Fast-advancing science has opened up the era of targeted therapy and precision medicine, and as a result pharma’s business model may no longer be fit for purpose to effectively develop and bring to market new treatments. How can pharma successfully transition from producing generalised medicine to getting the targeted therapies to specific subgroups of patients in a timely manner, while safeguarding its business?
To discuss these questions, GlobalData spoke to Dr Leeza Osipenko, head of scientific advice at the UK’s National Institute for Health and Care Excellence (Nice ), at the Pharma Integrates conference held on 15-16 November in London.
Accelerating R&D and speeding up access
Drug development has always been characterised by a high rate of failed clinical trials and products can take 14 years on average to reach the end user. Recent scientific advances offer more and more opportunities to develop personalised treatments which are likely to be more effective, if not curative; however, these therapies may offer less return on investment than blockbuster drugs that target broad populations. Developing transformative products is also more risky than pursuing incremental innovation.
Therefore, it is becoming increasingly important to speed up drug development and reduce the associated cost of high-risk, high-value medicine to compensate for the high attrition rate in clinical drug development. Careful selection of drug candidates that could bring value to the UK’s unique National Health Service (NHS), well-designed clinical trials, and appropriate selection of patient subgroups is essential to maximise chances of success, although some of this knowledge is not always readily available.
Osipenko explains how transparency and collaboration with regulatory agencies and payers could alleviate some of the barriers to speed up R&D and patient access to innovative medicine.
“Being more transparent, being more collaborative and coming to regulators, commissioners, and patients to discuss product development plans is essential for generating relevant evidence for key decision-makers and thus maximizing the efficiency of the product development process,” she says. “Companies are encouraged to design and discuss managed access agreements not just with Nice , but with the key stakeholders across the system. We are very transparent about our methods at Nice and we encourage the developers to engage with us and the regulators [earlier]. Such interactions can help industry and small biotechs increase their chances of success as well as faster identify programmes that may need to be changed or terminated.”
Appropriate drug pricing
Nice ’s rigorous methodology uses the quality-adjusted life-year (QALY) and other qualitative measures to assess the clinical benefit and determine the cost-effectiveness of healthcare interventions. Nice issues positive recommendations on about 80% of products undergoing technology appraisals. The vast majority of products coming to market, however, are incremental innovations and rarely do we see products offering cures or a substantial step-change in treatment.
While Osipenko emphasises the importance of these incremental innovations over time, she adds that they had to be reasonably priced to deliver value to the NHS, which has a fixed budget to provide access to the best affordable products within its reach. Meanwhile, she remarks that Nice would applaud breakthrough or curative therapies that come with a higher price-tag, if they deliver clinically significant outcomes to patients and/or resource savings to the healthcare system.
“Regulators and payers all want to make sure that patients get access to the best products out there and as soon as possible but they need to ensure the products’ safety, efficacy and value for money,” Osipenko says. “The systems cannot afford to pay high prices for mediocre products; however, at the time of licensing we frequently don’t know the true clinical potential – and adverse effects – of the new therapy and make decisions under a high level of uncertainty. We need to better learn how to manage and share risks and work collaboratively with the developers on generating evidence on their products, enabling patient access, ensuring affordability and most importantly, withdrawing the products which do not meet clinical and regulatory standards.”
Precompetitive collaboration and transparency
Transparency is crucial for R&D; however, both industry and academia are reluctant to share data, especially negative data, although it is widely recognised that these “failures” are critical steps to help avoid mistakes in the future. Osipenko pointed out to the audience at the Pharma Integrates meeting that too often, companies make the same mistakes independently and frequently concurrently, which is costly and unproductive.
When investigating new technologies, sharing information is not a straightforward process, strategic collaboration between suppliers and customers is required to help deliver personalised medicines faster and more cheaply. Nevertheless, to face up to the many changes of the healthcare and biopharma industries in the UK, sharing information is becoming one of the core solutions to produce world-class medicines. The biopharma industry needs to embrace innovation, open up to collaborative R&D initiatives with other companies and academic institutions and report on their failures and successes to accelerate clinical progress.