The “compassionate use” issue received national attention in March when the parents of 8-year-old Joshua Hardy requested and, ultimately, received for their son, access to brincidofovir, an anti-viral drug not as yet approved by the FDA.
Our question is about public access here in the United States to investigational drugs that have not yet been approved by the FDA, commonly referred to as ‘compassionate use’ policies. We’re coming from a public policy perspective — that is, we’re interested in knowing if Company management believes there are things that can be done by government or the health care industry, or changes to laws and regulations, or even funding, to streamline access to unapproved drugs for patients who are literally down to their last hope.
I think it’s very important… Pfizer has always been very supportive of the use of pre-FDA-approved products in compassionate use, and we have a long history of providing them.
I would say, to answer your question more broadly, that pharmaceutical industries as represented by its trade association — PhRMA — is having dialogues with the FDA and with other stakeholders on how to improve and to simplify the access of people to compassionate use, balancing it with the ethical needs to run clinical trials that will produce the evidence that will allow a larger population to use those medicine in… knowing their safety and knowing their efficacy.
We’re very proud of many of the investments that we’ve made in research and development over the past several years. We’re very proud of many of the investments that we’ve made in research and development over the past several years that we think have ended up having a remarkable impact as they’ve gone through extensive clinical trial and development programs so that we can ultimately bring them to market.
Dr. Paul Stoffels, chief scientific officer for pharmaceuticals at Johnson & Johnson, added:
We have a lot of new drugs which are life-saving and which are very important for cancer patients and even people with serious infectious diseases and other diseases. And we are working very diligently with the regulators as well as with the with the health care community to provide access as soon as possible.
Of course, we always have to take into consideration the safety and efficacy before we expand it into the larger population.
We have had, for tens of thousands of patients, therapies out for HIV; for many, many patients in oncology… we provide significant access to patients who need a drug to survive as soon as possible.
It’s our life to provide medicines to patients, and we — as physicians in the company — we do our ultimate best to make sure we can as rapidly as possible make life-saving drugs available.
“I was a bit disappointed by Gorsky’s and Stoffels’ responses,” said David Hogberg. “I was hoping for a bit more detailed answer. It’s great that they are trying to get new drugs to the public rapidly. But if a cancer drug still has three years to go before FDA approval, that does little to help a cancer patient for whom that drug may be the only hope of extending his life. I had hoped that they had some specific ideas on how to improve practices regarding compassionate use.”
Regarding Pfizer’s response, Jeff Stier said, “We appreciated the opportunity to speak with Pfizer CEO Ian Read today, and with other Pfizer executives on other occasions, about the challenge of crafting the best-possible compassionate use policies. Everyone wants to save lives. The challenge is to have policies that work to save as many lives as possible in both the short run and the long run. We are encouraged to learn from Mr. Read that a process is ongoing between the pharmaceutical industry and the FDA to ‘improve and simplify’ compassionate use access while protecting the valuable data learned through clinical trials.”
National Center Chairman Amy Ridenour added, “Compassionate use policies are one of the most difficult, if not the single-most difficult, regulatory challenges the country faces. The challenge lies in devising ways to save the most lives possible: extending compassionate use of drugs as much as possible to people for whom these drugs are their last hope, while not undermining or delaying the clinical trial process that gets appropriately safe and effective drugs to the broader public. Saving lives is the goal; everyone agrees on that, but how to design a system that helps as many people as possible is difficult.”
The National Center’s Free Enterprise Project is a leading free-market corporate activist program. In 2013, Free Enterprise Project representatives attended 33 shareholder meetings advancing conservative and free-market principles in the areas of health care, energy, taxes, subsidies, regulations, religious freedom, media bias, gun rights and many more important public policy issues. The National Center has participated in 11 shareholder meetings so far in 2014.