News from Brussels

Currently in Brussels for the Treat NMD Advisory Committee for Therapeutics Conference.

How many times have we all received Google Alerts suggesting a certain drug "is potentially promising," “improves function in the mdx mouse,” or ‘halts degeneration in the mdx”? And then, silence.

And how many of us have discussed the need for a certain drug to go to trial or how many parents/families have tried to persuade or were successful at persuading their physician to write the script for a drug based on a single paper about a mouse?

Some time ago, we were asked to recommend a drug for trial in Duchenne. What drug? What evidence? What does the trial look like? How will we measure success? What should we expect? A band-aid? Better? Better than steroids? Will it end Duchenne?

Now there is a process in place in order to objectively review what is known about a certain compound, what level of evidence, what barriers/obstacles might exist in the process of clinical trial development. At the end of the day, this process will help us prioritize drugs for trial and to the degree humanly possible, improve our likelihood for success.

TACT – Treat NMD Advisory Committee for Therapeutics

Treat NMD has established a committee and process to review drugs for trial in Duchenne. This committee provides broad-based expertise to help identify the potential of therapeutics and guide promising ideas to a realistic development path.

Drug development has dramatically changed in the last 12-18 months. The era of the blockbuster is coming to a close and is replaced by drugs targeted to subgroups with the goal of better outcomes for patients. Tools exist to revisit molecules used for other diseases and/or previously considered not useful. It is important for proof of concept trials which are crucial in exposing failure (of the molecule) before raising patient’s expectations and spending vast sums of money.

It is good timing for the TACT process because orphan drugs in chronic diseases with previously underestimated markets, are generating deals. (PTC/Genzyme; Prosensa/GSK; Summit/Biomarin)

The process of TACT will be highly educational and transparent. It will provide a comprehensive approach for review and a perspective so that plans for clinical trials can move forward with some surety. At the moment, TACT has a list of 46 that could be considered potential candidates for trials. The TACT will ensure a level of objectivity so that the most promising compounds are moved into trials. We have a limited number of boys and want to work really hard to do the best job, testing the best drugs with a high likelihood for success.

TACT process will include:
• Consistent standards of drug development considerations for an informed review
• Scientific rational (objective - preclinical support)
• Study design
• Study conduct
• Did the right experts and groups have input?
• Will it inform next steps (go/no go) and the field in general?
• Plans for Funding, initial and beyond

The goal is to objectively review and provide clear answers for developers to bring drugs to patients (Faster to clinic vs best informed to clinic).

Who can use TACT?
Researchers, funders, clinicians, industry

You can check out an application at: http://www.treat-nmd.eu/TACT/TACTFormNov09.pdf

I'm extremely enthusiastic about TACT - It will help us make the RIGHT decisions about moving drugs into trial.

More to come...