iStock_000027325808_SmallLast week, Chief Executive Paula Chadwick of the Roy Castle Lung Cancer Foundation in the United Kingdom labeled delay in NHS patients' access to cancer drugs a “disgrace” in an interview in The Guardian. The foundation was complaining that patients will not have access to immunotherapy drug Opdivo (nivolumab, BMS) until May 2016, after the National Institute for Health and Care Excellence has its cost-effectiveness review. Ironically, now that the drug is approved by the European Medicines Agency, availability under the Early Access to Medicines Scheme will stop. This is causing an extensive gap in availability of a drug treatment that is seen as a paradigm shift in the treatment of lung cancer. In the same article, Chadwick states: “The pharmaceutical companies want their price and the NHS has its processes, but it’s the patients here who get caught in the crossfire.” Is seems indeed reasonable to ask both parties to come to a better solution for patients.

The Timing Dilemma

Timing is posing a dilemma in many ways when considering market access for prescription drugs. Given a limited patent life, drug companies need to take their innovations through development at the fastest pace possible, but need to also ensure that the evidence package supports data requirements from both regulatory requirements and diverse payer requirements globally. A delay in launch not only limits the payback period, but also can result in being a second or third to market rather than the leader in a new drug category. For some individual patients, a delay in availability can mean a difference between life and death.

Government and private payers will understandably argue that ensuring rapid access to new drugs should not withhold them from making critical decisions in ensuring that drug expenses stay within a reasonable budget. Particularly as many new specialty products are coming to market, managing utilization of these high-cost drugs is seen as critical by many budget holders. However, how can we make sure that we maintain the right balance between opportunities to save lives and budget challenges?

Evidence at Launch

Particularly with the increasing interest in real-world data, the gap between payer wishes in terms of evidence, and what can be realistically demonstrated at the stage of regulatory (randomized clinical trial-based) marketing authorization is growing. A recent Tufts study on the cost of drug development states that recent rapid increases in the cost of drug development can be in part attributed to increasing payer evidence requirements. I argued in a previous blog (Cost of Developing a New Drug) that at least some restraint in evidence requirements by payers is in their own interest. For particularly innovative treatments, evidence of long-term outcomes is usually not available at launch. For those cases, we need to find a way to ensure that these treatments are available to patients, while monitoring long-term performance over time. Demonstrating impressive survival improvements inherently takes much longer than showing unimpressive ones. Payers and industry may have to find more innovative ways to address reasonable payment for yet uncertain long-term outcomes without withholding the treatments. Let’s talk …

Topics: pharmaceutical industry, Pharma Industry, Market Access & Pricing, Drug pricing, Ed Schoonveld