Regulating Drug Development Is a Winning Strategy for All
The FDA Office of New Drugs held a public meeting earlier this month to invite discussions and policy suggestions that promote effective drug development programs. Considering the unprecedented rate at which drugs are being developed, creating an appropriate set of guidelines and regulatory structure is of particular importance. The goal is for this to ultimately increase the likelihood of a drug being successfully and rapidly delivered to patients.
Consistency and Transparency in Open Data
One notable aspect of drug development that has transformed the field in recent history is the rapid growth of data use and storage capacity. As a result, entire fields of data processing and modeling have emerged.
But according to a presentation given by Sanofi at the meeting, advancements in this field will not optimally benefit the clinical world until a streamlined and regulated process is established regarding the collection, representation and availability of this data as it applies to the drug development process. The presentation emphasized that transparency and consistency from the FDA and other regulatory agencies, with respect to open data, are key to not only expedite the drug development process itself, but to also help drug makers anticipate the FDA’s regulatory approach when reviewing the product.
Further supporting these arguments, a recent report in The New England Journal of Medicine found that results from only 68 out of a total 135 completed trials per week are reported on ClinicalTrials.gov, even though posting trial results is mandated through federal law for a drug or device that sponsors hope will enter the market. Unfortunately, regulatory agencies have not enforced the law. Moreover, the data that is posted is often laden with inconsistencies and poor-quality records.
Innovations in Clinical Trials
Parexel, through their presentation at the meeting, provided a different suggestion for helping facilitate the drug development process: the need for industry to take advantage of the multitude of innovations available in trial designs.
- The presentation directly cites research conducted by The Economist Intelligence Unit (EIU), which specifically mentions four innovations:
Adaptive trial designs, which allow for modifications once a trial is in progress based on results accumulated in the trial and in accordance with pre-specified rules.
- Patient-centric trials, which aim to decrease the burden on patients by considering their needs in trial design and execution.
- Precision medicine trials, which test personalized medicines in trials, primarily using genotyping and biomarkers to identify relevant patient groups
- Real-world data trials, which use data about the impact of a therapy derived from real-world settings and sources.
These innovations are, importantly, models that have been well established such that there is sufficient data available for quantitative analysis of these methods. Indeed, The EIU highlights evidence that the models improve metrics of success in clinical trials, such as trial efficiency, drug launch and market access approval.
The research also demonstrated, however, that the adoption rates for these innovative models are extremely low. Specific barriers that seem to be responsible for this impediment include human capital that fails to keep pace with advances, organizations not going far enough to systemize initiatives in drug development and, relating to the previous discussion by Sanofi, data that is fragmented and not widely available.
A Larger Conversation
It is crucial to understand that these are not necessarily separate ideas – the call for sharing data more readily helps develop and implement new innovations in clinical trials, and vice-versa. For this reason, a collective approach toward action will be the most effective.
Companies and pharmaceuticals can help the larger community by organizing and making data available. Clinical trials should begin adopting some of the well-established innovations that are applicable to their research design. Although these transformations will come at an initial cost, industry will benefit from long-term savings through increased trial efficiency and drug launch success rates.
Anyone involved in the health industry with a stake in drug development should monitor policy updates and keep the pressure on the FDA and industry to encourage appropriate changes to be made. This is particularly true of JPA and the healthcare PR industry, which is well-positioned to help drive progress due to strong relations with industry, government as well as media. The hope is, ultimately, to deliver therapies to patients across all disease categories more rapidly and at a reduced cost.
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