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Scaling compassion

FDA, industry, patient groups charting compassionate use policies

Nearly two years after a viral media campaign saved seven-year-old Josh Hardy's life and put demands for access to unapproved medicines on front pages, drug companies, FDA, academic bioethicists, and patient advocates are beginning to chart a new course for compassionate access.

Johnson & Johnson is staking out a leadership position among pharmaceutical companies with a pilot program that uses an independent external advisory committee to review compassionate use requests for an investigational drug. The company told BioCentury it has learned several lessons about how to manage compassionate access that it plans to apply as it expands the program to additional drug candidates.

So far, J&J is an outlier. No other large pharma company has devoted comparable resources to preapproval access or made a similar commitment to independent third-party review of requests, and few small companies have put comprehensive plans in place.

Non-profit patient groups and for-profit companies are working on road maps to help physicians and patients navigate compassionate use, and some organizations are creating models for taking on the logistical responsibility and, in some cases, the expense of operating expanded access protocols.

In addition, some divisions at FDA are proactively working with sponsors to integrate expanded access programs into clinical development and encourage companies to provide treatment options for patients who are ineligible for trials.

Several of the experiments under way - if widely adopted - could lead to a more equitable and transparent compassionate use process for patients and their families. But none is sufficient to ensure that another patient like Hardy would be treated with a lifesaving experimental medicine, or that companies will be spared aggressive campaigns to provide access.

That would require a comprehensive effort by drug companies to create transparent and fair pathways for patients who have run out of options to obtain access to investigational medicines outside of clinical trials.

External advice

At a recent colloquium on preapproval access at the New York Academy of Science, J&J reported that its Compassionate-Use

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