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It is important to remember that MS is only one part of a person and not the person.

MS does not represent them, it is not their identity, they are as normal as the next person.  

Treatment Paradigm

With nearly a dozen new multiple sclerosis drugs in some phase of mid-to late-stage development - from the first oral options to infrequently administered biologics - the treatment paradigm for the debilitating disease appears on the cusp of significant change.

The idea that patients and physicians will jump at the chance to try or prescribe new disease-modifying agents seems obvious, given that existing drugs require injection dosing and have moderate long-term efficacy. In MS drug development, the hope of an oral treatment is often held out as the "holy grail."

But given the generally long-term progression of relapsing remitting MS, the young age at which many patients are diagnosed and the relative safety of existing medicines, drug switches might take longer than some expect.

While there is certainly a race among drug makers to be first-to-market with a breakthrough MS treatment, there's no guarantee that the stampede to try them will be as aggressive, at least initially. Pending final efficacy and safety data, it's quite possible that even a convenient oral option could be relegated to the second-line setting.

Neurologists Skittish On Side Effects                                                                                                                                                                                       

Neurologists specializing in MS, speaking during a panel discussion at the BIO CEO & Investor conference Feb. 10, appeared eager to add to their treatment arsenal. But at the same time, enthusiasm for the late-stage drugs in development was subdued given the potential for risky Serious events.

"One can imagine that if the first available oral drug were to have roughly similar efficacy and tolerability and safety measures [to current options] that probably that drug would steal the market," said John Richert, Exec VP for Research & Clinical Programs for the National Multiple Sclerosis Society

However, he added, "if the first or any of the oral drugs turned out to have the same or better efficacy data but turned out to have safety issues, then we've got the issue of will they be first-line drugs or second-line drugs, and I think there is not enough information to sort that out."

Unfortunately, what seemed a stellar field of potential MS candidates in early development has lost some lustre in larger later-stage trials on the long and winding road toward regulatory approval (1Pharmaceutical Approvals Monthly June 2006, p. 3). Serious side effects, including malignancies and autoimmune disorders, have emerged with some of the highest profile late-stage candidates.

Serious side effects may be problematic to neurologists, who are comfortable with the safety profile of existing first-line treatments for MS, mainly Biogen Idec's Avonex (interferon beta-1a), Merck Serono's Rebif (interferon beta-1a) and Teva's Copaxone (glatiramer). Avonex and Rebif carry warnings for depression and suicide, anaphylaxis and hepatic injury. Labelling for Copaxone doesn't include any warnings.

"You're thinking about this young, otherwise healthy woman who might want to have children in the next year or two and that plays a role," said Mark Tullman, Director of the Multiple Sclerosis Clinical Care and Research Center at Columbia University, referring to neurologists' treatment decisions and traditional newly diagnosed patients, frequently women in their 30s.

"The first generation of therapies has just been so remarkably safe, and that's a little bit of a problem I think for us and our colleagues," he said.

Physicians in areas like rheumatology and oncology have had more experience balancing the benefit/risk scale for drugs with serious side effects. "Neurologists are pretty new to this game," Richert said. "I think over the next five years or so neurologists might become more like rheumatologists when it comes to their ease at prescribing drugs that carry a small percentage of very risky side effects."

With Efficacy Comes Opportunity                                                                                                                                                                                               

What does bode positively for potential new drugs, however, is the modest efficacy of the interferons and Copaxone, and the clear unmet medical need that remains for patients. Drugs that show a clear efficacy benefit on disability outcomes - oral or injectable - will have a much clearer market opportunity.

"Disability by and large is really the gold standard," Richert said. "There are studies in Phase II trials that look at relapse rate and MRI findings, [but] personally, if it is not disability data, I tend not to pay much attention to it."

That said, patients are clearly unsatisfied with existing medications due to the injection schedule and breakthrough disease, Richert added. He pointed to data showing that about 12 percent to 15 percent of patients who start on the first-line drugs stop treatment permanently; another 25 percent who continue the drugs are not satisfied with treatment.

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