Regulatory Strategy in Biopharmaceuticals: Centerpiece of Corporate Planning


As you all know, the costs associated with the development of a new drug have been and still are extremely high. Furthermore, the process itself is very risky, very lengthy and highly regulated. Nonetheless, it is still an endeavor all the big biopharmaceutical companies undertake regularly and start-ups are created everyday with similar objectives. Just to give you rough numbers, in 2009, the total dollar amount spent on research and development of new medicines by pharmaceutical companies was of approximately $65 billion ($65,300,000,000)! The average cost to develop one drug was estimated to be of about $1.3 billion in 2005 and the time required to make said medicine was, and still is, of about 10-15 years.  Additionally the number of new medicines that reaches the break-even point or makes a profit is in the number of 2 out of the 10 that actually made it through. And each drug results from the screening of about 5,000-10,000 compounds and has no more than 12 years of effective patent life… and there is always the risk of a similar product hitting the shelves at the same time or few years later or even worse, few years earlier!

So why do biopharmaceuticals invest so much in drug development and why so many start-ups are being created? Well, the returns on the 1 drug that succeeds are huge and keep on increasing.  For example, Humira, the anti-TNF inhibitor from Abbott, had annual sales amounting to $9.3 billion for the year 2012, more than 7 times the cost of developing it in just one year!

Given these huge investments and potential returns, you can imagine that the critical factors for those kinds of endeavors are to get to the market quickly, ahead of competition and in the safest and most effective way possible: welcome to the science of regulatory intelligence and strategies.

Contrary to common beliefs, regulatory affairs is not just about preparing, submitting, and maintaining an investigational new drug application, and then submitting adverse event reports if needed and then just coordinating other routine communications with the FDA. It is not just about ensuring compliance… it is much more than that. Regulatory affairs is intertwined with every department in the company, and proper cooperation and coordination is of essence to insure corporate success. Indeed, regulatory affairs guides the direction of various corporate activities from a regulatory point of view with serious implications on the management and growth of the entire company or department. It is of the upmost necessity for corporate management to include regulatory thinking and planning in day-to-day decisions, as they not only affect drug development plans but also market determination, marketing, business development, finance, human resource, technology, manufacturing, risk management, insurance policies, etc.

Regulatory strategy is based on those exchanges with different internal, external and regulatory influences and is an ever changing, highly dynamic and determining science for any biopharmaceutical company, no matter its size. Indeed, it is the regulatory strategy that determines the plan for developing the drug with the objective of obtaining the right regulatory approval in the target market faster, safer and in a more cost effective manner than anyone else while properly managing and maintaining the life cycle of the drug after the approval was obtained.

The regulatory group must be aware of the very frequent changes in regulations and must be able to comply with or make use of them in the case they encompass the drug their company is developing—in consequence guiding the corporate management to take the appropriate measures to show that the drug does indeed meet expectations of the newly adopted regulations. The regulatory group must do so in record times while maintaining cost efficiency and safeguarding the competitiveness of their product compared to the competitors. A very good example of a regulatory influence that drastically changed drug development to treat serious diseases and fill an unmet medical need is the FDA Fast Track Development Program.

The reason behind why the FDA introduced that program was to get important new drugs on the market faster to patients suffering from a serious disease. The FDA considers a disorder serious on a case per case basis. However, it is generally centered on the impact the drug will have on clear factors such as survival, day-to-day functioning, or on the probability that the disease might progress from a less severe condition to a more severe one if left untreated. Examples of disorders considered serious are AIDS, Alzheimer’s, heart failure and cancer. Though, epilepsy, depression and diabetes are considered serious disorders as well. Unmet medical need on the other hand is defined as introducing a therapy, which was nonexistent or is potentially superior to the existing one(s), i.e. any drug to treat or prevent a disease which has no treatment available would qualify. Furthermore, even if there are existing therapies, the drug would still qualify for the Fast Track designation if it showed superior effectiveness, avoided grave side effects of the existing therapies, improved the diagnosis of a serious disease resulting in an enhanced outcome, or decreased accepted significant toxicity of available treatments.

That designation is quite useful and very sought-after by pharmaceutical companies since, if the drug qualifies for it, it becomes eligible for early and more frequent meetings with the FDA in order to ensure the drug’s development plan and collection of the needed data in order to support drug approval. But there is much more! The drug becomes entitled to more frequent written correspondence from the FDA about matters like the design of the proposed clinical trials. The drug can also become eligible for the Accelerated Approval regulation where surrogate endpoint—an indirect laboratory measurement that is used in clinical trials to represent a clinically meaningful outcome, such as survival, without however being itself a direct measure of it—becomes sufficient to likely predict clinical benefits of the drug, i.e. it shortens considerably the time required prior to receiving FDA approval… we are talking years shorter! Additionally, the drug becomes also eligible for Rolling Review where the biopharmaceutical company can submit completed sections of its New Drug Application for review by the FDA without having to wait until every section of the application is completed before the entire application can be reviewed—The New Drug Application does not usually starts before the company has submitted the entire application to the FDA! And of course, any drug in the Fast Track Program receives a Priority Review, and we all know how important that is when it comes to the FDA.

The pharmaceutical company can request the Fast Track designation at any time during drug development and, usually, the FDA will make its decision available within 60 days! It is important to note however, that the FDA requires the biopharmaceutical company to conduct additional clinical trials after it receives approval under the accelerated approval program, and while its drug is on the market, to confirm the drug’s direct benefits and validity.The regulatory affairs group cannot elaborate a strategy the same way when aiming for Fast Track designation as it would for the normal track. The average time saved using the Fast Track is between 3 and 6 years compared to the normal track—and sometimes half the time of the normal track is sufficient to have the new drug on the shelves with the associated cost savings and that much longer effective patent life! In fact, the strategy elaborated by the regulatory affairs group will determine how corporate management will handle company growth, day-to-day decisions, finance, human resource, technology, research and development, marketing, etc. In truth, the regulatory affairs group is in the “hot seat” in the drug development process.


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