Summary
There exists an important
link between scientifically proving a drug’s efficacy and safety and
maintaining the confidence of investors who fund the clinical trials; one
without the other likely means the drug will fail. When the drug company Zafgen
entered the third stage of clinical trials for their drug, Beloranib
(originally used to treat cancer, then tested for weight loss efficacy and,
ultimately, the treatment of the rare Prader-Willi Syndrome which causes insatiable
hunger in children), the death of two patients lead to investors pulling out
and stock prices plummeting from $35/share to $7. Despite proof that both
deaths were caused by a common condition to Prader-Willi patients not receiving
treatment, investor confidence appears to be shaken and share prices have not
returned to their original value. For small drug companies, like Zafgen, who do
not have multiple high-dollar revenue streams or alternative, ongoing research
opportunities, the results of a patient death (including long-term holds placed
on future trials and subsequent loss of investor support) often mean the end of
the business and the drugs for which patients throughout the country have been
waiting.
Link to Podcast: https://www.statnews.com/2016/04/04/podcast-clinical-trial-death/
Novartis introduced a breakthrough leukemia medication, Gleevec, 15
years ago with a list price of over $26,000 for a year; today, despite common
market standards that expect competition to lead to price reduction, a year of
Gleevec costs over $120,000. Unlike drugs sparking outrage with sudden,
unexplained price increases, Novartis chose to gradually increase the cost of
Gleevec on a year-over-year basis, oftentimes with the release of competitors
to the market. The rarity of the form of leukemia treated by Gleevec was,
according to Novartis, a partial explanation for the high cost; if more
patients took the drug, they would be able to lower the cost and still recoup
expenses. Today, Gleevec’s effectiveness has increased the population of
individuals living with the cancer by 300% but Novartis’ original pricing
explanation remains unseen. Novartis’ run with Gleevec has likely come to an
end, though, as the drug lost patent protection last month.
Read the Washington Posts article about it here.
The White House budget
proposal, released earlier this month, includes a requirement for
pharmaceutical companies to disclose various business measures, including the
cost of research and development; a requirement that would either prove or lay
to rest to pharma’s continued argument that drug prices are directly related to
their R&D costs. The measures mimic those proposed by states across the
country and received similar backlash from industry lobbyists – including
assertions that the requirement would stifle innovation, ignores R&D
failures, and requires the disclosure of confidential information. That final
argument is likely countered by an existing statute in the Affordable Care Act.
Read the coverage from Pharmalot's Ed Silverman here.
As
bacterial infections are becoming increasingly resistant to existing
antibiotics, the University of Illinois has released promising research
regarding the dual use of multiple drugs, including those used to treat parasitic
infections and cancers. In fighting their intended diseases, many FDA-approved drugs also kill bacteria, work as uncouplers to destroy bacterial energy
production and, in some cases, inhibit enzyme production and disrupt the cell
membrane. Testing has primarily focused on the treatment of tuberculosis and
staph infections. The researchers are also investigating tweaking the biology
of certain drugs that are metabolized within the cell (eg. Heartburn drugs) to
create an added level of uncoupling and, ultimately, cell death of bacteria.
The Senate Finance
Committee has determined that the prices charged by Gilead for the Hepatitis C
drugs, Sovaldi and Harvoni, do not reflect the actual research or cost incurred
in producing the drugs and is officially accusing Gilead of placing profits
ahead of affordability and accessibility. In 2014, Medicare and Medicaid spent
more than $5 billion on Sovaldi and Harvoni. Gilead released a statement saying
that with available rebates and discounts the cost is actually less expensive
than that of prior treatments, and, additionally, the improved efficacy of
their drugs reduce the cost of future medical complications.
To read the full Reuters article click here.
Pfizer’s announcement that it will buy Allergen
(the producer of Botox) will shift the manufacturer’s headquarters to Ireland
and help reduce their incurred domestic taxes by an estimated 8%. The deal will
make Pfizer the largest company in the industry, as well as mark the largest
instance of an international acquisition to lower a U.S.-based company’s taxes
– and has earned direct criticism from President Obama, Hillary Clinton, Bernie
Sanders, and Donald Trump. Both companies saw a drop in their stock prices
after the announcement.
Read Reuter's article on it here.
