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There are several hurdles that medicines can face before being assessed by health technology assessment (HTA) criteria.

By Anonymous on Nov 4, 2013

Using the French Transparency Committee, part of Haute Autorité de Santé (HAS), as an example, Driss Berdai, an alternate member of the commission and senior consultant at the University Hospital of Bordeaux, explains...

In 2011, a study commissioned by organisations such as the European Federation of Pharmaceutical Industries and Associations (EFPIA) and EuropaBio stated that there was little evidence that the current HTA mechanism improved patient access to medicines or developed treatments with a higher therapeutic value. However, the first international survey of methods used in HTA in 2012 demonstrated that this mechanism is used to determine the value of a new medicine for clinical guidance (70% of participants use it for this purpose) and reimbursement decisions (80%).

This showed that the HTA mechanism is still relevant in improving access to new medicines. Following the above, the EU Cross-Border Healthcare Directive (2011/24/EU) which must be implemented into national law by October 2013, allows the adoption of ‘necessary’ measures to initiate the creation of EU-wide HTA network to help the different national authorities co-operate on this issue.

Driss Berdai recognises the value of HTA criteria as he talks about the hurdles that medicines face before the French Transparency Committee, “As an alternate member, I have the benefit of participating in all of the meetings and get to evaluate all the relevant technical documents [related to a product] within the committee, just like the other members.”

“A key facet of our technical assessment [as a committee] is related to the added clinical value of the product, in comparison with what is already available [on the market]. This is only possible if patients in control groups in (phase III) clinical trials are exposed to adequate available treatments in optimal conditions [related to dose, duration etc.]”

“If not, we do not have the necessary optimum data to evaluate the new product. Differences in treatment in various territories may also exist which means that we do not always have a [national] reference for best practice. But even when these differences do not exist, the choice of placebo, as a means of comparison when treatments are available can complicate the evaluation. Sometimes, the comparative reference, within their study, does not represent the best local practice, differing for example, from the way the product should be administered in practice or it does not fit in with the applicable marketing authorisation procedures.”

“We should build on comparative efficacy or effectiveness research, for the purposes of the HTA, by using a variety of reference methods. So a new product should not only be compared to a placebo but also to the ‘gold’ standard.”

The field of medicine often concerns complex systems [as well as] large quantities of data and research, decisions are not easy to make, even when we do have [criteria] to follow.

The lack of choice of comparators seems to be a recognised problem in France since 2010 when Driss, along with other colleagues, provided recommendations for improving the variety of comparative data for new treatment. These recommendations include the expansion and international harmonization of clinical practice guidelines as well as early scientific consultations at phase III of a trial which would improve the therapeutic value of the relevant product.

So, while building on comparative effectiveness research (CER) is a work in progress, Driss believes that the Transparency Committee offers a scientific assessment of the product, using a variety of stakeholders, as required by HTA practice, “The field of medicine often concerns complex systems [as well as] large quantities of data and research, decisions are not easy to make, even when we do have [criteria] to follow. The benefit of having this committee is that we have different types of [evidence] made up of internal assessors, the committee members, specific experts as well as the data from the applicant, to build our opinion.”

“Applications received by the HAS are evaluated by internal assessors who can be physicians or pharmacists working within the HAS to prepare draft [evaluation] reports. These confidential reports as well as the application documents are sent in advance to all the members of the Transparency Committee, for the [forthcoming] meeting."

“In addition, the internal assessors can contact specific external experts in the field of the related product, focusing on its clinical aspect. After a conflict of interest check, these experts are invited to present their own opinions [of the product] before the Transparency Committee. In the end, our decision is based on a majority vote.”

We [the French Transparency Committee] are only allowed to evaluate the clinical value of the product. Price evaluation is the competence of another committee which is independent from the HAS.

This demonstrates that the French Transparency Committee produces an adequate amount of scientific information on the therapeutic value of a product. More importantly, it only has a technical role in the evaluative process while the pricing and reimbursement decision is left to other independent bodies such Comité Economique des Produits de Santé (CEPS) and reimbursement rate fixing by the Union Nationale des Caisses d’Assurance Maladie (UNCAM):

“We [the French Transparency Committee] are only allowed to evaluate the clinical value of the product. Price evaluation is the competence of another committee which is independent from the HAS. The process of evaluating the price in the EU is very different in terms of timing and launch of the product on the market. In France, prices are discussed before launching the product on the market. Processes applicable to pricing of medicines are variable between EU Members states and this is different in the UK and Germany, making it difficult to agree on a standardised price.”

While the harmonisation of pricing mechanisms, in relation to new medicines is unlikely, a standardised comparative reference system would be important in improving the quality of new products given market access. Additionally, advanced scientific consultations, at the phase III stage of a clinical trial would allow collaboration between technical authorities and manufacturers that would improve the clinical added value of a medicinal product.