical studies, especially in the case of very targeted orphan treatments where it is generally challenging or even impossible to create those conditions of a control, either for ethical or for feasibility reasons. This necessitates reliance on alternative data sources.

By extension, the calibre and quality of those data sources must be highly robust – something we ensure through our deeply experienced global data sciences team.

Certainly, the whole industry has a duty to ensure that we aren’t departing from the gold standards of controlled trials towards historical controls in the interests of speeding the approval of pioneering new treatments. That’s the balance we help ambitious biotechs and their investors strike in the work that we do.

Aligning cost/risk/benefit messaging

We’re also impressing on our clients the need for tighter teamwork, including the earlier involvement of Market Access to ensure that cost-benefit and risk-benefit are well aligned from an early stage. Last year bluebird bio wound down its European operations having been unable to negotiate a price with some EU member states for its authorised gene therapy.

Generally speaking, such an impasse can easily occur when the risk-benefit assessed by the regulators is poorly aligned with the cost-benefit presented to the payers (apart from many other reasons). To avoid such situation, all contributing disciplines must join forces early on to reach a consensus. It’s a theme I’ve posted about recently.

Bolder regulatory moves, sparked by biosimilars

Regulatory conditions will continue to improve steadily, as authorities take the initiative. Take what’s happening with the multiplying biosimilars opportunity, as patents expire for more complex medicines, inspiring a new surge in market activity.

Fanning this interest has been the move by the British Medicines and Healthcare products Regulatory Agency (MHRA) last year to drop the requirement for most comparative clinical efficacy and safety studies, which has also had the effect of streamlining the path to market.

It’s possible that a similar approach could apply in other scenarios too in future, where previously the default requirement would have been a new dedicated clinical trial. Regulators are already exploring options to make better use of other available data, and we’ll see more thinking around this, I’m sure.

Innovation at a trials level

Looking ahead, I see needs increasing around single-arm trials, as more treatments are authorised – especially CAR T-cell therapies targeting cancer. Having access to a greater range of potential treatments is great for patients of course, but it makes trials increasingly complicated. Where authorised products already exist, any new CAR T-cell product would, according to conventional regulatory rules, have to be compared against these. Yet this isn’t typically feasible since the new therapies are individually developed, rather than off the shelf. This introduces questions around how clinical trials should be designed going forward.

It’s a growing challenge that will affect new developments trying to find a niche within the major treatment cascades. As ever, we’re ready to do the creative thinking together with our clients, to design the ideal clinical trial protocol for the given treatment.

We’re already rolling up our sleeves, excited to see what the next year brings.

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