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Early days, early dialogue with payers and regulators--a useful waste of time?

05 November 2013 Gustav Ando

Fans of the TV series South Park may be familiar with the appearance of the satirical "Captain Hindsight", a superhero who does not help people when disaster strikes, but rather tells them what they should have done to prevent the disaster after the fact. Pharmaceutical companies have faced a similar conundrum in their feedback communication with payers and HTA agencies: only after the clinical trial programmes were completed, and the product dossier was submitted, were they told about potential flaws in the trial design and why the new product will not be reimbursed.

This issue lies at the heart of initiatives to improve early communication between pharmaceutical companies, payers, and regulators to ensure that consistent, co-ordinated advice is given before final decisions on clinical trial design for new, innovative candidates are made.

Here at the 16th annual congress of ISPOR in Dublin, there was a wide-ranging discussion around the topic yesterday morning with representatives from EMA, NICE, EUnetHTA and the industry. Over the last few years, a variety of bilateral, multilateral and parallel initiatives have been launched by these institutions to increase early engagement, but to date there has been little analysis over the overall value of the process. We are now at the stage where we can begin to see some of the early results.

Since 2010, EMA has been involved in parallel scientific advice (SA) together with HTA agencies, and whilst the EMA representative admitted that they have "not been flooded with applications", a total of 19 procedures have so far been finalised, with 6 ongoing. The key questions that have been raised by applicants have invariably related to endpoint selection (over 35%) and trial design (nearly 20%).

NICE was the first HTA body to provide early SA, and whilst the NICE representative highlighted 54% repeat business as a strong sign of the value it is providing, ultimately it means that nearly half of the applicants have not found the process useful enough to warrant another go. Only one of the 77 SA requests has resulted in a completed technology appraisal (which did result in a positive decision) - but nearly 30% of the requests has been for drugs where development has been terminated. Ultimately, the jury is still out on its relative success - 60% of the drugs are either still in trials or at the regulatory filing stage.

In parallel, there is a multi-HTA dialogue initiative involving 12 HTA agencies from 9 countries. Having run a variety of pre-pilot and pilot programmes, the initiative is now able to begin establishing its procedures and templates in order to eventually create a model for a permanent network, including briefing books. Again, the evidence for its usefulness is very preliminary, and EUnetHTA is in the process of finalising a survey over how the applicants have found the experience.

From the industry perspective, the emphasis was on how critical it is to have the relevant engagement at the time decisions are being made. Whilst the early dialogue initiatives have been very important, there was still a general feeling that more needs to be done to ensure that there is more flexibility in getting contact with the HTA agency. There is still also the overriding concern over the "beware what you ask" effect - that the company might not like what the HTA agencies have to say.

Ultimately, pharmaceutical companies have a variety of options at their disposal: they can engage with HTA agencies and payers early in the development timeline, but it is an expensive and time-consuming process. They can instead still focus on its own expertise or advisory boards, but ultimately the lack of early input into the trial can come back and haunt the company. The general consensus seems to be that it is still too early to conclude what the best approach is.

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