Editorial Content Director, Orphan Drugs Summit
A note from the editor
Changing times ask for solid relationships.
Times are changing. A renewed interest from big pharma in the rare disease landscape has awakened due to large-scale patent expirations, competition from generics & biosimilars, anemic pipelines, escalating clinical trial costs and a global health-care reform. This means that the traditional blockbuster model has become less viable while the revenue-generating potential of orphan drugs has shown to be huge with a greater return on investment than non-orphan drugs. According to EvaluatePharma, the orphan drugs sales will grow at an annual rate of 11% and constitute 19% of the total share of prescription drugs by 2020, totalling 176 billion dollars.
Finally, the pricing environment remains favourable, but payers will be looking to
manage escalating drugs bills as patent expiries slow in coming years. Although orphan
populations are by definition the smallest, they represent big per-patient outlays, and
insurers will be looking carefully at new tools to arrest cost growth as more and more
orphan drugs launch. Expect more pressure on the drugs that represent the biggest
budgetary drain, especially as increasing numbers of US patients will be receiving public
subsidies with the implementation of the Affordable Care Act.
Looking ahead, there does not appear to be much to slow down the sector’s rush into
orphan indications. Revisions to the US Orphan Drug Act do tighten up some of the
language in order to prevent manufacturers from creating subsets of patients that could
inappropriately qualify as an orphan indication, but does not appear to have a major effect
on development. With designations continuing to be on the rise, in all likelihood the
number of orphan drugs that successfully complete the clinical and registration stages will
be on the rise. However, the US has created the new breakthrough therapy category, so it
is possible that certain orphan-type drugs could be diverted into that regulatory scheme.
Reduced R&D Costs
USA: 50% Tax Credit on R&D Cost
USA: R&D Grants for Phase I to Phase III Clinical Trials ($30m for each of fiscal years 2008-12)
USA: User fees waived (FFDCA Section 526: Company WW Revenues
AKon, you're a great historian. Tell us again, the history. Haha.
You're shorting a 6 cent stock with all the catalysts online this year? You're toast.
That's just one big (US) market. Double that for US + EU. Break into Asia - and its even crazier money.
I've purchased more shares than I ever intended. Especially this year, I'm ALL in. GLTA
I am down, but I've been buying steady since I've learned about DCTH. It will rollover and move the other way for me, soon enough.
Poor guy. Haven't learned to take responsibility for your own decisions yet, eh? I'll buy your remaining shares, so you can let go and move on.
Yep. Don't buy Akon. Sell, and I'll buy your shares. Great spot to average down. Stock is being sold at a discount.
Delcath Adds New U.S. Center To Global Phase 2 Hepatocellular Carcinoma Program
Montefiore Medical Center in New York Opens for Enrollment
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NEW YORK, April 7, 2015 /PRNewswire/ -- Delcath Systems, Inc. (NASDAQ: DCTH) announces the expansion of its global Phase 2 program for the treatment of patients with unresectable hepatocellular carcinoma (HCC) or primary liver cancer. Montefiore Medical Center in the Bronx, New York has joined the U.S. Phase 2 HCC study and is now open for patient enrollment. The Company now has two centers participating in the U.S. Phase 2 HCC study, with another three centers in Germany enrolling patients in the EU Phase 2 HCC study. The Company expects to include up to seven centers in Europe and the United States in its global Phase 2 HCC program, and will seek to enroll approximately 30 patients in total.
HCC is the most common primary cancer of the liver, with approximately 700,000 new cases diagnosed worldwide annually. Surgical removal is not possible for an estimated 80-90 percent of primary liver cancer patients. In the U.S., the Phase 2 study will investigate the safety and efficacy of Melphalan/HDS treatment followed by sorafenib in patients with unresectable liver cancer confined to the liver, evaluate tumor response (objective response rate), as measured by modified Response Evaluation Criteria in Solid Tumor (mRECIST), assess progression-free survival, safety, and the safety of sorafenib following treatment with Melphalan/HDS. Additional analyses will be conducted to characterize the systemic exposure of melphalan administered by Melphalan/HDS, as well as an assessment of patient-reported clinical outcomes, or quality-of-life.
"We are pleased to have Montefiore Medical Center, a nationally recognized Center of Excellence for cancer care and clinical research, as a part of our global Phase 2 HCC program," said Jennifer Simpson, Ph.D., Delcath's Interim President and CEO. "We now have five centers open for patient enrollment in the program, and with our prospective commercial registry in Europe also now open for enrollment, we are continuing to execute on all elements of our Clinical Development Program."
The takeaway from Ocular's after hours plunge on Monday, however, is that people who are asking if there is a biotech bubble will see this latest blow up as further evidence of a sector that has become overheated, as investors are fleeing for the exits at the first sign of bad news.
But the thing to keep in mind is that investing in biotech stocks isn't like buying a share of Apple or General Motors. These stocks have the potential to go to the moon... or quickly head towards zero when trial results don't go their way.
The market is treating Delcath as a going concern. Ridiculously cheap PPS down here. The exciting factors are the variables not discussed often anymore. Off label, partnering, buy out by BP, doubling procedures, EU reimbursement validation, Orphan Status. Loading up here before May. GLTA