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Concerted efforts

Closer working between companies' own departments and between international regulators will streamline the drug approval process

books_hammerRegulatory professionals take on a multitude of specialist roles during the pharmaceutical product lifecycle, from concept development to marketing. Despite the diversity of functions in this field, the goal of protecting and promoting public health is a universal priority. In the complex global regulatory environment, all involved need to know how to work together to bring effective and affordable drugs to market as quickly as possible. Education and communication between the pharmaceutical industry, regulatory organisations and the bodies responsible for bringing products to local frontline healthcare are becoming essential in an increasingly global market.

In response to the challenge of improving access to medicines in the changing regulatory environment, how are regulatory organisations streamlining their working practice and how can they improve further?

The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are increasingly communicating with one another 'behind closed doors'. From the early stages of product approval, they are taking a bilateral approach to applicant consultations. A mutual confidentiality agreement, which was extended indefinitely in September 2010, protects non-public information and ensures that decisions are made based on the same data.

The first parallel scientific advice, issued in October 2003, and joint telephone consultations are used by licence applicants to help align agency positions in the early stages of applications. Exchange of expertise and opinion, as well as administrative simplification, has gone some way to coordinating outcomes in both regions. In fact, over 77 per cent of decisions resulting from independent US and European review processes are in agreement for the same product. It is now important that scientific working parties on both sides of the Atlantic are able to endorse the outcomes of parallel agency advice to maintain the credibility of such cooperative efforts.

Across Europe, nations are building Health Technology Assessment (HTA) bodies to assess the risks, benefits and clinical relevance of new products. Such national organisations have to fit into the healthcare system of their country and respond to local political and market forces. In an ideally coordinated European assessment system, different HTA bodies would be able to interact extensively, aided by the development of a set of core assessment methodologies.

In the foreseeable future, any directive for HTA bodies to adopt core methodologies is going to be voluntary rather than legislative. The process of adopting a single HTA methodology would encounter numerous barriers and success would be neither simple nor immediate, but RA professionals are striving for  harmonisation.

The implementation of the Clinical Trials Directive (CTD) provides a good example of where the transposition of centrally directed policies into local guidelines can encounter pitfalls. While the CTD went some way towards harmonising procedures for clinical trials in Europe, some parties felt that it was difficult to resolve contrasting national policies, which in many cases resulted in increased bureaucracy rather than meeting the aim of reducing redundancy.

In recent years, clinical trials in Europe have seen a 10 per cent reduction in the number of protocols submitted and a 30 per cent reduction in the number of patients enrolled in studies. The decline has been attributed to the misalignment of national systems and a lack of confidence about satisfying all parties' requirements with study design and relevant endpoints.

Learning lessons from the CTD, it is important to strengthen implementation guidelines using unambiguous definitions and ultimately to establish legislation to improve communication between Member States on clinical trials.

The facilitation of information sharing using central clinical trials databases is perceived as an important step towards organisational transparency and simplification. Web-based resources, like and the EU clinical trial register make all clinical trial information searchable and available to the public, except phase 1 studies in adults.

Overall, the main stumbling block for getting a drug to market across different regions is going to remain the cost. There is now a call for systematic reliability on pricing across all countries. Whether Member States can find sufficient similarity in their healthcare systems to be able to implement harmonisation directives remains to be seen. At the very least, convergence between some of the more similar Member States should be possible, with enough support.

Parallel advice
At both national and European levels, the regulatory bodies and the 'payers' are being brought closer together. The European Commission has been discussing legislation to make payer/regulator collaborations and administration simpler.

When planning clinical trials in the earliest stages of product development, industry would benefit from clear and consistent guidance on appropriate study design and endpoints. Implementing guidelines to help payers and regulators to work together would make the eventual outcome of lengthy assessment more predictable. Those submitting applications would be more confident that the requirements of all parties had been considered and were likely to be met.

Progress towards increased collaboration is being seen through the provision of parallel advice sessions from regulatory and HTA bodies. In the UK, for example, the National Institute for Health and Clinical Excellence (NICE) and the Medicines and Healthcare products Regulatory Agency (MHRA) now offer simultaneous consultations with applicants and, although their advice is offered independently, all parties gain a greater awareness of the issues surrounding a particular product.

When all parties contribute to the debate from the early developmental phases (I/II), each learns about the others' priorities and there is more clarity on the evidence required to satisfy regulators (in terms of quality, safety and efficacy) and payers (in terms of relative effectiveness of the drug compared to others on the market) in a product licensing application.

As communication channels open up between regulatory agencies on a regional, national and global scale, it is important that the sentiment of collaboration is mirrored in industry. Departments overseeing regulation and those overseeing market access have evolved as separate entities within organisations. At the moment, there are still walls between departments. Companies need to reassess whether they still need regulatory and access departments to be separate or whether greater integration would improve productivity.

Obtaining advice during product development has always been an important part of a successful application. Bruno Flamion, chair of the scientific advice working party at the EMA has presented evidence that a product is 15 times more likely to result in a successful market authorisation application if it closely follows scientific advice in study design. Increasingly, there will be a requirement to justify reactions to the scientific or HTA advice obtained; where advice is not followed, the applicant will need to provide evidence of the reasons.

Communication of risks, benefits and adverse events reporting procedure between regulators and patients is lacking under the current system. Research presented by the MHRA shows that patients perceive equivalence between licensed drugs and safe drugs. Post-approval, only 1 per cent of patients would report adverse events caused by medical devices or pharmaceuticals to the appropriate regulatory body.

Future communication developments must focus on providing benefit and risk information to the public and thereby open channels for stakeholder feedback. Until recently, the HTA bodies have primarily consulted academic panels to inform their decisions. In the future, it is expected that feedback from both healthcare professionals and patient representatives will increasingly be taken into account in HTA assessments in Europe.

There is widespread hope that increased communication within and between regulatory parties will increase confidence in the regulatory process and contribute to the delivery of safe and effective drugs to market. Regulation of medicines in Europe is continually adapting to emerging challenges, such as the rise of fraudulent medical products available through the internet and the increased involvement of patients in directing their own healthcare.

Dedicated symposia give regulatory professionals and agencies the chance to highlight their achievements, hopes and concerns for the future. The common goal is to achieve better global access to safer and effective medicines.

The Author
Zubair Hussain is head of country regulatory affairs at Pfizer and president of The Organisation for Professionals in Regulatory Affairs (TOPRA)

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23rd February 2011


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