Many critical and innovative new therapies today, including those for rare and untreated diseases, are in the biotech pipeline. In fact, today biotech represents more than 80 percent of the drugs currently in development. However, careful planning at the earliest stages of development is required to ensure that these life-changing innovations have the best chance of success and reach the patients that need them. In this Q&A discussion, Parexel regulatory and strategy experts tackle the question of how we can ensure that worthy innovations in the biotech pipeline make it to patients sooner, and how companies can optimize their plans earlier to mitigate risks of delay or failure along the journey to patients.
The Current State of Biotech
Alberto: You both work with biotech companies every day. Can you tell me what you are seeing in the market today? And what is the current climate for biotech?
Sheela: The good news is that we are in a favorable investment climate for biotech today. From a financing, M&A and IPO perspective, we are seeing a very strong market and it looks like that will continue for the foreseeable future. What that means is that we have many well-capitalized biotech companies that have the “gunpowder” to run the studies they need to advance novel therapies, to investigate their mechanisms and their platforms, and to evaluate multiple indications. All of this is really good news for the industry and for patients.
Leslie: From a regulatory perspective, it’s been a common sentiment in the biotech industry that over the past decade regulatory authorities have come a long way in reducing the barriers to market entry. We’re seeing, especially in the US and Europe, that we now have well-defined regulatory pathways for products such as biosimilars and cell therapies. We’ve certainly seen an opening up of the regulators with respect to accommodating the innovations in drug development, and that is encouraging.
Focusing on the Patient
Alberto: The focus on the patient has certainly become much more intense in recent years. I traveled recently to Japan and spoke to a number of companies there. There’s great interest in virtual trials, and generally in making clinical research more convenient for patients. Are you seeing this as a shift globally?
Sheela: I think it’s in everyone’s interest to make that happen. Making trials easier for patients would get them enrolled faster, would help get to readouts faster, get to approvals faster, and get to markets faster. The Economist Intelligence Unit research commissioned by Parexel indicates that trials that do have a strong patient focus are much faster to enroll, and even lead to a higher probability of launch and reimbursement1, which I think is very provocative, and something that we should help more companies achieve.
Alberto: Leslie, you’ve had so much experience dealing directly with patients in hospital settings as a former nurse, what are your views about involving patients early in the drug development process?
Leslie: There is so much we can be doing to involve patients in the process and while we have initiated this approach, as an industry I think we could do it more often, and we could do it better. Patients have so much to bring to the table with respect to understanding their own disease, understanding their treatment decisions, how they decide to participate in the clinical study, how they report the experiences that they have, the adverse events, the experience of participating in a clinical study. This input can help influence the design of studies to make life more bearable for patients and also for their caregivers. Regulatory agencies are also taking notice. In the US, and also in many other regions, companies and health authorities are meeting with patients and patient advocacy groups. Patient input is extremely impactful and important for everyone who is part of the drug development process to hear.
Getting New Medicines to Market Faster
Alberto: Now to tackle the big question – how can companies ensure they are getting these much-needed medicines to the patients who need them? What do biotech companies need to be thinking about even at the earliest stages of development?
Sheela: I think it’s critical for biotech companies to plan as early as possible to optimize investments, to really think about what it’s going to take to succeed commercially and to make those factors just as important as the scientific and regulatory ones. Sometimes we’ll see companies overly focused on the science and on the requirements to get from one phase to the next. But where they can fall short is missing a key opportunity or not having the value proposition well thought out or not having the right story to attract investors. We advise companies to step back, to really think about the end goal. Where is the technology or the product that they’re aiming to develop going to fit in the market in 10 or 15 years? Who is the patient that they’re hoping to get this product to? In order to get to that patient, what evidence are they going to have to show, not only to regulators, but also to physicians, patients, and payers? It may be challenging in the earliest stages to commit time and resources to this effort, but even in the preclinical stages, doing a high-level market analysis on different indications can help a company choose the right indications and really understand the market opportunity.
Leslie: Speaking from a regulatory strategy perspective, I absolutely agree with Sheela that these events really need to be taking place in parallel. What we often see is that the clinical strategy takes the priority position. But we find that it’s best to holistically consider the regulatory and commercial strategies early on so that we can understand the regulatory and commercial road map as we’re building out the clinical development plan. The first goal line is the regulatory authority approval for a marketing authorization, but you also need to know where you’re going in the market place because you want to understand what the requirements are for each one of the targeted markets in advance of designing your clinical trial. Marketing authorization does NOT equal market access. However, if these strategies are paired together so that we may optimize how we will most successfully drive a product through its life cycle, we have a greater chance of seeing that therapy reach the patients who need it. It also drives more efficient development.
Alberto: Final question: we all know that drug development is fraught with difficulties, challenges, traps and failures. Based on your experience, what are the most common missteps biotechs make along the drug development journey, and how could they be avoided?
Leslie: Most of the pitfalls I see are in the early product development strategy that includes clinical, regulatory and commercial. The absence of a plan and the lack of a product development road map leads to bumps in the road with respect to health authority approvals, and often requires additional work to be done before the product can progress. We encourage companies to take advantage of the opportunity for early regulator meetings – the INTERACT meeting with CBER, for instance – to engage with the development process very early on and get early advice on a wide range of development topics. Also, in biotech, many companies target orphan indications in limited patient populations in the pursuit of personalized treatments. It becomes increasingly difficult to complete studies quickly, especially in a crowded research space, often requiring more clinical sites. So, if you are focusing on some of those more orphan or unmet medical need indications, be prepared for these challenges and incorporate regulators’ feedback into early planning.
At Parexel Biotech, we share biotech companies’ drive to make life better for patients, to involve those patients in the process and to plan with the end in mind from the very earliest stages. This is critical to success. For more information on Parexel Biotech and insights from our experts, please visit http://biotech.parexel.com
¹The Innovation Imperative: The Future of Drug Development, commissioned by Parexel and conducted by the Economist Intelligence Unit (EIU), Available at: https://druginnovation.eiu.com/
