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HD InsightsBringing the Trial to Life: Scientific Advisor

As Chief Scientific Officer, I chair the HSG Scientific Advisory Council, with Co-chair Sandra Kostyk. We generally play the most active part before before the trial starts. We have interactions with many companies, including during the preclinical stages or when they’re planning their trials, and particularly when it is a first trial.

Pre-Trial Guidance

Scientific Advisors offer guidance on issues like patient selection, outcome measures, how long the trial should be, whether and what kind of control group there should be — all important input to help companies design their trials. Some of these are very basic questions that help shape a trial in a way that will make it successful.

The Study Principal Investigator (SPI) often has not been designated by the time we are providing our advice, but if they have, they are a key part of the process.

Hand-in-Hand with Patient Advisory Boards

To provide this guidance to sponsors, we will often work with Patient Advisors (aka Patient Advisory Board or Community Advisory Boards), soliciting their views on what will be required to ensure trial participation and retention. Sometimes this enables us to really make a big difference in terms of how to focus the trial and how to make it more efficient and more likely to have useful results.

Once a Patient Advisor or Patient Advisory Board has been designated, a Scientific Advisor such as myself or others will present the concept, the scientific rationale and the outline for the trial in order to elicit reactions of patients and families and help guide the conception of the trial.  

After Start-Up

Once the trial is underway, we mostly serve in a consulting role, as needed. When questions are posed to one of us, we may bring in other members of the Scientific Advisory Board, or others with the relevant expertise.

Sponsor Relationships

We have an ongoing relationship with many sponsors. When we go to CHDI conferences, for example, we will be talking to lots of different companies, keeping in touch, seeing how their programs are developing and what we can do to be helpful. This way, we hopefully foster a relationship where they ask us to run their clinical trials in the future.

In some cases, those contacts blossom into a clinical trial, and in other cases we just work to maintain contact, holding the community of people interested in HD together.

Essentials of a Scientific Advisory Board

Most people who take on this role are very invested professionally in the cause of fostering HD treatment and cure. I would say we all seek to serve as a continuous backdrop to the story of HD research, putting in the new pieces of the puzzle as they appear and adjusting the lens through which we view HD and HD research.

Scientific Advisors have many areas of expertise — basic neurobiology, genetics and pharmacology, clinical trial design, statistics, pharmacokinetics, clinical medicine, ethics, and of course care of individuals with HD. In my case, I have both clinical and basic science expertise, which I feel is helpful in integrating the many aspects of clinical trials.