PharmExec Blog

Expanded access – Myths, Truth and Behavior

Not all companies are big, small companies are scared, and myths drive behavior, stated Kay Holcombe, Senior Vice President, Science Policy at Biotechnology Industry Organization (BIO).

The National Organization for Rare Disease (NORD) ended its Rare Diseases and Orphan Products Breakthrough Summit in Alexandria, Virginia on Wednesday with a lively discussion on expanded access.

Holcombe’s statements came after Nancy Goodman, Founder and Executive Director, Kids v Cancer described the case of Josh Hardy, an 8-year-old boy, suffering from cancer who developed an adenovirus infection after being immunosuppressed following bone marrow transplantation. The family, along with mounting public pressure from Facebook, Twitter, etc., demanded a chance at Chimerix’ anti-viral drug, brincidofovir (CMX001) via expanded access, as Josh was not eligible to take part in the company’s trial.

Holcombe was also preceded by a pair of FDA officials, Richard Moscicki, Deputy Center Director for Science Operations, Center for Drug Evaluation and Research (CDER), and Amy McKee, Clinical Team Leader, Office of Hematology and Oncology Products, CDER, who attempted to dispel myths surrounding expanded access – chief of these myths being that granting expanded use for compassionate treatment may result in negative impact on a drug’s adverse event data.

Both FDA officials stated that after reviewing significant FDA proceedings, they had put to rest the idea that AEs in expanded access had affected the approvability of any product they had seen in their time at FDA.

Holcombe insisted that there needs to be greater transparency into how FDA makes decisions about using data from expanded access.

Small companies are desperately afraid of something going wrong, and FDA needs to do a better job helping companies understand what will happen if something goes wrong in expanded access, she said.

Though they look heartless, these companies do care about patients, Holcombe stressed. But the company’s priority remains focused on its primary trial(s).

Additionally, companies worry about fairness and do not want to be put in a position where they grant expanded access to one patient then have to say “no” to another.

One FDA official emphasized a need for balance as these patients are desperate; the potential for therapeutic benefit is often overstated, and the risk, understated. Patient autonomy must be balanced as well as the benefits to the individual versus the potential societal benefits.

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Pharma Feels Impact of US Tax Inversion Legislation

The recent termination of AbbVie’s deal to acquire Shire has “jolted the industry out of its reverie” by becoming pharma’s first major casualty of new US tax inversion legislation,  says research and consulting firm GlobalData.

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US Healthcare Reform: A Pharm Exec Digest Special

This month’s special issue of Pharm Exec Global Digest looks atUS_healthcare_reform_special

  • Dealing with the “side effects” of Obamacare
  • The “value” of Rxs under Obamacare
  • The escalating cost of US oncology care
  • And more…

Click here to read the issue.

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The Shrinking CMO Industry

By Jim Miller.

Exits from the CMO industry are a more recent development that seems to be picking up momentum. Examples of CMOs leaving the industry in the past year include the contract manufacturing units of Bayer and Boehringer-Ingelheim; Pharmalucence; and SCM Pharma. There have been two principal drivers of these developments: growing demand for captive manufacturing capacity and regulatory compliance difficulties. Read More »

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The Baltic States: A Location for Your Next Clinical Trial

The Baltic States with its appropriate population size, sufficient infrastructure, and experienced and motivated investigators, make it an attractive choice for a clinical trial.

In this article, Doatas Grina, Indre Tutlyte and Auudius Sveikata, PhD, that ran in the February 2013 issue of Applied Clinical Trials, presents a persuasive case for the area’s present desirability as well as a history of steady growth in the past that indicates the Baltic clinical research market is poised to grow. Already it has proven rich in clinical research potential. Consider:

  • The Baltic States approve around 80 new clinical trial applications per country each year.
  • The biggest part of clinical trials performed are within the therapeutic areas of oncology, neurology/psychiatry, and endocrinology.
  • Nearly 70% of the studies performed are Phase III trials.
  • States legislation is harmonized and the regulatory process is similar to other EU countries.
  • The timelines are attractive—the approval process takes approximately 60 days.
  • The region has an appropriate population size to populate large trials.
  • Its infrastructure is sufficient and their investigators are experienced and motivated.
  • A considerably large pool of treatment-naïve subjects is available, similar to most of the Central and Eastern European counties. Read More »
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