What Is a Pilot Study?

The First Step to Any Medical Breakthrough

A scientist works in a lab.
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A pilot study is the scientific equivalent of dipping your toe into the water. A pilot study may involve pretesting a hypothesis to see if the idea is feasible. It may be used to get a clearer sense of the cost, time, and potential impact of the research before embarking on a costlier, large-scale trial.

Pilot studies are essentially the smaller version of a larger proposed trial conducted over shorter periods of time with a smaller number of participants.

They allow researchers to identify any shortcomings in the study design and to work out the kinks that may exist in the study protocol.

Pilot studies can also be used in clinical trials to test different drug doses, routes of administration (pills, injection), drug safety, and any barriers that may undermine treatment adherence.

Funding of Pilot Studies

Pilot studies are usually paid for from a variety of sources. Grants awarded by the governmental, non-governmental, and non-profit agencies are most often the first sources of funding. Depending on the field of research, funding may come from pharmaceutical manufacturers or other allied industries.

By and large, private sector organizations are less common sources of seed funding and tend to step in when a drug either has greater market potential (such as with a new vaccine or drug class) or is near market-ready. Major pharmaceuticals today are more likely to acquire the lucrative drug rights by buying a company outright rather than investing in smaller, foundational research that may not go anywhere.

Much of the current funding for pilot research comes from governmental agencies like the National Institutes of Health (NIH) or from non-profit charities focused on a particular area of research. Well-known examples include the Bill and Melinda Gates Foundation (HIV), the Susan G. Komen Foundation (breast cancer), and the Michael J. Fox Foundation (Parkinson's disease).

New Trends in Research Funding

Getting an NIH grant can be difficult and time-consuming, so much so that it can take the principal investigators away from the very research they hope to conduct. Because of this, it can difficult for smaller research teams to access funding.

Many hopefuls try to overcome this by teaming with institutions, colleges, or research hospitals which have the resources to sustain such efforts. Doing so, however, often require a person to surrender or share certain rights to research.

As a result, some entrepreneurs have begun to embrace crowdsourcing as a means to advance their research while retaining most, if not all, of their rights. There are two models commonly used for this:

  • Crowdsourcing for science wherein the internet is used as mean by which individuals can freely contribute to active or ongoing research, often for altruistic reasons.
  • Crowdsourcing for funds (crowdfunding) by which researchers make a direct appeal to the public through crowdfunding sites like Kickstarter and TechCrunch. As opposed to the traditional funding model which requires peer-reviewed, evidence-based science, crowdfunding efforts are largely aimed at capturing the public's imagination. Public funding of these projects is also largely altruistic.

    Examples of crowdsourced science involve inviting members of the public or scientific community to conduct surveys, donate genetic test results, or run computer simulations to collaboratively find solutions.

    Crowdfunding research, by contrast, has come under scrutiny for aiming at the heart of the public without having to provide the breadth of evidence needed to support the scientific claims.

    In 2014, the Oakland, California-based Immunity Project raised $460,570 for a synthetic HIV vaccine which they promised to provide free to the public if it works. While there is no evidence to suggest that their intent is anything less sincere, there has also been no evidence of feasibility either.

    Source:

    Hayden, E. "Crowd-funded HIV vaccine project sparks debate." Nature; published February 10, 2014.

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