Talking R&D: US regulatory guidelines
pharmafile | October 4, 2010 | Feature | Manufacturing and Production, Research and Development | Talking R&D, eCTD submissions
Recent speculation surrounding current diabetes drugs and their long term effect on cardiovascular health has heads spinning in the pharmaceutical industry.
The continued scrutiny on diabetes drug development and the need to conduct long term outcomes studies can be a challenge for companies trying to navigate an efficient development programme.
Diabetes, as well as other metabolic disorders, continues to burden our healthcare system at an alarming rate. And yet, the public suspicion around the approval of drugs used to treat these disorders puts both companies and regulatory authorities in difficult positions
Putting safe and effective medications on the market will never be a no-risk situation, but the FDA’s challenge is to get these drugs to market without the least bit of compromise on public wellbeing and safety. Therefore, the approval process for diabetes agents, as well as other highly demanded therapeutic products, is not only complicated – it is pathway watched and assessed by so many industry persons around the world.
There continues to be spirited debates as how best to balance the interests of individual drug developers, the possible self-motivations of investigators, and the protection of the public. New FDA guidelines surrounding these drug approvals will surely increase. Not only will diabetes research feel the increased pressure, but obesity disorders and metabolic diseases are most certainly next on the horizon.
Current Medpace executive director, regulatory, David G. Orloff, MD is a frequent industry speaker on issues of diabetes research with respect to regulatory concern. In a recent interview with Closer Look, a newsletter aimed at improved patient advocacy for diabetes, he commented on the current FDA regulatory guidance with regard to CV health and its effect on diabetes research. On the current regulatory environment for both big and small companies developing diabetes drugs he noted: “It’s quite obvious that the CV risk assessment requirement for new diabetes drugs have caused companies to pause before entering into the diabetes space in therapeutics development.
“Given the burden of increased time and cost, I don’t think anyone would disagree that the guidelines have had an impact on funding in the diabetes space. Having said that, at the very least, the FDA has laid down very clear rules so companies are aware of where and what the target is; the goalposts have been moved, perhaps, but we know where they are. Viewed optimistically, I don’t believe this adds any more real risk to the proposition of developing new diabetes drugs. In other words, if a company otherwise finds no evidence to support potential CV risk based upon a drug’s mechanism of action, animal studies, and earlier phase clinical trials, then the real development risk associated with that drug (i.e. it failing at the finish line because of a CV risk signal in an outcomes study required by the FDA) does not change appreciably
“But of course, the amount of money that has to be placed on the bet of success is greater, even if the change of success is the same. When all is said and done, then, the size of the potential investment loss is greater, while the chance of losing it may be unchanged. I am not saying that those facts ought to make anyone feel particularly good, of course. So again, the observation (not from me, but the community at large) is that there is a concern about entering into the diabetes space. By the same token, when the FDA issues guidance that’s interpretable and clear, then at least drug and biotech companies know where they stand.”
The bottom line
Anticipated FDA guidance will mean new hurdles for sponsors, ensuring that the most successful projects will emerge as the projects with expert strategic outsourcing partners who have a successful track record of approvable NDA sub-missions and rich therapeutic expertise.
The solution
Keep a responsive eye to the FDA’s continued regulatory fine tuning and scrutiny on outcome trials. This initiative will be considered a best practice in an industry where speed to market ultimately guarantees the commercial success or failure of new treatments.
These regulatory guidelines will most certainly add time and money to companies already struggling to accelerate drugs through the pipeline. This new twist in regulatory oversight will encourage sponsors to seek outsourcing partners with noted regulatory expertise.
1) Outsourcing partners equipped to handle changing FDA guidance on long-term cardiac outcomes
Sponsors are well served to design smart, efficient trials that are both cost-effective while addressing regulatory requirements for cardiac safety with help from strategic outsourcing partners who manage all aspects of a clinical trial, from regulatory submissions, study start-up, protocol and development plans to endpoint adjudication.
2) Partner with leaders in therapeutic and regulatory best practices in designing endpoint adjudication trials
Successful development teams will ask for a secondary review with regard to diabetes and obesity development plans. Be sure that your second opinion includes a therapeutically focused partner with a state-of-the-art Electronic Adjudication System to keep your project on track.
3) Electronic Adjudication System integrating into the overall project management system for the trial
An endpoint adjudication module capable of delivering effective communication. The system must offer real-time information on patient outcome data. Communication between sites, adjudication members, safety and clinical depart-ments must be available on a singular system that allows all parties to query, question and arrive at the critical endpoint for study decisions. This communication system not only allows relevant patient data to flow quickly to all decision makers, but also keeps the trial timelines intact by managing endpoint target numbers.
Catherine Soldano is the executive director for business development at Medpace. More information can be found at: www.medpace.com
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