Emerging therapies to get a class of their own

Once upon a time, biological therapies were plant extracts, tinctures and pills. Physicians also had at their disposal a variety of medical devices. But as new technologies have emerged in the last few years the boundaries between these categories have become increasingly blurred and novel therapies such as pancreatic islet transplantation, haemopoietic stem cells and cellular based vaccines have presented themselves for categorisation by the Therapeutic Goods Administration, under a regime which had, perhaps, become a little outmoded.

“One limitation we’ve had in the TGA is that we only have two classes of therapeutic goods,” says Albert Farrugia, head of the TGA’s blood and tissues unit. “A therapy was either a medicine or a medical device. In terms of mainstream manufacture that serves you well, but for these new therapies that has limitations.

“What little there is in the current regulatory framework of the Therapeutic Goods Act exempts these therapies on the basis of them coming under the classification of medical practice rather than manufacturing.

“Therapies [often now] involve manufacturing, manipulation, and modification of the cells and tissues. They have moved away from the straightforward medical environment.”

When the new regulatory framework replacing the current Therapeutic Goods Act of 1989 comes into force -- a change expected to take place on July 1, 2005 -- it will establish an office with the specific brief of overseeing these new therapies.

“The area we’re developing is the area of regulation of what we loosely call emerging biological therapies – mostly those therapies based on transplantation of cells and tissues,” says Farrugia. “The framework will involve the regulation of all cell and tissue therapies, involving all therapeutic goods which are composed of cells and tissues which are infused or implanted in a human being.”

The new framework has been in gestation since the late 1990s, and has already gone through a series of developments, issue papers, ministerial directives and public consultations.

Therapies with lower levels of risk such as organ transplantation will be monitored to minimise infectious disease risk, and the level of scrutiny will climb for tissue banking and be even more stringent for cell transplantation, and cellular based vaccines, where GMP procedures, pre-market review and clinical trials may be required.

The system of exemptions will also be changing, and only normal medical procedures will be excluded from regulation. “At the moment the exemptions are a hotchpotch,” says Farrugia. “What there will be is one general exemption for everything which is still considered medical practice -- without intervening manufacture or manipulation, within one procedure.”

A new expert advisory group will also be established, bringing together transplant physicians and surgeons working in the area. It is expected to be chaired by the government’s chief medical officer, Prof John Horvath.

The US Food and Drug Administration now has a comprehensive framework dealing with medical developments in transplantation and manipulation of tissue, as do the EU and Canada. “We’re anxious to harmonise as closely as possible to the FDA model, because many of the groups [working in these fields] in Australia are also making Investigational New Drug applications to the FDA,” says Farrugia.