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Working Towards An Adaptive Licensing Pathway Insights From Decision Makers

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Date: 27/6/2016

Making sure that patients have timely access to effective and safe treatments remains one of our key challenges in the global healthcare system. In a state funded NHS, where resources are limited, the health technology assessment (HTA) process remains the cornerstone of access to new treatments. Here we examine the barriers to patient access of new medicines and how pharma, regulators and NICE can work together to overcome them.


The evidence needed

While randomised clinical trials remain the 'gold standard' for demonstrating safety and efficacy of new medicines, how trial data are considered alongside complementary evidence is dependent on the disease, medicine and confounding factors remains a challenge for decision makers. Real world evidence can demonstrate effectiveness in much broader disease populations, with patient-level data providing insights into the real burden of disease. Such evidence is incredibly valuable, but it is important to ensure that its use has a clear objective. This clarity of purpose allows decision makers to best interpret the data and helps them to understand the confounders and sources of bias.


How can we make collaboration work?

When it comes to making decisions about funding access to new treatments, currently the HTA framework works in a sequential way, first come regulators, followed by HTA assessors. In the not-uncommon event of an unfavourable HTA decision, the onus lies on the commercial sponsor whether to provide further evidence for consideration. Plus, in situations where there is uncertainty about the appropriateness of a decision, there is currently no clear way for decision makers and pharma to collaborate to identify and address evidence gaps. Given the variety of different real world evidence methodologies out there, working together to design one which IS well-considered, not only works in the interests of the commercial stakeholders, but also key decision makers and, of course, ultimately patients.


Implementing an adaptive licensing framework

One approach to developing an adaptive framework could be to have a concurrent regulatory and HTA review of the evidence base for new medicines. Ideally, concurrent result would allow funding agreements to be put in place earlier, allowing patients quicker access to innovative treatments. Collaboration at this early stage would also make it easier to identify evidence gaps within the initial submission, allowing the various stakeholders to mutually agree on the ideal approach to tackle these gaps, by harnessing the best research within NHS system. By working together at this stage, appropriate patient access arrangements could be brokered prior to final regulatory and HTA review.

Critically, the number of patients treated with new medicines at phase 3 of the drug development cycle increases. This means that patients for which there is no current treatment choice will have access to innovative medicines, and that are often subject to intense media interest. By making this change, decision makers will have increased amounts of relevant real world data in which to inform their regulatory and funding decisions, ensuring that patient safety remains at the cornerstone of access to new medicines.


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