Introduction:Where Do Physicians and Payers Signal the Greatest
Need—and Greatest Opportunity—for New Therapeutic Advances?
The market for disease-modifying therapies for the treatment
of relapsing-remitting multiple sclerosis (RR-MS) is active and commercially
compelling. Despite a small diagnosed prevalent population relative to many
other neurological indications, drug-treatment rates for RR-MS are high, and
neurologists emphasize early treatment intervention to improve long-term
outcomes in this chronic disease. The number of disease-modifying therapies
approved to treat RR-MS is expanding steadily—including the launches of the
first oral disease-modifying drugs in this historically injectable market—but
marketed agents have drawbacks that leave ample opportunity for new therapies
offering clinical improvements in efficacy, safety and tolerability, and
Questions Answered in This Report:
A drug’s performance on at least eight efficacy end points,
including a reduction in the annualized relapse rate (ARR) over two years, is
important for drug approval and physician use. What are the key primary and
secondary clinical trial end points on which new therapies are evaluated? How
do U.S. and European neurologists weight efficacy measures and other drug
attributes in their prescribing decisions for RR-MS?
A greater effect on sustained disability progression and
fewer/less burdensome monitoring requirements are key areas of unmet need in
RR-MS, according to the insights of surveyed U.S. and European neurologists. Which
emerging therapies, if any, are poised to fulfill these needs? What clinical
and/or regulatory challenges must drug developers overcome in order to
capitalize on these areas of unmet need? What degree of improvement over
currently available therapies do surveyed U.S. MCO PDs seek from new
disease-modifying MS therapies on key clinical attributes for which surveyed
physicians indicate there is high unmet need?
The incidence of serious or life-threatening side effects and a
reduction in the ARR relative to placebo are key drivers of physicians’
prescribing decisions and/or are the focus of drug development of new RR-MS
therapies. What trade-offs across these and other clinical attributes are
U.S. neurologists willing to make when considering the use of emerging
disease-modifying therapies for RR-MS? Based on the trade-offs in price and
performance across key drug attributes that U.S. neurologists are willing to
make, how do physician preference and prescribing likelihood vary across
different target product profiles for RR-MS?
By 2016, Genzyme/Sanofi/Bayer HealthCare’s alemtuzumab
(Lemtrada) will emerge as the gold-standard therapy in our Drug Comparator
Model because of its superior clinical profile over the key current therapies
we evaluated. On what clinical attributes is alemtuzumab most differentiated
from its competitors? Which current therapies are at greatest risk of being
replaced by alemtuzumab?
Attributes included in
conjoint-analysis-based assessment of target product profiles for RR-MS:
- Reduction in ARR relative to placebo.
- Reduction in risk of six-month sustained disability progression
(i.e., Kurtzke Expanded Disability Status Scale [EDSS] progression) relative to
- Incidence of serious or life-threatening side effects (e.g.,
opportunistic infections, bradycardia, autoimmune adverse events).
- Incidence of less-serious side effects (e.g., injection-site
reactions, flu-like symptoms, flushing).
- Monitoring burden: frequency (e.g., first-dose only, monthly)
and complexity (e.g., number/difficulty of unique tests).
- Delivery burden (i.e., dosing frequency and formulation).
Attributes included in assessment of U.S. payers’
receptivity to new therapies for RR-MS:
- Effect on ARR.
- Effect on risk of six-month sustained disability progression.
- Monitoring burden.
- Burden of delivery (dosing frequency and formulation).
Physicians surveyed: 61 U.S.
and 30 European neurologists.
Payers surveyed: 20 U.S. MCO PDs.
Comprehensive List of Therapies Included in Our Research and
- Glatiramer acetate (Teva’s Copaxone)
- Interferon-beta-1a intramuscular (Biogen Idec’s Avonex)
- Natalizumab (Biogen Idec/Elan’s Tysabri)
- Fingolimod (Novartis/Mitsubishi Tanabe Pharma’s
- Teriflunomide (Sanofi/Genzyme’s Aubagio)
- BG-12 (Biogen Idec’s Tecfidera)
- Alemtuzumab (Genzyme/Sanofi/Bayer HealthCare’s Lemtrada)
- Daclizumab (AbbVie/Biogen Idec)
- Ocrelizumab (Roche/Genentech)
- Laquinimod (Teva/Active Biotech)